Ignite Creation Date:
2025-12-24 @ 12:55 PM
Ignite Modification Date:
2026-01-07 @ 8:28 AM
Study NCT ID:
NCT05644561
Status:
RECRUITING
Last Update Posted:
2025-12-11
First Post:
2022-12-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009157', 'term': 'Myasthenia Gravis'}], 'ancestors': [{'id': 'D020361', 'term': 'Paraneoplastic Syndromes, Nervous System'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010257', 'term': 'Paraneoplastic Syndromes'}, {'id': 'D020274', 'term': 'Autoimmune Diseases of the Nervous System'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000629409', 'term': 'ravulizumab'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 12}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-06-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2028-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-05', 'studyFirstSubmitDate': '2022-12-01', 'studyFirstSubmitQcDate': '2022-12-01', 'lastUpdatePostDateStruct': {'date': '2025-12-11', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-12-09', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-07-15', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Serum Concentration of Ravulizumab', 'timeFrame': 'Day 1 predose through Week 18 predose'}, {'measure': 'Serum Free C5 Concentration', 'timeFrame': 'Day 1 predose through Week 18 predose'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18', 'timeFrame': 'Baseline, Up to Week 18'}, {'measure': 'Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18', 'timeFrame': 'Baseline, Up to Week 18'}, {'measure': 'Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18', 'timeFrame': 'Baseline, Up to Week 18'}, {'measure': 'Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18', 'timeFrame': 'Week 10, Up to Week 18'}, {'measure': 'Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18', 'timeFrame': 'Baseline, Up to Week 18', 'description': 'Participants ≥8 years of age will be evaluated.'}, {'measure': 'Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18', 'timeFrame': 'Baseline, Up to Week 18', 'description': 'Participants \\<8 years of age will be evaluated.'}, {'measure': 'Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18', 'timeFrame': 'Baseline through Week 18'}, {'measure': 'Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18', 'timeFrame': 'Baseline through Week 18'}, {'measure': 'Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline', 'timeFrame': 'Baseline through Week 18', 'description': 'Stable is defined as a ±5-point change from Baseline.'}, {'measure': 'Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline', 'timeFrame': 'Baseline through Week 18', 'description': 'Stable is defined as a ±3-point change from baseline.'}, {'measure': 'Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events', 'timeFrame': 'Baseline up to Week 126 (8 weeks after last dose of study drug)'}, {'measure': 'Number of Participants With Anti-Drug Antibody (ADA) at Week 18', 'timeFrame': 'Baseline through Week 18'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Generalized Myasthenia Gravis', 'gMG'], 'conditions': ['Generalized Myasthenia Gravis', 'gMG']}, 'descriptionModule': {'briefSummary': 'The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period\n* Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening\n* Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.\n* Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.\n* All participants must be vaccinated against meningococcal infection\n\nExclusion Criteria:\n\nMedical Conditions\n\n* Any untreated thymic malignancy, carcinoma, or thymoma.\n* Participants with a history of treated benign thymoma\n* History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening\n* History of N meningitidis infection\n* Known to be human immunodeficiency virus (HIV) positive\n* History of unexplained infections\n* Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period'}, 'identificationModule': {'nctId': 'NCT05644561', 'briefTitle': 'Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)', 'orgStudyIdInfo': {'id': 'ALXN1210-MG-319'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ravulizumab Intravenous (IV) Infusion', 'description': 'All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing \\< 20 kg, for a total of 122 weeks of treatment.', 'interventionNames': ['Drug: Ravulizumab']}], 'interventions': [{'name': 'Ravulizumab', 'type': 'DRUG', 'description': 'Ravulizumab will be administered by intravenous (IV) infusion.', 'armGroupLabels': ['Ravulizumab Intravenous (IV) Infusion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90078', 'city': 'Los Angeles', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94143', 'city': 'San Francisco', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '27514', 'city': 'Chapel Hill', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '44308', 'city': 'Akron', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 41.08144, 'lon': -81.51901}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '76208', 'city': 'Denton', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 33.21484, 'lon': -97.13307}}, {'zip': '13385', 'city': 'Marseille', 'status': 'RECRUITING', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'zip': '75571', 'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '20133', 'city': 'Milan', 'status': 'RECRUITING', 'country': 'Italy', 'facility': 'Research Site', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '00165', 'city': 'Roma', 'status': 'RECRUITING', 'country': 'Italy', 'facility': 'Research Site', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '10126', 'city': 'Torino', 'status': 'RECRUITING', 'country': 'Italy', 'facility': 'Research Site', 'geoPoint': {'lat': 44.88856, 'lon': 11.99138}}, {'zip': '173-0003', 'city': 'Itabashi-ku', 'status': 'RECRUITING', 'country': 'Japan', 'facility': 'Research Site'}, {'zip': '2333 ZA', 'city': 'Leiden', 'status': 'WITHDRAWN', 'country': 'Netherlands', 'facility': 'Research Site', 'geoPoint': {'lat': 52.15833, 'lon': 4.49306}}, {'zip': '11000', 'city': 'Belgrade', 'status': 'RECRUITING', 'country': 'Serbia', 'facility': 'Research Site', 'geoPoint': {'lat': 44.80401, 'lon': 20.46513}}, {'zip': '11070', 'city': 'Belgrade', 'status': 'RECRUITING', 'country': 'Serbia', 'facility': 'Research Site', 'geoPoint': {'lat': 44.80401, 'lon': 20.46513}}, {'zip': '28040', 'city': 'Madrid', 'status': 'WITHDRAWN', 'country': 'Spain', 'facility': 'Research Site', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '29010', 'city': 'Málaga', 'status': 'WITHDRAWN', 'country': 'Spain', 'facility': 'Research Site', 'geoPoint': {'lat': 36.72016, 'lon': -4.42034}}, {'zip': '3010', 'city': 'Bern', 'status': 'RECRUITING', 'country': 'Switzerland', 'facility': 'Research Site', 'geoPoint': {'lat': 46.94809, 'lon': 7.44744}}], 'centralContacts': [{'name': 'Alexion Pharmaceuticals, Inc. (Sponsor)', 'role': 'CONTACT', 'email': 'clinicaltrials@alexion.com', 'phone': '1-855-752-2356'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'ipdSharing': 'YES', 'description': 'Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}