Ignite Creation Date:
2025-12-24 @ 11:28 PM
Ignite Modification Date:
2026-02-25 @ 9:44 PM
Study NCT ID:
NCT04200456
Status:
TERMINATED
Last Update Posted:
2025-03-18
First Post:
2019-12-13
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Austria', 'Belgium', 'Croatia', 'Czechia', 'Spain'], 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-06-12', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D016553', 'term': 'Purpura, Thrombocytopenic, Idiopathic'}], 'ancestors': [{'id': 'D011696', 'term': 'Purpura, Thrombocytopenic'}, {'id': 'D011693', 'term': 'Purpura'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D057049', 'term': 'Thrombotic Microangiopathies'}, {'id': 'D013921', 'term': 'Thrombocytopenia'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D006470', 'term': 'Hemorrhage'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D012877', 'term': 'Skin Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000627812', 'term': 'rozanolixizumab'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'UCBCares@ucb.com', 'phone': '001 844 599 2273', 'title': 'UCB', 'organization': 'Cares'}, 'certainAgreement': {'restrictionType': 'GT60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From Baseline to end of Safety Follow-Up Period (up to Week 31)', 'description': 'TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose. TEAEs were analyzed for Safety Set.', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.', 'otherNumAtRisk': 12, 'deathsNumAtRisk': 12, 'otherNumAffected': 9, 'seriousNumAtRisk': 12, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.', 'otherNumAtRisk': 21, 'deathsNumAtRisk': 21, 'otherNumAffected': 16, 'seriousNumAtRisk': 21, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 12, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 9, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 34, 'numAffected': 9}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Enterocolitis infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Body temperature increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Hyperglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Type 2 diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 8, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 48, 'numAffected': 14}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Rash macular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}], 'seriousEvents': [{'term': 'Immune thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Urethritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}, {'term': 'Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v24.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '19.0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'During the last 12 weeks (Week 13 to Week 25)', 'description': 'Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10\\^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized. No formal analysis was carried out as the program was terminated.'}, {'type': 'SECONDARY', 'title': 'Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '7'}, {'value': '3.0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '24'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Week 1 up to Week 25', 'description': 'Total number of weeks with platelet counts ≥50×10\\^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25) were reported.', 'unitOfMeasure': 'Weeks', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized.'}, {'type': 'SECONDARY', 'title': 'Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: Time From Starting Treatment to Achievement of First Response of ≥50×10^9/L', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '44.0', 'comment': 'NA signifies that upper confidence limit for placebo is not provided for the 95% CI of the median time to first CMPR as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.', 'groupId': 'OG000', 'lowerLimit': '6.0', 'upperLimit': 'NA'}, {'value': '8.0', 'comment': 'NA signifies that upper confidence limit for rozanolixizumab is not provided for the 95% CI of the median time to first CMPR as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.', 'groupId': 'OG001', 'lowerLimit': '6.0', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)', 'description': 'Time from starting treatment to achievement of first Clinically Meaningful Platelet Response of ≥50×10\\^9/L was defined as date of first clinically meaningful response - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate.', 'unitOfMeasure': 'days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '16.7', 'groupId': 'OG000'}, {'value': '52.4', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to Day 8', 'description': 'Clinically meaningful platelet response was defined as platelet count of ≥50×10\\^9/L.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '8.3', 'groupId': 'OG000'}, {'value': '33.3', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline during Treatment Period (up to Week 25)', 'description': 'Response was defined as platelet count ≥30×10\\^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized.'}, {'type': 'SECONDARY', 'title': 'Time to First Rescue Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '34.5', 'comment': 'NA signifies that upper confidence limit for placebo is not provided for the 95% CI of the median time to rescue therapy as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.', 'groupId': 'OG000', 'lowerLimit': '4.0', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'NA for median signifies that the probability of participants requiring rescue medication did not reach 0.5 so the Kaplan-Meier median could not be estimated.\n\nNA signifies that upper confidence limit for rozanolixizumab is not provided for the 95% CI of the median time to rescue therapy as there is no time at which the upper bound of the CI for the Kaplan-Meier estimator is less than or equal to 0.5.', 'groupId': 'OG001', 'lowerLimit': '23.0', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'From Baseline to first rescue therapy (up to Week 25)', 'description': 'Time to first rescue therapy was defined as date of first rescue therapy use - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate. The probability of requiring rescue medication did not reach 0.5 so the KM median in the rozanolixizumab arm could not be estimated.', 'unitOfMeasure': 'Days', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '6.9', 'spread': '13.8', 'groupId': 'OG000'}, {'value': '5.5', 'spread': '9.2', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline during Treatment Period (up to Week 25)', 'description': "The ITP-PAQ Version 1 is a 44 item disease-specific Health-Related Quality of Life questionnaire developed for use in adults with chronic ITP. It includes 10 scales, Four of the scales measure physical health: Symptoms (6 items), Bother (3 items), Fatigue (4 items), and Activity (2 items). Two of the scales measure emotional health: Fear (5 items) and Psychological (5 items) Health. The remaining four scales measure other aspects of quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women's Reproductive QOL (6 items) and Overall QOL (5 items). Each item is rated on a Likert-type scale containing 4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted equally to derive individual scale scores and the total score (0-100) is calculated as per the formula: Sum of item scores within the scale/raw sum range\\*100. Higher scores indicate better health status.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Randomized Set consisted of all enrolled study participants who were randomized. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '75.0', 'groupId': 'OG000'}, {'value': '85.7', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline to end of Safety Follow-Up Period (up to Week 31)', 'description': 'An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety set included all randomized study participants who received at least one dose of IMP.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'OG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '4.8', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline to end of Safety Follow-Up Period (up to Week 31)', 'description': 'An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.', 'unitOfMeasure': 'Percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety set included all randomized study participants who received at least one dose of IMP.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo subcutaneous (sc) infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'FG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}, {'groupId': 'FG001', 'numSubjects': '21'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}, {'groupId': 'FG001', 'numSubjects': '15'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Adverse event, non-fatal', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Administration of Rescue and concern about IMP', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'The study started to enroll study participants in Jan 2020 and terminated on April 2022.', 'preAssignmentDetails': 'Participant Flow refers to the Randomized Set.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '33', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Participants received a fixed-unit starting dose of placebo sc infusion matched to rozanolixizumab Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of placebo sc infusion matched to rozanolixizumab Dose B every 2 weeks until Week 23. Participants were followed up to a maximum of Week 31.'}, {'id': 'BG001', 'title': 'Rozanolixizumab', 'description': 'Participants received a fixed-unit starting dose of rozanolixizumab sc infusion equivalent to Dose A on Day 1. Following the initial dose, participants received a fixed-unit dose of rozanolixizumab sc infusion equivalent to Dose B every 2 weeks until Week 23. After protocol amendment 3, the starting dose was removed and the frequency of administration of the Dose B was changed to weekly. Participants were followed up to a maximum of Week 31.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '18', 'groupId': 'BG001'}, {'value': '28', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '51.4', 'spread': '15.9', 'groupId': 'BG000'}, {'value': '41.4', 'spread': '12.8', 'groupId': 'BG001'}, {'value': '45.1', 'spread': '14.6', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '27', 'groupId': 'BG002'}]}, {'title': 'Missing', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '20', 'groupId': 'BG001'}, {'value': '32', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Platelet count', 'classes': [{'categories': [{'measurements': [{'value': '17.2', 'spread': '11.3', 'groupId': 'BG000'}, {'value': '17.0', 'spread': '9.4', 'groupId': 'BG001'}, {'value': '17.1', 'spread': '9.9', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'cells*10^9/L', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Baseline Characteristics refer to Randomized Set which consisted of all enrolled study participants who were randomized.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-12-03', 'size': 4084696, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-05-15T10:07', 'hasProtocol': True}, {'date': '2022-08-16', 'size': 2083147, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-05-15T10:07', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Investigators are blinded to the treatment code, they will see the platelet values.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 33}}, 'statusModule': {'whyStopped': 'Strategic Business Decision; Not a safety decision', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2020-01-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'dispFirstSubmitDate': '2022-12-19', 'completionDateStruct': {'date': '2022-04-27', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-03-06', 'studyFirstSubmitDate': '2019-12-13', 'resultsFirstSubmitDate': '2023-05-15', 'studyFirstSubmitQcDate': '2019-12-13', 'dispFirstPostDateStruct': {'date': '2023-08-08', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-03-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-08-04', 'studyFirstPostDateStruct': {'date': '2019-12-16', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-08-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-21', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50x10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks', 'timeFrame': 'During the last 12 weeks (Week 13 to Week 25)', 'description': 'Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10\\^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported.'}], 'secondaryOutcomes': [{'measure': 'Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period', 'timeFrame': 'Week 1 up to Week 25', 'description': 'Total number of weeks with platelet counts ≥50×10\\^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25) were reported.'}, {'measure': 'Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: Time From Starting Treatment to Achievement of First Response of ≥50×10^9/L', 'timeFrame': 'Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)', 'description': 'Time from starting treatment to achievement of first Clinically Meaningful Platelet Response of ≥50×10\\^9/L was defined as date of first clinically meaningful response - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate.'}, {'measure': 'Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8', 'timeFrame': 'Baseline to Day 8', 'description': 'Clinically meaningful platelet response was defined as platelet count of ≥50×10\\^9/L.'}, {'measure': 'Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding', 'timeFrame': 'From Baseline during Treatment Period (up to Week 25)', 'description': 'Response was defined as platelet count ≥30×10\\^9/L and at least doubling of baseline, at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding.'}, {'measure': 'Time to First Rescue Therapy', 'timeFrame': 'From Baseline to first rescue therapy (up to Week 25)', 'description': 'Time to first rescue therapy was defined as date of first rescue therapy use - date of first treatment + 1. Median was calculated based upon the Kaplan-Meier estimate. The probability of requiring rescue medication did not reach 0.5 so the KM median in the rozanolixizumab arm could not be estimated.'}, {'measure': 'Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score', 'timeFrame': 'From Baseline during Treatment Period (up to Week 25)', 'description': "The ITP-PAQ Version 1 is a 44 item disease-specific Health-Related Quality of Life questionnaire developed for use in adults with chronic ITP. It includes 10 scales, Four of the scales measure physical health: Symptoms (6 items), Bother (3 items), Fatigue (4 items), and Activity (2 items). Two of the scales measure emotional health: Fear (5 items) and Psychological (5 items) Health. The remaining four scales measure other aspects of quality of life (QOL): Work QOL (4 items), Social QOL (4 items), Women's Reproductive QOL (6 items) and Overall QOL (5 items). Each item is rated on a Likert-type scale containing 4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted equally to derive individual scale scores and the total score (0-100) is calculated as per the formula: Sum of item scores within the scale/raw sum range\\*100. Higher scores indicate better health status."}, {'measure': 'Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)', 'timeFrame': 'From Baseline to end of Safety Follow-Up Period (up to Week 31)', 'description': 'An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.'}, {'measure': 'Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)', 'timeFrame': 'From Baseline to end of Safety Follow-Up Period (up to Week 31)', 'description': 'An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of IMP, whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['ITP', 'UCB7665', 'Rozanolixizumab', 'Primary immune thrombocytopenia'], 'conditions': ['Primary Immune Thrombocytopenia']}, 'referencesModule': {'references': [{'pmid': '39552477', 'type': 'RESULT', 'citation': 'Cooper N, Bussel JB, Kazmierczak M, Miyakawa Y, Cluck S, Lledo Garcia R, Haier B, Lavrov A, Singh P, Snipes R, Kuter DJ. Inhibition of FcRn with rozanolixizumab in adults with immune thrombocytopenia: Two randomised, double-blind, placebo-controlled phase 3 studies and their open-label extension. Br J Haematol. 2025 Feb;206(2):675-688. doi: 10.1111/bjh.19858. Epub 2024 Nov 18.'}], 'seeAlsoLinks': [{'url': 'http://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm', 'label': 'FDA Safety Alerts and Recalls'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment and assess safety and tolerability of rozanolixizumab in adult study participants with primary immune thrombocytopenia (ITP).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Study participant must be ≥18 years of age at the time of the Screening Visit\n* Study participant has a diagnosis of persistent (\\>3 months duration) or chronic (\\>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit\n* Study participant has a documented intolerance or insufficient response to two or more appropriate standard of care ITP treatments prior to Screening\n* Study participants must have prior history of a response to a previous ITP therapy\n* If taking allowed drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1)\n* Study participant has a documented history of low platelet count (\\<30×10\\^9/L) prior to Screening\n* Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two \\<30×10\\^9/L and no single count may be \\>35×10\\^9/L (using local laboratories)\n* Study participant has a current or history of a peripheral blood smear consistent with ITP\n* Study participants may be male or female:\n\n 1. A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period\n 2. A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test and not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies:\n\nNot a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment\n\nExclusion Criteria:\n\n* Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires current anticoagulant treatment\n* Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant drop in hemoglobin)\n* Study participant has a known hypersensitivity to any components of the study medication or any other anti-neonatal Fc receptor (FcRn) medications\n* Study participant has evidence of a secondary cause of immune thrombocytopenia (clear association with other medical conditions, eg, untreated H. pylori infection, leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease or is drug induced), participant has a multiple immune cytopenia (eg, Evan's syndrome)\n* Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)\n* Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)\n* Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant\n* Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit\n* Study participant has a history of coagulopathy disorders other than ITP\n* Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA \\<50 mg/dL at the Screening Visit\n* Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit"}, 'identificationModule': {'nctId': 'NCT04200456', 'acronym': 'myOpportunITy1', 'briefTitle': 'A Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'UCB Pharma'}, 'officialTitle': 'A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)', 'orgStudyIdInfo': {'id': 'TP0003'}, 'secondaryIdInfos': [{'id': '2019-000884-26', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Rozanolixizumab', 'description': 'Study participants randomized to this arm will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.', 'interventionNames': ['Drug: Rozanolixizumab']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'Rozanolixizumab', 'type': 'DRUG', 'otherNames': ['UCB7665'], 'description': 'Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.', 'armGroupLabels': ['Rozanolixizumab']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Study participants receive placebo by subcutaneous infusion at pre-specified time points.', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '98104', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': 'Tp0003 50244', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'Tp0003 40188', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'city': 'Amiens', 'country': 'France', 'facility': 'Tp0003 40197', 'geoPoint': {'lat': 49.9, 'lon': 2.3}}, {'city': 'Pessac', 'country': 'France', 'facility': 'Tp0003 40196', 'geoPoint': {'lat': 44.80565, 'lon': -0.6324}}, {'city': 'Tbilisi', 'country': 'Georgia', 'facility': 'Tp0003 20050', 'geoPoint': {'lat': 41.69143, 'lon': 44.83412}}, {'city': 'Thessaloniki', 'country': 'Greece', 'facility': 'Tp0003 40558', 'geoPoint': {'lat': 40.64072, 'lon': 22.93493}}, {'city': 'Győr', 'country': 'Hungary', 'facility': 'Tp0003 40202', 'geoPoint': {'lat': 47.68333, 'lon': 17.63512}}, {'city': 'Nyíregyháza', 'country': 'Hungary', 'facility': 'Tp0003 40178', 'geoPoint': {'lat': 47.95539, 'lon': 21.71671}}, {'city': 'Pécs', 'country': 'Hungary', 'facility': 'Tp0003 40204', 'geoPoint': {'lat': 46.07617, 'lon': 18.22814}}, {'city': 'Florence', 'country': 'Italy', 'facility': 'Tp0003 40208', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'city': 'Chūōku', 'country': 'Japan', 'facility': 'Tp0003 20030', 'geoPoint': {'lat': 33.63867, 'lon': 130.67068}}, {'city': 'Iruma-gun', 'country': 'Japan', 'facility': 'Tp0003 20039'}, {'city': 'Shibuya-ku', 'country': 'Japan', 'facility': 'Tp0003 20159'}, {'city': 'Chisinau', 'country': 'Moldova', 'facility': 'Tp0003 20051', 'geoPoint': {'lat': 47.00902, 'lon': 28.85938}}, {'city': 'Gdansk', 'country': 'Poland', 'facility': 'Tp0003 40218', 'geoPoint': {'lat': 54.35227, 'lon': 18.64912}}, {'city': 'Lodz', 'country': 'Poland', 'facility': 'Tp0003 40221', 'geoPoint': {'lat': 51.77058, 'lon': 19.47395}}, {'city': 'Skorzewo', 'country': 'Poland', 'facility': 'Tp0003 40222', 'geoPoint': {'lat': 54.16909, 'lon': 17.97006}}, {'city': 'Bucharest', 'country': 'Romania', 'facility': 'Tp0003 40225', 'geoPoint': {'lat': 44.43225, 'lon': 26.10626}}, {'city': 'Bucharest', 'country': 'Romania', 'facility': 'Tp0003 40226', 'geoPoint': {'lat': 44.43225, 'lon': 26.10626}}, {'city': 'Saint Petersburg', 'country': 'Russia', 'facility': 'Tp0003 20055', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'city': 'Daegu', 'country': 'South Korea', 'facility': 'Tp0003 20218', 'geoPoint': {'lat': 35.87028, 'lon': 128.59111}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Tp0003 20207', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Taipei', 'country': 'Taiwan', 'facility': 'Tp0003 20099', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'city': 'Dnipropetrovsk', 'country': 'Ukraine', 'facility': 'Tp0003 20060', 'geoPoint': {'lat': 48.46664, 'lon': 35.04066}}, {'city': 'Ivano-Frankivsk', 'country': 'Ukraine', 'facility': 'Tp0003 20062', 'geoPoint': {'lat': 48.92312, 'lon': 24.71248}}, {'city': 'Kyiv', 'country': 'Ukraine', 'facility': 'Tp0003 20063', 'geoPoint': {'lat': 50.45466, 'lon': 30.5238}}, {'city': 'Zaporizhzhia', 'country': 'Ukraine', 'facility': 'Tp0003 20100', 'geoPoint': {'lat': 47.15214, 'lon': 35.74246}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Tp0003 40238', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Plymouth', 'country': 'United Kingdom', 'facility': 'Tp0003 40234', 'geoPoint': {'lat': 50.37153, 'lon': -4.14305}}], 'overallOfficials': [{'name': 'UCB Cares', 'role': 'STUDY_DIRECTOR', 'affiliation': '001 844 599 2273'}]}, 'ipdSharingStatementModule': {'url': 'https://www.Vivli.org', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'timeFrame': 'Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.', 'ipdSharing': 'YES', 'description': 'Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.', 'accessCriteria': 'Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'UCB Biopharma SRL', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}