Ignite Creation Date:
2025-12-24 @ 8:03 PM
Ignite Modification Date:
2025-12-28 @ 9:09 PM
Study NCT ID:
NCT05131204
Status:
TERMINATED
Last Update Posted:
2025-04-08
First Post:
2021-11-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2024-08-01', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D006457', 'term': 'Hemoglobinuria, Paroxysmal'}], 'ancestors': [{'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C481642', 'term': 'eculizumab'}, {'id': 'C000629409', 'term': 'ravulizumab'}]}}, '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}, 'limitationsAndCaveats': {'description': '20Dec2022 - Due to feasibility challenges related to enrollment and not due to efficacy or safety concerns, the sponsor terminated the study early and discontinued participant enrollment.'}}, 'adverseEventsModule': {'timeFrame': 'From first dose to week 29', 'eventGroups': [{'id': 'EG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1', 'otherNumAtRisk': 2, 'deathsNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1', 'otherNumAtRisk': 1, 'deathsNumAtRisk': 1, 'otherNumAffected': 1, 'seriousNumAtRisk': 1, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Injection site rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Blood glucose increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}, {'term': 'Eczema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 2, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 1, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (26.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percent Change in Lactate Dehydrogenase (LDH) From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Transfusion Avoidance After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Participants who do not receive a red blood cell (RBC) transfusion as per protocol algorithm based on post baseline hemoglobin values', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Transfusion Avoidance From Week 4 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 4 through week 36', 'description': 'Participants who do not receive an RBC transfusion as per protocol algorithm based on post baseline hemoglobin values', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Breakthrough Hemolysis After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Breakthrough Hemolysis From Week 4 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 4 (day 29) through week 36', 'description': 'Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Hemoglobin Stabilization After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Hemoglobin Stabilization From Week 4 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 4 (day 29) through week 36', 'description': 'Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Adequate Control of LDH From Week 8 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 8 (day 57) through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Adequate Control of LDH After Day 1 Though Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Maintained Adequate Control of Hemolysis From Week 8 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 8 through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Maintained Adequate Control of Hemolysis After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Normalization of LDH From Week 8 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 8 (day 57) through week 36', 'description': 'Percentage of participants with normalization of LDH as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Normalization of LDH After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Percentage of participants with normalization of LDH as defined in the protocol', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Change in Fatigue as Measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'description': "The FACIT-Fatigue is a 13 item, self-administered clinical outcome assessment (COA) assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-Fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.", 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Change in Physical Function (PF) Score on the European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire Core 30 Items (EORTC-QLQ-C30) From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'description': 'EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Change in Global Health Status (GHS)/QoL Scale Score on the EORTC-QLQ-C30 From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'description': 'EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints..'}, {'type': 'SECONDARY', 'title': 'Rate of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Per protocol algorithm', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Rate of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 4 through week 36', 'description': 'Per protocol algorithm', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Number of Units of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Day 1 through week 36', 'description': 'Per protocol algorithm', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Number of Units of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Week 4 through week 36', 'description': 'Per protocol algorithm', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Change in Hemoglobin Levels From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'description': 'Per protocol algorithm', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Incidence of Treatment Emergent Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to week 29', 'description': 'Treatment period and safety follow up period', 'unitOfMeasure': 'Events', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Treatment-emergent Adverse Events (TEAEs) of Special Interest', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to week 29', 'description': 'Treatment period and safety follow up period', 'unitOfMeasure': 'Events', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of TEAEs Leading to Treatment Discontinuation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Up to week 29', 'description': 'Treatment period and safety follow up period', 'unitOfMeasure': 'Events', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in Total CH50 From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Percent Change in Total CH50 From Baseline to Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'From baseline to week 36', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Concentration of Total C5 in Plasma Through Week 62', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Concentrations of Total Pozelimab in Serum Through Week 62', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Concentrations of Total Cemdisiran in Plasma Through Week 32', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 32', 'description': 'Treatment period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Concentrations of Total Eculizumab in Serum Through Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 40', 'description': 'Treatment period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Concentrations of Total Ravulizumab in Plasma Through Week 44', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 44', 'description': 'Treatment period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Incidence of Treatment Emergent Anti-drug Antibodies (ADAs) to Pozelimab Through Week 62', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}, {'type': 'SECONDARY', 'title': 'Incidence of Treatment Emergent ADAs to Cemdisiran Through Week 62', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'OG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'FG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Sponsor request', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'A total of 140 participants were expected to be enrolled, however, due to feasibility: five participants were screened and 3 were randomized and treated. There were 2 screen failures.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1'}, {'id': 'BG001', 'title': 'Anti-C5 Standard-of-care', 'description': 'Randomized 1:1'}, {'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': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Black or African American', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Asian', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'American Indian or Alaska Native', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Other', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Unknown', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Not Reported', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-06-21', 'size': 1395972, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-07-09T17:20', 'hasProtocol': True}, {'date': '2023-07-22', 'size': 577194, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-07-09T17:20', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'whyStopped': 'Sponsor decision not related to efficacy or safety.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2022-10-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2023-07-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-04-03', 'studyFirstSubmitDate': '2021-11-12', 'resultsFirstSubmitDate': '2024-07-09', 'studyFirstSubmitQcDate': '2021-11-12', 'lastUpdatePostDateStruct': {'date': '2025-04-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-08-22', 'studyFirstPostDateStruct': {'date': '2021-11-23', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-09-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-07-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent Change in Lactate Dehydrogenase (LDH) From Baseline to Week 36', 'timeFrame': 'From baseline to week 36'}], 'secondaryOutcomes': [{'measure': 'Percentage of Participants With Transfusion Avoidance After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Participants who do not receive a red blood cell (RBC) transfusion as per protocol algorithm based on post baseline hemoglobin values'}, {'measure': 'Percentage of Participants With Transfusion Avoidance From Week 4 Through Week 36', 'timeFrame': 'Week 4 through week 36', 'description': 'Participants who do not receive an RBC transfusion as per protocol algorithm based on post baseline hemoglobin values'}, {'measure': 'Percentage of Participants With Breakthrough Hemolysis After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol'}, {'measure': 'Percentage of Participants With Breakthrough Hemolysis From Week 4 Through Week 36', 'timeFrame': 'Week 4 (day 29) through week 36', 'description': 'Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol'}, {'measure': 'Percentage of Participants With Hemoglobin Stabilization After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol'}, {'measure': 'Percentage of Participants With Hemoglobin Stabilization From Week 4 Through Week 36', 'timeFrame': 'Week 4 (day 29) through week 36', 'description': 'Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol'}, {'measure': 'Percentage of Participants With Adequate Control of LDH From Week 8 Through Week 36', 'timeFrame': 'Week 8 (day 57) through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol'}, {'measure': 'Percentage of Participants With Adequate Control of LDH After Day 1 Though Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol'}, {'measure': 'Percentage of Participants Who Maintained Adequate Control of Hemolysis From Week 8 Through Week 36', 'timeFrame': 'Week 8 through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol'}, {'measure': 'Percentage of Participants Who Maintained Adequate Control of Hemolysis After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Percentage of participants with adequate control of LDH as defined in the protocol'}, {'measure': 'Percentage of Participants With Normalization of LDH From Week 8 Through Week 36', 'timeFrame': 'Week 8 (day 57) through week 36', 'description': 'Percentage of participants with normalization of LDH as defined in the protocol'}, {'measure': 'Percentage of Participants With Normalization of LDH After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Percentage of participants with normalization of LDH as defined in the protocol'}, {'measure': 'Change in Fatigue as Measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline to Week 36', 'timeFrame': 'From baseline to week 36', 'description': "The FACIT-Fatigue is a 13 item, self-administered clinical outcome assessment (COA) assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-Fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue."}, {'measure': 'Change in Physical Function (PF) Score on the European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire Core 30 Items (EORTC-QLQ-C30) From Baseline to Week 36', 'timeFrame': 'From baseline to week 36', 'description': 'EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."'}, {'measure': 'Change in Global Health Status (GHS)/QoL Scale Score on the EORTC-QLQ-C30 From Baseline to Week 36', 'timeFrame': 'From baseline to week 36', 'description': 'EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."'}, {'measure': 'Rate of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Per protocol algorithm'}, {'measure': 'Rate of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36', 'timeFrame': 'Week 4 through week 36', 'description': 'Per protocol algorithm'}, {'measure': 'Number of Units of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36', 'timeFrame': 'Day 1 through week 36', 'description': 'Per protocol algorithm'}, {'measure': 'Number of Units of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36', 'timeFrame': 'Week 4 through week 36', 'description': 'Per protocol algorithm'}, {'measure': 'Change in Hemoglobin Levels From Baseline to Week 36', 'timeFrame': 'From baseline to week 36', 'description': 'Per protocol algorithm'}, {'measure': 'Incidence of Treatment Emergent Serious Adverse Events (SAEs)', 'timeFrame': 'Up to week 29', 'description': 'Treatment period and safety follow up period'}, {'measure': 'Incidence of Treatment-emergent Adverse Events (TEAEs) of Special Interest', 'timeFrame': 'Up to week 29', 'description': 'Treatment period and safety follow up period'}, {'measure': 'Incidence of TEAEs Leading to Treatment Discontinuation', 'timeFrame': 'Up to week 29', 'description': 'Treatment period and safety follow up period'}, {'measure': 'Change in Total CH50 From Baseline to Week 36', 'timeFrame': 'From baseline to week 36'}, {'measure': 'Percent Change in Total CH50 From Baseline to Week 36', 'timeFrame': 'From baseline to week 36'}, {'measure': 'Concentration of Total C5 in Plasma Through Week 62', 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period'}, {'measure': 'Concentrations of Total Pozelimab in Serum Through Week 62', 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period'}, {'measure': 'Concentrations of Total Cemdisiran in Plasma Through Week 32', 'timeFrame': 'Through week 32', 'description': 'Treatment period'}, {'measure': 'Concentrations of Total Eculizumab in Serum Through Week 40', 'timeFrame': 'Through week 40', 'description': 'Treatment period'}, {'measure': 'Concentrations of Total Ravulizumab in Plasma Through Week 44', 'timeFrame': 'Through week 44', 'description': 'Treatment period'}, {'measure': 'Incidence of Treatment Emergent Anti-drug Antibodies (ADAs) to Pozelimab Through Week 62', 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period'}, {'measure': 'Incidence of Treatment Emergent ADAs to Cemdisiran Through Week 62', 'timeFrame': 'Through week 62', 'description': 'Treatment period and safety follow up period'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['PNH'], 'conditions': ['Paroxysmal Nocturnal Hemoglobinuria']}, 'referencesModule': {'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:\n\nTo evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy\n\nThe secondary objectives of the study are to:\n\n* Evaluate the effect of pozelimab and cemdisiran combination treatment versus anti-C5 standard-of-care treatment (eculizumab or ravulizumab) on the following:\n\n * Transfusion requirements and transfusion parameters\n * Measures of hemolysis: LDH control, breakthrough hemolysis, and inhibition of CH50\n * Hemoglobin levels\n * Fatigue as assessed by Clinical Outcome Assessments (COAs)\n * Health-related quality of life (HRQoL) as assessed by COAs\n * Safety and tolerability\n* To assess the concentrations of total pozelimab and either total eculizumab or total ravulizumab in serum and total cemdisiran and total C5 protein in plasma\n* To assess the immunogenicity of pozelimab and cemdisiran'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing\n2. Treated with eculizumab or ravulizumab prior to screening visit as described in the protocol Note: Biosimilars are not permitted, unless approved by the Sponsor\n\nKey Exclusion Criteria:\n\n1. Patients with a screening LDH \\>1.5 × ULN who have not taken their C5 inhibitor within the labeled dose interval at the dose prior to the screening LDH assessment\n2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant\n3. Body weight \\< 40 kilograms at screening visit\n4. Any use of complement inhibitor therapy other than eculizumab or ravulizumab in the 26 weeks prior to the screening visit or planned use during the study with the exception of study treatments\n5. Not meeting meningococcal vaccination requirements for eculizumab or ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit.\n6. Any contraindication for receiving Neisseria meningitidis vaccination.\n7. Positive for hepatitis B, and/ or hepatitis C as described in the protocol\n8. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer\n9. Participation in another interventional clinical study (except R3918-PNH-2021) or use of any experimental therapy within 30 days before screening visit or within 5 half-lives of that investigational product, whichever is greater, with the exception of eculizumab or ravulizumab.\n10. Patients with functional or anatomic asplenia\n\nNote: Other protocol-defined Inclusion/ Exclusion Criteria apply'}, 'identificationModule': {'nctId': 'NCT05131204', 'acronym': 'ACCESS 2', 'briefTitle': 'Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria', 'organization': {'class': 'INDUSTRY', 'fullName': 'Regeneron Pharmaceuticals'}, 'officialTitle': 'A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Currently Treated With Eculizumab or Ravulizumab', 'orgStudyIdInfo': {'id': 'R3918-PNH-2022'}, 'secondaryIdInfos': [{'id': '2020-002761-33', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Pozelimab and Cemdisiran', 'description': 'Randomized 1:1', 'interventionNames': ['Drug: Cemdisiran', 'Drug: Eculizumab', 'Drug: Pozelimab', 'Drug: Ravulizumab']}, {'type': 'EXPERIMENTAL', 'label': 'Anti-C5 standard-of-care', 'description': 'Randomized 1:1', 'interventionNames': ['Drug: Eculizumab', 'Drug: Ravulizumab']}], 'interventions': [{'name': 'Cemdisiran', 'type': 'DRUG', 'otherNames': ['ALN-CC5'], 'description': 'Administered per protocol', 'armGroupLabels': ['Pozelimab and Cemdisiran']}, {'name': 'Eculizumab', 'type': 'DRUG', 'otherNames': ['Soliris'], 'description': 'Administered per protocol', 'armGroupLabels': ['Anti-C5 standard-of-care', 'Pozelimab and Cemdisiran']}, {'name': 'Pozelimab', 'type': 'DRUG', 'otherNames': ['REGN3918'], 'description': 'Administered per protocol', 'armGroupLabels': ['Pozelimab and Cemdisiran']}, {'name': 'Ravulizumab', 'type': 'DRUG', 'otherNames': ['ALXN1210', 'Ultomiris'], 'description': 'Administered per protocol', 'armGroupLabels': ['Anti-C5 standard-of-care', 'Pozelimab and Cemdisiran']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90603', 'city': 'Whittier', 'state': 'California', 'country': 'United States', 'facility': 'Regeneron Research Facility', 'geoPoint': {'lat': 33.97918, 'lon': -118.03284}}], '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': 'When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.', 'ipdSharing': 'YES', 'description': 'All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing', 'accessCriteria': "Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Regeneron Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}