Ignite Creation Date:
2025-12-25 @ 2:34 AM
Ignite Modification Date:
2025-12-27 @ 11:13 PM
Study NCT ID:
NCT04209634
Status:
COMPLETED
Last Update Posted:
2025-08-29
First Post:
2019-12-20
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-10-10', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D011504', 'term': 'Protein-Losing Enteropathies'}], 'ancestors': [{'id': 'D007410', 'term': 'Intestinal Diseases'}, {'id': 'D005767', 'term': 'Gastrointestinal Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@regeneron.com', 'phone': '844-734-6643', 'title': 'Clinical Trials Administrator', 'organization': 'Regeneron Pharmaceuticals, Inc.'}, 'certainAgreement': {'otherDetails': "The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From signing of informed consent up to 164 weeks (end of study)', 'eventGroups': [{'id': 'EG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 10, 'seriousNumAtRisk': 10, 'deathsNumAffected': 0, 'seriousNumAffected': 5}], 'otherEvents': [{'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Acarodermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Iron deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Metabolic acidosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Alopecia areata', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Dermatitis contact', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Gingival bleeding', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Anaemia folate deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Immunisation reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 6, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Coronavirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Upper limb fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Folate deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Hypoalbuminaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Vitamin B12 deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Chronic spontaneous urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Skin mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Xerosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Activated partial thromboplastin time prolonged', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': '"Blood 1,25-dihydroxycholecalciferol increased"', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Blood bilirubin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Blood immunoglobulin M decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Blood zinc decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Eyelid oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Dysuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}], 'seriousEvents': [{'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Head injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Upper limb fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (27.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000', 'lowerLimit': '74.1', 'upperLimit': '100'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'At Week 24', 'description': 'Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of \\<2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The FAS included all enrolled participants who received the study drug.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Participants with TEAEs', 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': 'Participants with mild severity', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Participants with moderate severity', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Participants with severe severity', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From start of study drug administration up to approximately 144 weeks', 'description': "TEAEs are defined as AEs that developed or worsened during the on-treatment period. The on-treatment period is defined as the time from first dose of investigational product up to 21 weeks after the last dose of investigational product. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the patient's normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Improvement in Most Bothersome Signs and Symptoms at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'At Week 24', 'description': "Improvement in most bothersome sign/symptom determined using semi-structured concept elicitation interview, from 'core' clinical endpoints of frequency of bowel movements, peripheral edema, facial edema, abdominal pain frequency, nausea, vomiting, stool consistency.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS included all enrolled participants who received the study drug.'}, {'type': 'SECONDARY', 'title': 'Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.64', 'groupId': 'OG000', 'lowerLimit': '0.5', 'upperLimit': '3.4'}]}]}, {'title': 'Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.13', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '3.0'}]}]}, {'title': 'Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.36', 'groupId': 'OG000', 'lowerLimit': '0.7', 'upperLimit': '3.4'}]}]}, {'title': 'Week 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.43', 'groupId': 'OG000', 'lowerLimit': '0.7', 'upperLimit': '2.4'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.50', 'groupId': 'OG000', 'lowerLimit': '0.8', 'upperLimit': '2.6'}]}]}, {'title': 'Week 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.57', 'groupId': 'OG000', 'lowerLimit': '0.5', 'upperLimit': '2.3'}]}]}, {'title': 'Week 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.21', 'groupId': 'OG000', 'lowerLimit': '0.6', 'upperLimit': '3.0'}]}]}, {'title': 'Week 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.21', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '2.5'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.07', 'groupId': 'OG000', 'lowerLimit': '0.2', 'upperLimit': '2.3'}]}]}, {'title': 'Week 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '2.0'}]}]}, {'title': 'Week 10', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.5', 'upperLimit': '1.7'}]}]}, {'title': 'Week 11', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.2', 'upperLimit': '2.0'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.79', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '1.9'}]}]}, {'title': 'Week 13', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.93', 'groupId': 'OG000', 'lowerLimit': '0.1', 'upperLimit': '2.3'}]}]}, {'title': 'Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.93', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '2.1'}]}]}, {'title': 'Week 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.07', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '2.3'}]}]}, {'title': 'Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.10', 'groupId': 'OG000', 'lowerLimit': '0.4', 'upperLimit': '2.0'}]}]}, {'title': 'Week 17', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.13', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '2.0'}]}]}, {'title': 'Week 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '1.9'}]}]}, {'title': 'Week 19', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '1.9'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.83', 'groupId': 'OG000', 'lowerLimit': '0.2', 'upperLimit': '2.1'}]}]}, {'title': 'Week 21', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.5', 'upperLimit': '1.7'}]}]}, {'title': 'Week 22', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.83', 'groupId': 'OG000', 'lowerLimit': '0.4', 'upperLimit': '1.7'}]}]}, {'title': 'Week 23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.86', 'groupId': 'OG000', 'lowerLimit': '0.5', 'upperLimit': '1.8'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.90', 'groupId': 'OG000', 'lowerLimit': '0.3', 'upperLimit': '2.0'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24', 'description': 'Daily bowel movements captured by e-diary. The number of bowel movements per day was calculated each week of the study. It was based on a 1-week average and calculated as the sum of the number of bowel movements in a given week divided by the number of days with non-missing bowel movement frequency data. If more than 3 days of bowel movement data was missing in a given week, bowel movement frequency data was considered missing for that week.', 'unitOfMeasure': 'Bowel movements per day', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '7.0'}]}]}, {'title': 'Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.50', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.0'}]}]}, {'title': 'Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '2.0'}]}]}, {'title': 'Week 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '3.0'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '4.0'}]}]}, {'title': 'Week 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.0'}]}]}, {'title': 'Week 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.20', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.0'}]}]}, {'title': 'Week 7', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.50', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '2.8'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.50', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '3.0'}]}]}, {'title': 'Week 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.50', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '3.0'}]}]}, {'title': 'Week 10', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '4.0'}]}]}, {'title': 'Week 11', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '2.3'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '2.3'}]}]}, {'title': 'Week 13', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '4.0'}]}]}, {'title': 'Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.58', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '4.7'}]}]}, {'title': 'Week 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '7.0'}]}]}, {'title': 'Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.8'}]}]}, {'title': 'Week 17', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.28', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.6'}]}]}, {'title': 'Week 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.50', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '7.0'}]}]}, {'title': 'Week 19', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.0'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.0'}]}]}, {'title': 'Week 21', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '6.0'}]}]}, {'title': 'Week 22', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '6.0'}]}]}, {'title': 'Week 23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '6.0'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.00', 'groupId': 'OG000', 'lowerLimit': '0.0', 'upperLimit': '5.6'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24', 'description': 'The number of days per week with \\>=1 loose/watery bowel movement, is calculated each week of the study as the sum of the number of days with \\>=1 loose/watery bowel movement in a given week divided by the number of days with non-missing stool consistency data and then multiplied by 7, is presented. If more than 3 days of stool consistency data was missing in a given week, stool consistency data was considered missing for that week.', 'unitOfMeasure': 'Days per week', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS included all enrolled participants who received any study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Abdominal Ascites at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 24', 'description': "The measurement of abdominal ascites (excess abdominal fluid) was based on abdominal circumference. Abdominal circumference was measured regardless of the physician's assessment of the presence or absence of ascites.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS included all enrolled participants who received any study drug.'}, {'type': 'SECONDARY', 'title': 'Absolute Value of Albumin at Specified Timepoints up to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.160', 'spread': '0.5296', 'groupId': 'OG000'}]}]}, {'title': 'Day 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.267', 'spread': '0.4000', 'groupId': 'OG000'}]}]}, {'title': 'Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.311', 'spread': '0.4485', 'groupId': 'OG000'}]}]}, {'title': 'Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.589', 'spread': '0.4428', 'groupId': 'OG000'}]}]}, {'title': 'Week 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.025', 'spread': '0.3955', 'groupId': 'OG000'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.167', 'spread': '0.3841', 'groupId': 'OG000'}]}]}, {'title': 'Week 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.411', 'spread': '0.7026', 'groupId': 'OG000'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.188', 'spread': '0.3137', 'groupId': 'OG000'}]}]}, {'title': 'Week 10', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.322', 'spread': '0.2279', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.288', 'spread': '0.2748', 'groupId': 'OG000'}]}]}, {'title': 'Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.363', 'spread': '0.2615', 'groupId': 'OG000'}]}]}, {'title': 'Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.367', 'spread': '0.3674', 'groupId': 'OG000'}]}]}, {'title': 'Week 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.344', 'spread': '0.2555', 'groupId': 'OG000'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.449', 'spread': '0.2539', 'groupId': 'OG000'}]}]}, {'title': 'Week 22', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.422', 'spread': '0.2906', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.544', 'spread': '0.2603', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Day 2, Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22 and 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of albumin. Absolute value of albumin at specified timepoints was reported.', 'unitOfMeasure': 'g/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Protein at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '37.50', 'spread': '6.996', 'groupId': 'OG000'}]}]}, {'title': 'Protein at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '72.22', 'spread': '4.969', 'groupId': 'OG000'}]}]}, {'title': 'IgG at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.183', 'spread': '0.9103', 'groupId': 'OG000'}]}]}, {'title': 'IgG at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.301', 'spread': '2.0503', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of protein and IgG. Absolutes values of protein and IgG measured as g/L at baseline and Week 24 was reported.', 'unitOfMeasure': 'g/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Ig at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '318.2', 'spread': '113.36', 'groupId': 'OG000'}]}]}, {'title': 'Ig at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1430.1', 'spread': '263.36', 'groupId': 'OG000'}]}]}, {'title': 'IgM at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '52.9', 'spread': '28.36', 'groupId': 'OG000'}]}]}, {'title': 'IgM at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '180.3', 'spread': '74.81', 'groupId': 'OG000'}]}]}, {'title': 'IgA at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '47.0', 'spread': '18.51', 'groupId': 'OG000'}]}]}, {'title': 'IgA at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '119.8', 'spread': '52.46', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of Ig, IgM and IgA. Absolute value of Ig, IgM and IgA measured as mg/dL at baseline and Week 24 was reported.', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Vitamin B12 at Baseline and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '158.5', 'spread': '52.32', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'categories': [{'measurements': [{'value': '383.1', 'spread': '102.73', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of vitamin B12. Absolute values of vitamin B12 at baseline and Week 24 was reported.', 'unitOfMeasure': 'picomoles per liter (pmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Iron at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.40', 'spread': '1.478', 'groupId': 'OG000'}]}]}, {'title': 'Iron at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.38', 'spread': '5.036', 'groupId': 'OG000'}]}]}, {'title': 'Unsaturated Binding Capacity at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '38.24', 'spread': '12.357', 'groupId': 'OG000'}]}]}, {'title': 'Unsaturated Iron Binding Capacity at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '73.95', 'spread': '12.049', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of unsaturated iron and unsaturated iron binding capacity measured as micromoles per liter (mcmol/L) at baseline and Week 24 was reported.', 'unitOfMeasure': 'mcmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Vitamin B9 up to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17.80', 'spread': '15.017', 'groupId': 'OG000'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '22.07', 'spread': '14.342', 'groupId': 'OG000'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17.32', 'spread': '8.042', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17.89', 'spread': '9.646', 'groupId': 'OG000'}]}]}, {'title': 'Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.01', 'spread': '10.610', 'groupId': 'OG000'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19.91', 'spread': '15.436', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.93', 'spread': '6.538', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Week 24', 'description': 'Absolute Values of Vitamin B9 - Central Lab', 'unitOfMeasure': 'nanomoles per liter (nmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Number of participants based on the total in the safety analysis set (SAF)'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Ferritin at Baseline and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Baseline', 'categories': [{'measurements': [{'value': '10.0', 'spread': '5.90', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'categories': [{'measurements': [{'value': '12.0', 'spread': '7.86', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of ferritin was reported.', 'unitOfMeasure': 'microgram per liter (mcg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Magnesium at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.793', 'spread': '0.0381', 'groupId': 'OG000'}]}]}, {'title': 'Magnesium at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.858', 'spread': '0.0245', 'groupId': 'OG000'}]}]}, {'title': 'Total Cholesterol at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.819', 'spread': '0.8786', 'groupId': 'OG000'}]}]}, {'title': 'Total Cholesterol at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.604', 'spread': '0.3118', 'groupId': 'OG000'}]}]}, {'title': 'Triglycerides at Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.033', 'spread': '0.7946', 'groupId': 'OG000'}]}]}, {'title': 'Triglycerides at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.094', 'spread': '0.3745', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of magnesium, total cholesterol, and triglycerides. Absolute values of magnesium, total cholesterol, and triglycerides measured as mmol/L at baseline and Week 24 was reported.', 'unitOfMeasure': 'mmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Alpha-1 Antitrypsin Levels in Stool at Week 12 and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Change from Baseline to Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-527.80', 'spread': '318.818', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-463.71', 'spread': '204.250', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 12, Week 24', 'unitOfMeasure': 'milligrams per deciliter (mg/dL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The FAS included all enrolled participants who received the study drug.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Alpha-1 Antitrypsin Levels in Blood at Week 12 and Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Change from Baseline to Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-57.8', 'spread': '33.15', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-43.4', 'spread': '35.89', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 12, Week 24', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Number of participants based on the total in the safety analysis set (SAF)'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Weeks 12 to 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 12 to 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 24 to 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 48 to 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 96 to 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Weeks 12 to 48; Weeks 12 to 144; Weeks 24 to 48; Weeks 48 to 96; Weeks 96 to 144', 'description': 'Measured by normalization of serum albumin, no worsening of facial or peripheral edema, increase in bowel movement, or increase in abdominal pain frequency, no increase in dose of permitted concomitant medication for the treatment of PLE at any time as described in the protocol', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5, and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Physician Assessment of Facial Edema Based on a 5-point Likert Rating Scale', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.7', 'spread': '0.87', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': 'The physician assessment of facial edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Physician Assessment of Peripheral Edema Based on a 5-point Likert Rating Scale', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.7', 'spread': '0.71', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': 'The physician assessment of peripheral edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': "Change From Baseline in Food and Drink Limitations as Assessed by the PedsQL™ GI Symptom Scales' Food and Drink Limits Sub-scale", 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '47.40', 'spread': '36.653', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': 'Frequency of limitations is assessed on a 5-point Likert response scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse scored and linearly transformed to a 0 to 100 scale, where lower scores indicate more frequent problems with food and drink limitations.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Week 144 Change from Baseline Total score of PedsQL Core Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.55', 'spread': '17.023', 'groupId': 'OG000'}]}]}, {'title': 'Week 144 Change from Baseline Subscale score: Physical functioning', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14.45', 'spread': '39.900', 'groupId': 'OG000'}]}]}, {'title': 'Week 144 Change from Baseline Subscale score: Emotional functioning', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '26.3', 'spread': '28.88', 'groupId': 'OG000'}]}]}, {'title': 'Week 144 Change from Baseline Subscale score: Social functioning', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '30.6', 'spread': '20.43', 'groupId': 'OG000'}]}]}, {'title': 'Week 144 Change from Baseline Subscale score: School functioning', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '41.43', 'spread': '19.518', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': 'Physical functioning, emotional functioning, social functioning, and school/work/studies functioning is assessed using a 5-point Likert scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse-scored and linearly transformed to a 0 to 100 scale, with higher scores indicating better HRQoL. The total scale score is computed as the sum of all the items over the number of items answered on individual scales. Subscale score is calculated as the sum of the items in the scale divided by the number of items answered in the scale.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Albumin Infusion by 24 Week Periods', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Weeks 0 to 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 24 to 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 48 to 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 72 to 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 96 to 120', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Weeks 120 to 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5, and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Albumin Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.400', 'spread': '0.6103', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'grams per deciliter (g/dL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Percentage Change From Baseline in Albumin Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '127.489', 'spread': '81.0666', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'percentage of change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Albumin Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '15.5', 'groupId': 'OG000', 'lowerLimit': '8', 'upperLimit': '28'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Protein Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '37.13', 'spread': '10.789', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'grams per liter (g/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Total Protein', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '22.0', 'groupId': 'OG000', 'lowerLimit': '9', 'upperLimit': '35'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Immunoglobulin (Ig) Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1271.2', 'spread': '261.91', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Ig Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '341', 'groupId': 'OG000', 'lowerLimit': '341', 'upperLimit': '341'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants with available Ig lab reference ranges.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in IgG Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '10.626', 'spread': '2.2486', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'g/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for IgG Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '29.0', 'groupId': 'OG000', 'lowerLimit': '8', 'upperLimit': '29'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in IgM Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '112.0', 'spread': '51.21', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for IgM Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '9'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 44', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug who were evaluable for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in IgA Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '96.7', 'spread': '28.78', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for IgA Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '9'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Vitamin B12 Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '138.8', 'spread': '56.07', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'picomoles per liter (pmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Vitamin B12 Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '8'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Vitamin B9 (Folate) Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.92', 'spread': '10.241', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'nanomole per liter (nmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Vitamin B9 (Folate) Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '8'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Iron Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '7.82', 'spread': '4.091', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'micromole/liter (umol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Iron Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '97.5', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '337'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Unsaturated Iron Binding Capacity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '16.20', 'spread': '13.811', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'umol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Unsaturated Iron Binding Capacity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '56'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Ferritin Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '32.0', 'spread': '23.67', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'microgram per liter (ug/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Ferritin Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '505'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Magnesium Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.083', 'spread': '0.0606', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'millimoles per liter (mmol/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Magnesium Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '2'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Fasting Cholesterol Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.072', 'spread': '0.7299', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'mmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Fasting Cholesterol Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '29'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Fasting Triglycerides Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.710', 'spread': '0.9086', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'mmol/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Time to First Normalization for Fasting Triglycerides Values', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '57'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Used Concomitant Medication', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 144', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Hospitalization Days by 24 Week Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Weeks 0 to 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '4'}]}]}, {'title': 'Weeks 24 to 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Weeks 48 to 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Weeks 72 to 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}]}]}, {'title': 'Weeks 96 to 120', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '2'}]}]}, {'title': 'Weeks 120 to 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144', 'unitOfMeasure': 'days', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5 and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Body Weight Z-Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.924', 'spread': '0.7529', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': "Weight-for-age z-score compares a participant's weight to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Body weight z-score, regardless of magnitude, represents catch-up growth.", 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Height Z-Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.079', 'spread': '0.6469', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': "Height-for-age z-score compares a participant's height to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Any increase in height z-score, regardless of magnitude, represents catch-up growth.", 'unitOfMeasure': 'z-score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Total Complement Activity Complement Hemolytic Assay (CH50)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '-241.6', 'spread': '39.79', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'unitOfMeasure': 'international units/milliliter (IU/mL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Concentrations of Total Pozelimab in Serum', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'title': 'Week 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '182', 'spread': '68.9', 'groupId': 'OG000'}]}]}, {'title': 'Week 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '185', 'spread': '103', 'groupId': 'OG000'}]}]}, {'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '227', 'spread': '110', 'groupId': 'OG000'}]}]}, {'title': 'Week 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '268', 'spread': '106', 'groupId': 'OG000'}]}]}, {'title': 'Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '306', 'spread': '111', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '341', 'spread': '115', 'groupId': 'OG000'}]}]}, {'title': 'Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '402', 'spread': '141', 'groupId': 'OG000'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '418', 'spread': '136', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '421', 'spread': '129', 'groupId': 'OG000'}]}]}, {'title': 'Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '467', 'spread': '127', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '517', 'spread': '142', 'groupId': 'OG000'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '492', 'spread': '131', 'groupId': 'OG000'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '461', 'spread': '115', 'groupId': 'OG000'}]}]}, {'title': 'Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '430', 'spread': '134', 'groupId': 'OG000'}]}]}, {'title': 'Week 145', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': 'NA', 'spread': 'NA', 'comment': 'Mean and Standard Deviation (SD) not calculable with n=1', 'groupId': 'OG000'}]}]}, {'title': 'Week 146', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': 'NA', 'spread': 'NA', 'comment': 'Mean and Standard Deviation (SD) not calculable with n=1', 'groupId': 'OG000'}]}]}, {'title': 'Week 164', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '410', 'spread': '104', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Week 164', 'unitOfMeasure': 'mg/L', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Pharmacokinetic Analysis Set (PKAS) includes all treated participants who received any amount of study drug and had at least 1 non-missing total pozelimab measurement following the first dose of study drug. The PKAS is based on the actual treatment received (as treated) rather than as randomized.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment-emergent Anti-drug Antibodies (ADA) to Pozelimab', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 164', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The Anti-Drug Antibody Analysis Set (AAS) includes all treated participants who received any amount of study drug and had at least 1 non-missing ADA result following the first dose of study drug. The AAS is based on the actual treatment received (as treated) rather than as randomized.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 milligrams per kilogram (mg/kg) intravenous (IV) injection on day 1, followed by weekly subcutaneous (SC) injection as maintenance dosing of 125 mg for body weight less than (\\<) 10 kilograms (kg), 200 mg for greater than or equal to (\\>=) 10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'comment': 'Completed study', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'A total of 11 participants were screened for this study. Of these, 1 participant did not meet inclusion/exclusion criteria, and 10 were assigned to study treatment.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Pozelimab Injection', 'description': 'Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \\<10 kg, 200 mg for \\>=10 kg and \\<20 kg, 350 mg for \\>=20 kg and \\<40 kg, 500 mg for \\>=40 kg and \\<60 kg, and 800 mg for \\>=60 kg over the treatment period.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '9.3', 'spread': '4.88', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The full analysis set (FAS) included all enrolled participants who received the study drug.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-02-01', 'size': 948089, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-09-15T09:32', 'hasProtocol': True}, {'date': '2022-06-23', 'size': 622372, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-09-15T09:33', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-01-27', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'dispFirstSubmitDate': '2022-11-08', 'completionDateStruct': {'date': '2024-05-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-27', 'studyFirstSubmitDate': '2019-12-20', 'resultsFirstSubmitDate': '2023-09-15', 'studyFirstSubmitQcDate': '2019-12-20', 'dispFirstPostDateStruct': {'date': '2023-10-30', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-08-29', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2023-10-25', 'studyFirstPostDateStruct': {'date': '2019-12-24', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-10-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-11-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24', 'timeFrame': 'At Week 24', 'description': 'Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of \\<2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs', 'timeFrame': 'From start of study drug administration up to approximately 144 weeks', 'description': "TEAEs are defined as AEs that developed or worsened during the on-treatment period. The on-treatment period is defined as the time from first dose of investigational product up to 21 weeks after the last dose of investigational product. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the patient's normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health."}, {'measure': 'Number of Participants With Improvement in Most Bothersome Signs and Symptoms at Week 24', 'timeFrame': 'At Week 24', 'description': "Improvement in most bothersome sign/symptom determined using semi-structured concept elicitation interview, from 'core' clinical endpoints of frequency of bowel movements, peripheral edema, facial edema, abdominal pain frequency, nausea, vomiting, stool consistency."}, {'measure': 'Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24', 'timeFrame': 'Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24', 'description': 'Daily bowel movements captured by e-diary. The number of bowel movements per day was calculated each week of the study. It was based on a 1-week average and calculated as the sum of the number of bowel movements in a given week divided by the number of days with non-missing bowel movement frequency data. If more than 3 days of bowel movement data was missing in a given week, bowel movement frequency data was considered missing for that week.'}, {'measure': 'Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24', 'timeFrame': 'Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24', 'description': 'The number of days per week with \\>=1 loose/watery bowel movement, is calculated each week of the study as the sum of the number of days with \\>=1 loose/watery bowel movement in a given week divided by the number of days with non-missing stool consistency data and then multiplied by 7, is presented. If more than 3 days of stool consistency data was missing in a given week, stool consistency data was considered missing for that week.'}, {'measure': 'Number of Participants With Abdominal Ascites at Week 24', 'timeFrame': 'Baseline up to Week 24', 'description': "The measurement of abdominal ascites (excess abdominal fluid) was based on abdominal circumference. Abdominal circumference was measured regardless of the physician's assessment of the presence or absence of ascites."}, {'measure': 'Absolute Value of Albumin at Specified Timepoints up to Week 24', 'timeFrame': 'Baseline, Day 2, Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22 and 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of albumin. Absolute value of albumin at specified timepoints was reported.'}, {'measure': 'Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of protein and IgG. Absolutes values of protein and IgG measured as g/L at baseline and Week 24 was reported.'}, {'measure': 'Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of Ig, IgM and IgA. Absolute value of Ig, IgM and IgA measured as mg/dL at baseline and Week 24 was reported.'}, {'measure': 'Absolute Values of Vitamin B12 at Baseline and Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of vitamin B12. Absolute values of vitamin B12 at baseline and Week 24 was reported.'}, {'measure': 'Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of unsaturated iron and unsaturated iron binding capacity measured as micromoles per liter (mcmol/L) at baseline and Week 24 was reported.'}, {'measure': 'Absolute Values of Vitamin B9 up to Week 24', 'timeFrame': 'Baseline up to Week 24', 'description': 'Absolute Values of Vitamin B9 - Central Lab'}, {'measure': 'Absolute Values of Ferritin at Baseline and Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of ferritin was reported.'}, {'measure': 'Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Blood samples were collected from participants at defined time points for the assessment of magnesium, total cholesterol, and triglycerides. Absolute values of magnesium, total cholesterol, and triglycerides measured as mmol/L at baseline and Week 24 was reported.'}, {'measure': 'Change From Baseline in Alpha-1 Antitrypsin Levels in Stool at Week 12 and Week 24', 'timeFrame': 'Week 12, Week 24'}, {'measure': 'Change From Baseline in Alpha-1 Antitrypsin Levels in Blood at Week 12 and Week 24', 'timeFrame': 'Week 12, Week 24'}, {'measure': 'Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control', 'timeFrame': 'Weeks 12 to 48; Weeks 12 to 144; Weeks 24 to 48; Weeks 48 to 96; Weeks 96 to 144', 'description': 'Measured by normalization of serum albumin, no worsening of facial or peripheral edema, increase in bowel movement, or increase in abdominal pain frequency, no increase in dose of permitted concomitant medication for the treatment of PLE at any time as described in the protocol'}, {'measure': 'Change From Baseline in Physician Assessment of Facial Edema Based on a 5-point Likert Rating Scale', 'timeFrame': 'Baseline and Week 144', 'description': 'The physician assessment of facial edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5).'}, {'measure': 'Change From Baseline in Physician Assessment of Peripheral Edema Based on a 5-point Likert Rating Scale', 'timeFrame': 'Baseline and Week 144', 'description': 'The physician assessment of peripheral edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5).'}, {'measure': "Change From Baseline in Food and Drink Limitations as Assessed by the PedsQL™ GI Symptom Scales' Food and Drink Limits Sub-scale", 'timeFrame': 'Baseline and Week 144', 'description': 'Frequency of limitations is assessed on a 5-point Likert response scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse scored and linearly transformed to a 0 to 100 scale, where lower scores indicate more frequent problems with food and drink limitations.'}, {'measure': 'Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales', 'timeFrame': 'Baseline and Week 144', 'description': 'Physical functioning, emotional functioning, social functioning, and school/work/studies functioning is assessed using a 5-point Likert scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse-scored and linearly transformed to a 0 to 100 scale, with higher scores indicating better HRQoL. The total scale score is computed as the sum of all the items over the number of items answered on individual scales. Subscale score is calculated as the sum of the items in the scale divided by the number of items answered in the scale.'}, {'measure': 'Number of Participants With Albumin Infusion by 24 Week Periods', 'timeFrame': 'Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144'}, {'measure': 'Change From Baseline in Albumin Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Percentage Change From Baseline in Albumin Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Albumin Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Protein Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Total Protein', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Immunoglobulin (Ig) Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Ig Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in IgG Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for IgG Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in IgM Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for IgM Values', 'timeFrame': 'Baseline up to Week 44'}, {'measure': 'Change From Baseline in IgA Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for IgA Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Vitamin B12 Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Vitamin B12 Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Vitamin B9 (Folate) Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Vitamin B9 (Folate) Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Iron Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Iron Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Unsaturated Iron Binding Capacity', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Unsaturated Iron Binding Capacity', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Ferritin Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Ferritin Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Magnesium Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Magnesium Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Fasting Cholesterol Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Fasting Cholesterol Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Change From Baseline in Fasting Triglycerides Values', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Time to First Normalization for Fasting Triglycerides Values', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Number of Participants Who Used Concomitant Medication', 'timeFrame': 'Baseline up to Week 144'}, {'measure': 'Number of Hospitalization Days by 24 Week Period', 'timeFrame': 'Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144'}, {'measure': 'Change From Baseline in Body Weight Z-Score', 'timeFrame': 'Baseline and Week 144', 'description': "Weight-for-age z-score compares a participant's weight to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Body weight z-score, regardless of magnitude, represents catch-up growth."}, {'measure': 'Change From Baseline in Height Z-Score', 'timeFrame': 'Baseline and Week 144', 'description': "Height-for-age z-score compares a participant's height to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Any increase in height z-score, regardless of magnitude, represents catch-up growth."}, {'measure': 'Change From Baseline in Total Complement Activity Complement Hemolytic Assay (CH50)', 'timeFrame': 'Baseline and Week 144'}, {'measure': 'Concentrations of Total Pozelimab in Serum', 'timeFrame': 'Baseline up to Week 164'}, {'measure': 'Number of Participants With Treatment-emergent Anti-drug Antibodies (ADA) to Pozelimab', 'timeFrame': 'Baseline up to Week 164'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['CD55-deficient PLE', 'complement hyperactivation, angiopathic thrombosis, protein-losing enteropathy (CHAPLE disease)'], 'conditions': ['CD55-deficient Protein-losing Enteropathy', 'CHAPLE']}, 'referencesModule': {'references': [{'pmid': '39934837', 'type': 'DERIVED', 'citation': 'Litcher-Kelly L, Ozen A, Ollis S, Feldman HB, Yaworsky A, Medrano P, Chongsrisawat V, Perlee L, Walker M, Pradeep S, Turner-Bowker DM, Kurolap A, Adiv OE, Lenardo MJ, Harari OA, Jalbert JJ. The patient experience of CHAPLE disease: results from interviews conducted as part of a clinical trial for an ultra-rare condition. Orphanet J Rare Dis. 2025 Feb 11;20(1):68. doi: 10.1186/s13023-024-03436-y.'}, {'pmid': '39118150', 'type': 'DERIVED', 'citation': 'Litcher-Kelly L, Ozen A, Ollis S, Feldman HB, Yaworsky A, Medrano P, Chongsrisawa V, Brackin T, Perlee L, Walker M, Pradeep S, Lenardo MJ, Harari OA, Jalbert JJ. Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments. Orphanet J Rare Dis. 2024 Aug 8;19(1):290. doi: 10.1186/s13023-024-03277-9.'}, {'pmid': '38278170', 'type': 'DERIVED', 'citation': 'Ozen A, Chongsrisawat V, Sefer AP, Kolukisa B, Jalbert JJ, Meagher KA, Brackin T, Feldman HB, Baris S, Karakoc-Aydiner E, Ergelen R, Fuss IJ, Moorman H, Suratannon N, Suphapeetiporn K, Perlee L, Harari OA, Yancopoulos GD, Lenardo MJ; Pozelimab CHAPLE Working Group. Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 study. Lancet. 2024 Feb 17;403(10427):645-656. doi: 10.1016/S0140-6736(23)02358-9. Epub 2024 Jan 23.'}], 'seeAlsoLinks': [{'url': 'https://www.trialsummaries.com/', 'label': 'A Plain Language Summary is available on TrialSummaries.com'}]}, 'descriptionModule': {'briefSummary': 'The primary objective of the study is to determine the effect of pozelimab on active CD55-deficient protein-losing enteropathy (PLE; CHAPLE).\n\nThe secondary objectives of the study are:\n\n* To evaluate the safety and tolerability of pozelimab in patients with CD55-deficient PLE disease\n* To evaluate the effect of pozelimab on CD55-deficient PLE (both patients with active disease at baseline and those with inactive disease on eculizumab, switching to pozelimab)\n* To determine the effects of pozelimab on albumin and other serum proteins (total protein, immunoglobulins)\n* To determine the effects of pozelimab on ascites\n* To determine the effects of pozelimab on stool consistency\n* To determine the effect of pozelimab on health-related quality of life\n* To determine the effect of pozelimab on lab abnormalities observed in CD55-deficient PLE such as hypertriglyceridemia, thrombocytosis, and hypovitaminosis B12\n* To describe the effects of pozelimab on the sparing of concomitant medications and reduction in hospitalization days\n* To determine the effects of pozelimab on growth\n* To characterize the concentration of pozelimab in patients with CD55-deficient PLE\n* To assess the incidence of treatment-emergent ADA for pozelimab in patients with CD55-deficient PLE disease'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Clinical diagnosis of CD55-deficient PLE/CHAPLE disease (based on a history of PLE), confirmed by biallelic CD55 loss-of-function mutation detected by genotype analysis\n* Active disease as defined by the protocol or inactive disease on eculizumab therapy (and whose treating physician has the expectation of future access to renewed eculizumab treatment should this be required), and is willing to discontinue eculizumab during screening and start pozelimab at baseline with no eculizumab wash-out\n\nKey Exclusion Criteria:\n\n* History of meningococcal infection\n* No documented meningococcal vaccination within 3 years prior to screening and patient unwilling to undergo vaccination during the study\n* No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening and patient unwilling to undergo vaccination during the study if required per local practice or guidelines\n* Presence of a concomitant disease that leads to hypoproteinemia at the time of starting pozelimab\n* A concomitant disease that leads to secondary intestinal lymphangiectasia such as a fontan procedure for congenital heart disease\n\nNote: Other protocol-defined Inclusion/Exclusion criteria apply.'}, 'identificationModule': {'nctId': 'NCT04209634', 'briefTitle': 'Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Regeneron Pharmaceuticals'}, 'officialTitle': 'An Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)', 'orgStudyIdInfo': {'id': 'R3918-PLE-1878'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Active PLE', 'description': 'Patients aged 1 year and older with a clinical diagnosis of CD55-deficient PLE disease', 'interventionNames': ['Drug: Pozelimab']}], 'interventions': [{'name': 'Pozelimab', 'type': 'DRUG', 'otherNames': ['REGN3918'], 'description': 'Single loading intravenous (IV) dose on day 1, then fixed doses sub-cutaneous (SC) (based on body weight) QW (±2 days) over the treatment period.', 'armGroupLabels': ['Active PLE']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20892', 'city': 'Bethesda', 'state': 'Maryland', 'country': 'United States', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 38.98067, 'lon': -77.10026}}, {'zip': '10330', 'city': 'Pathum Wan', 'state': 'Bangkok', 'country': 'Thailand', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 13.73649, 'lon': 100.5239}}, {'zip': '34890', 'city': 'Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Regeneron Research Site', 'geoPoint': {'lat': 41.01384, 'lon': 28.94966}}], 'overallOfficials': [{'name': 'Clinical Trial Management', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Regeneron Pharmaceuticals'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'ICF', 'CSR', 'ANALYTIC_CODE'], 'timeFrame': 'Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.', 'ipdSharing': 'YES', 'description': 'All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing', 'accessCriteria': 'Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, EMA, PMDA, etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Regeneron Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}