An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Zilucoplan.
- Who it may be relevant to
- Registry conditions: Generalized Myasthenia Gravis. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy, Poland, South Korea, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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
Interventions
- Drug Zilucoplan
Zilucoplan will be administered subcutaneously to pediatric study participants
Primary outcome measures
- Occurence of treatment emergent adverse events during the course of the study [Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
- Occurence of treatment-emergent serious adverse events (TESAEs) [Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
- Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product [Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
- Occurence of treatment-emergent infections [Time frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)]
Secondary outcome measures (5)
- Plasma concentration of Zilucoplan at Week 52 [Time frame: Week 52]
- Sheep red blood cell (sRBC) lysis activity at Week 52 [Time frame: Week 52]
- Blood complement component 5 (C5) levels at Week 52 [Time frame: Week 52]
- Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52 [Time frame: Week 52]
- Quantitative Myasthenia Gravis (QMG) score at Week 52 [Time frame: Week 52]
Eligibility criteria
Inclusion criteria
United States of America (USA) specific inclusion criterion:
\- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.
Rest of World (ROW) specific inclusion criterion:
\- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.
Global specific inclusion criteria:
- 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
- Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care
Exclusion criteria
- Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
- Participant has known positive serology for muscle-specific kinase
- Participant has known hypersensitivity to any components of the IMP
- Participant has a prior history of meningococcal disease
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Poland · 2 centers
- Mg0015 40774 — Katowice
- Mg0015 40218 — Warsaw
United Kingdom · 2 centers
- Mg0015 40735 — Glasgow
- Mg0015 40736 — London
Italy · 1 center
- Mg0015 40144 — Milan
South Korea · 1 center
- Mg0015 20220 — Seoul
Identifiers
NCT: NCT06435312 · MG0015 · 2022-502073-42 · U1111-1290-3806