Clinical trial NCT06435312: An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
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
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: generalized Myasthenia Gravis
gMG
RA101495
Zilucoplan
pediatric
MG0015
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2024-05-30
Enrollment: 8
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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


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

Primary outcomes:
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)
measure: Occurence of treatment-emergent serious adverse events (TESAEs)
timeFrame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
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)
measure: Occurence of treatment-emergent infections
timeFrame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
Secondary outcomes:
measure: Plasma concentration of Zilucoplan at Week 52
timeFrame: Week 52
measure: Sheep red blood cell (sRBC) lysis activity at Week 52
timeFrame: Week 52
measure: Blood complement component 5 (C5) levels at Week 52
timeFrame: Week 52
measure: Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52
timeFrame: Week 52
measure: Quantitative Myasthenia Gravis (QMG) score at Week 52
timeFrame: Week 52
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Mg0015 50168
state: Illinois
city: Denton
country: United States
facility: Mg0015 50574
state: Texas
city: Milano
country: Italy
facility: Mg0015 40144
city: Seoul
country: Korea, Republic of
facility: Mg0015 20104
city: Seoul
country: Korea, Republic of
facility: Mg0015 20220
city: Katowice
country: Poland
facility: Mg0015 40774
city: Warszawa
country: Poland
facility: Mg0015 40218
city: Glasgow
country: United Kingdom
facility: Mg0015 40735
city: London
country: United Kingdom
facility: Mg0015 40736
Study officials:
affiliation: 001 844 599 2273 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References: