Ignite Creation Date:
2025-12-24 @ 10:09 PM
Ignite Modification Date:
2025-12-30 @ 2:25 AM
Study NCT ID:
NCT04557735
Status:
COMPLETED
Last Update Posted:
2025-10-02
First Post:
2020-09-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['France', 'Germany'], 'submissionTracking': {'submissionInfos': [{'resetDate': '2025-12-05', 'mcpReleaseN': 22, 'releaseDate': '2025-11-25'}, {'releaseDate': '2025-12-23'}], 'estimatedResultsFirstSubmitDate': '2025-11-25'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D057049', 'term': 'Thrombotic Microangiopathies'}], 'ancestors': [{'id': 'D013921', 'term': 'Thrombocytopenia'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000095542', 'term': 'Cytopenia'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000629409', 'term': 'ravulizumab'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 41}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-12-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2025-05-27', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-10-01', 'studyFirstSubmitDate': '2020-09-02', 'studyFirstSubmitQcDate': '2020-09-18', 'lastUpdatePostDateStruct': {'date': '2025-10-02', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-09-22', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-11-26', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'TMA Response', 'timeFrame': '26 weeks (treatment period)'}], 'secondaryOutcomes': [{'measure': 'Time To TMA Response', 'timeFrame': '26 weeks (treatment period)'}, {'measure': 'TMA Relapse', 'timeFrame': '52 weeks (follow-up period)'}, {'measure': 'Overall Survival', 'timeFrame': '26 weeks (treatment period) and 52 weeks'}, {'measure': 'Proportion of Participants with Hematologic Response', 'timeFrame': '26 weeks (treatment period)', 'description': 'Hematologic Response is measured by improvement in TMA markers (LDH, platelets, and schistocytes).'}, {'measure': 'Proportion of Participants with Platelet Response', 'timeFrame': '26 weeks (treatment period)', 'description': 'Platelet Response is determined by improvement in platelet count without the need for platelet transfusion.'}, {'measure': 'Proportion of Participants with Change from Baseline in TMA-associated Organ Dysfunction', 'timeFrame': '26 weeks (treatment period) and 52 weeks', 'description': 'Changes from baseline in the renal system are measured by improvements in laboratory tests (eGFR, protein/creatinine ratio, and serum creatinine).\n\nChanges from baseline in the cardiovascular and pulmonary systems are measured by vital signs, Chest X-ray and/or Chest-CT, ECG, echocardiography, and changes in requirement for ventilatory/respiratory support.\n\nChanges from baseline in the CNS are measured by posterior reversible encephalopathy syndrome (PRES) status.\n\nChanges from baseline in the GI system are measured by reported AEs with GI involvement.'}, {'measure': 'Non-relapse mortality', 'timeFrame': '26 weeks (treatment period) and 52 weeks'}, {'measure': 'Time to TMA Response for each individual component of TMA Response for Participants who Demonstrate TMA Response', 'timeFrame': '26 weeks (treatment period)', 'description': 'Time to TMA Response is measured by monitoring the time it takes to meet the criteria for TMA Response, based on lab assessment for TMA markers.'}, {'measure': 'Time to Hematologic Response', 'timeFrame': '26 weeks (treatment period)'}, {'measure': 'Proportion of Participants with Hemoglobin Response', 'timeFrame': '26 weeks (treatment period)', 'description': 'Hemoglobin Response is measured by stability of hemoglobin confirmed by two laboratory assessments at least 24h apart, after the lack of need for RBC transfusion for at least 7 days.'}, {'measure': 'Proportion of Participants with Partial Response', 'timeFrame': '26 weeks (treatment period)', 'description': 'Partial response is when the participant meets at least one of the TMA Response criteria, but not all.'}, {'measure': 'Proportion of Participants with Loss of TMA Response', 'timeFrame': '26 weeks (treatment period)', 'description': 'Loss of TMA Response is measured in participants who met TMA Response criteria but no longer meet these criteria.'}, {'measure': 'Duration of TMA Response', 'timeFrame': '26 weeks (treatment period) and 52 weeks'}, {'measure': 'Proportion of Participants with Changes from Baseline in Selected Laboratory Tests', 'timeFrame': '26 weeks (treatment period) and 52 weeks', 'description': 'Changes from baseline in haptoglobin are measured by blood tests assessing haptoglobin levels.\n\nChanges from baseline in platelets are measured by blood tests assessing platelet counts.\n\nChanges from baseline in LDH are measured by blood tests assessing LDH levels. Changes from baseline in hemoglobin are measured by blood tests assessing hemoglobin levels.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Thrombotic Microangiopathy (TMA)', 'Ultomiris', 'Ravulizumab', 'Hematopoietic Stem Cell Transplant'], 'conditions': ['Thrombotic Microangiopathy']}, 'descriptionModule': {'briefSummary': 'This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to \\< 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. ≥ 28 days of age up to \\< 18 years of age at the time of signing the informed consent.\n2. Received HSCT within the past 12 months.\n3. Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.\n4. A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period.\n5. Body weight ≥ 5 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).\n6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.\n7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants \\<18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.\n8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent.\n\nExclusion Criteria:\n\n1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.\n2. Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.\n3. Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.\n4. Clinical diagnosis of disseminated intravascular coagulation (DIC).\n5. Known bone marrow/graft failure for the current HSCT.\n6. Diagnosis of veno-occlusive disease (VOD) which is unresolved at the time of Screening.\n7. Human immunodeficiency virus (HIV) infection.\n8. Unresolved meningococcal disease.\n9. Presence of sepsis requiring vasopressor support.\n10. Pregnancy or breastfeeding.\n11. Hypersensitivity to murine proteins or to 1 of the excipients of Ravulizumab.\n12. Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.\n13. Respiratory failure requiring mechanical ventilation.\n14. Previously or currently treated with a complement inhibitor.\n15. Participation in an interventional treatment study of any therapy for TMA.'}, 'identificationModule': {'nctId': 'NCT04557735', 'briefTitle': 'Study of Ravulizumab in Pediatric Participants With HSCT-TMA', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplantation (HSCT)', 'orgStudyIdInfo': {'id': 'ALXN1210-TMA-314'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ravulizumab plus Best Supportive Care', 'description': 'Participants will receive ravulizumab plus Best Supportive Care as background therapy.', 'interventionNames': ['Drug: Ravulizumab', 'Other: Best Supportive Care']}], 'interventions': [{'name': 'Ravulizumab', 'type': 'DRUG', 'otherNames': ['Ultomiris'], 'description': 'Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight.', 'armGroupLabels': ['Ravulizumab plus Best Supportive Care']}, {'name': 'Best Supportive Care', 'type': 'OTHER', 'description': 'Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).', 'armGroupLabels': ['Ravulizumab plus Best Supportive Care']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85724', 'city': 'Tucson', 'state': 'Arizona', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 32.22174, 'lon': -110.92648}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '75235', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '53792', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}, {'zip': '91096', 'city': 'Haifa', 'country': 'Israel', 'facility': 'Research Site', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'zip': '91120', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Research Site', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'zip': '4920235', 'city': 'Petah Tikva', 'country': 'Israel', 'facility': 'Research Site', 'geoPoint': {'lat': 32.08707, 'lon': 34.88747}}, {'zip': '5265601', 'city': 'Ramat Gan', 'country': 'Israel', 'facility': 'Research Site', 'geoPoint': {'lat': 32.08227, 'lon': 34.81065}}, {'zip': '00165', 'city': 'Roma', 'country': 'Italy', 'facility': 'Research Site', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '960-1295', 'city': 'Fukushima', 'country': 'Japan', 'facility': 'Research Site', 'geoPoint': {'lat': 37.75, 'lon': 140.46667}}, {'zip': '650-0047', 'city': 'Kobe', 'country': 'Japan', 'facility': 'Research Site', 'geoPoint': {'lat': 34.6913, 'lon': 135.183}}, {'zip': '466-8560', 'city': 'Nagoya', 'country': 'Japan', 'facility': 'Research Site', 'geoPoint': {'lat': 35.18147, 'lon': 136.90641}}, {'zip': '534-0021', 'city': 'Osaka', 'country': 'Japan', 'facility': 'Research Site', 'geoPoint': {'lat': 34.69379, 'lon': 135.50107}}, {'zip': '03080', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Research Site', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '5505', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Research Site', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '08041', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Research Site', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '8950', 'city': 'Esplugues de Llobregat', 'country': 'Spain', 'facility': 'Research Site', 'geoPoint': {'lat': 41.37732, 'lon': 2.08809}}, {'zip': '28046', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Research Site', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '37007', 'city': 'Salamanca', 'country': 'Spain', 'facility': 'Research Site', 'geoPoint': {'lat': 40.96882, 'lon': -5.66388}}, {'zip': 'B4 6NH', 'city': 'Birmingham', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'zip': 'BS2 8BJ', 'city': 'Bristol', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 51.45523, 'lon': -2.59665}}, {'zip': 'NE1 4LP', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}