Ignite Creation Date:
2025-12-26 @ 10:45 PM
Ignite Modification Date:
2026-01-17 @ 7:49 PM
Study NCT ID:
NCT06435312
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-12-19
First Post:
2024-05-23
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
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': 'C000719268', 'term': 'zilucoplan'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 8}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2024-11-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2027-12-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-18', 'studyFirstSubmitDate': '2024-05-23', 'studyFirstSubmitQcDate': '2024-05-23', 'lastUpdatePostDateStruct': {'date': '2025-12-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-05-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-10-26', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Occurence of treatment emergent adverse events during the course of the study', 'timeFrame': 'Baseline (Day 1) to Safety Follow-up (up to Week 60)', 'description': 'An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.'}, {'measure': 'Occurence of treatment-emergent serious adverse events (TESAEs)', 'timeFrame': 'Baseline (Day 1) to Safety Follow-up (up to Week 60)', 'description': 'A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose:\n\nResults in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.'}, {'measure': 'Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product', 'timeFrame': 'Baseline (Day 1) to Safety Follow-up (up to Week 60)', 'description': 'An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.'}, {'measure': 'Occurence of treatment-emergent infections', 'timeFrame': 'Baseline (Day 1) to Safety Follow-up (up to Week 60)', 'description': 'Percentage of participants who experienced treatment-emergent infections as adverse events.\n\nAn adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.'}], 'secondaryOutcomes': [{'measure': 'Plasma concentration of Zilucoplan at Week 52', 'timeFrame': 'Week 52', 'description': 'Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52.'}, {'measure': 'Sheep red blood cell (sRBC) lysis activity at Week 52', 'timeFrame': 'Week 52', 'description': 'Blood samples for measurement of sRBC lysis will be collected at Week 52.'}, {'measure': 'Blood complement component 5 (C5) levels at Week 52', 'timeFrame': 'Week 52', 'description': 'Blood samples for measurement of C5 will be collected at Week 52.'}, {'measure': 'Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52', 'timeFrame': 'Week 52', 'description': 'The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability.'}, {'measure': 'Quantitative Myasthenia Gravis (QMG) score at Week 52', 'timeFrame': 'Week 52', 'description': 'QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['generalized Myasthenia Gravis', 'gMG', 'RA101495', 'Zilucoplan', 'pediatric', 'MG0015'], 'conditions': ['Generalized Myasthenia Gravis']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nUnited States of America (USA) specific inclusion criterion:\n\n\\- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.\n\nRest of World (ROW) specific inclusion criterion:\n\n\\- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.\n\nGlobal specific inclusion criteria:\n\n* Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion\n* Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care\n\nExclusion Criteria:\n\n* Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP\n* Participant has known positive serology for muscle-specific kinase\n* Participant has known hypersensitivity to any components of the IMP\n* Participant has a prior history of meningococcal disease'}, 'identificationModule': {'nctId': 'NCT06435312', 'acronym': 'ziMyG+', 'briefTitle': 'An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis', 'organization': {'class': 'INDUSTRY', 'fullName': 'UCB Pharma'}, 'officialTitle': 'An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis', 'orgStudyIdInfo': {'id': 'MG0015'}, 'secondaryIdInfos': [{'id': '2022-502073-42', 'type': 'REGISTRY', 'domain': 'EU Clinical Trials'}, {'id': 'U1111-1290-3806', 'type': 'OTHER', 'domain': 'World Health Organization (WHO)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Zilucoplan Arm', 'description': 'Study participants will receive Zilucoplan at a pre-defined dose based on their weight.', 'interventionNames': ['Drug: Zilucoplan']}], 'interventions': [{'name': 'Zilucoplan', 'type': 'DRUG', 'otherNames': ['RA101495'], 'description': 'Zilucoplan will be administered subcutaneously to pediatric study participants', 'armGroupLabels': ['Zilucoplan Arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Mg0015 50168', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '75028', 'city': 'Flower Mound', 'state': 'Texas', 'country': 'United States', 'facility': 'Mg0015 50574', 'geoPoint': {'lat': 33.01457, 'lon': -97.09696}}, {'city': 'Milan', 'country': 'Italy', 'facility': 'Mg0015 40144', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'city': 'Katowice', 'country': 'Poland', 'facility': 'Mg0015 40774', 'geoPoint': {'lat': 50.2597, 'lon': 19.02173}}, {'city': 'Warsaw', 'country': 'Poland', 'facility': 'Mg0015 40218', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Mg0015 20104', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Mg0015 20220', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Glasgow', 'country': 'United Kingdom', 'facility': 'Mg0015 40735', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Mg0015 40736', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'UCB Cares', 'role': 'STUDY_DIRECTOR', 'affiliation': '001 844 599 2273 (UCB)'}]}, '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'}}}}