Actively Recruiting
A Multicenter Open-Label Study to Evaluate Subcutaneous Zilucoplan in Children and Adolescents With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
Led by UCB Biopharma SRL · Updated on 2026-05-08
8
Participants Needed
9
Research Sites
5 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the effects of the drug zilucoplan in children and adolescents with generalized myasthenia gravis (gMG), a condition confirmed by specific antibody tests. The study aims to evaluate how the drug behaves in the body, its safety, tolerability, immune response, and activity in pediatric patients between 2 and 18 years old with this condition. This trial is in phases 2 and 3 and sponsored by UCB Biopharma SRL. Participants will receive zilucoplan through subcutaneous injections, with doses adjusted according to their weight. The treatment period includes monitoring plasma drug levels and immune markers at week 4. The study is open-label and uncontrolled, meaning all participants receive the drug. Vaccination against meningococcal infections and antibiotic prophylaxis are part of the preparatory measures. During the trial, participants will undergo assessments including blood tests to measure drug concentration and immune activity, as well as evaluations of muscle strength and daily living activities related to myasthenia gravis. Safety is monitored through tracking adverse events until up to 15 weeks after starting treatment. Quality of life and clinical status changes will also be recorded. The study is expected to continue until December 2026.
CONDITIONS
Brief Title
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant aged 12 to less than 18 years in the USA or 2 to less than 18 years elsewhere at time of consent
- Diagnosed with generalized myasthenia gravis confirmed by positive acetylcholine receptor antibody test
- Meets Myasthenia Gravis Foundation of America Clinical Classification II to IV
- MG-activities of daily living total score of 6 or more for adolescents 12 to less than 18 years
- Documented weakness in at least one limb, neck, or bulbar muscle for children 2 to less than 12 years (not applicable in USA)
- Documented vaccination against meningococcal infections within 3 years or willing to receive prophylactic antibiotics until 2 weeks after initial vaccine dose
You will not qualify if you...
- Known positive serology for muscle-specific kinase
- Medical or psychiatric condition that could affect ability to participate
- Thymectomy within 6 months before baseline
- Minimal manifestation status of myasthenia gravis
- Current or recent systemic infection within 2 weeks prior to baseline or infection needing intravenous antibiotics within 4 weeks prior to baseline
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Screening and enrollment visit
Duration - 4 weeks
Participants receive subcutaneous zilucoplan according to a predefined dose based on their weight.
Visits at baseline and at Week 4 (Day 29)
Duration - Up to 11 weeks after treatment
Participants are monitored for safety and any adverse events after treatment ends.
Safety follow-up visits up to Week 15
Trial Site Locations
Total: 9 locations
1
Mg0014 50168
Chicago, Illinois, United States, 60611
Withdrawn
2
Mg0014 50574
Denton, Texas, United States, 76208
Withdrawn
3
Mg0014 40144
Milan, Italy
Actively Recruiting
4
Mg0014 40774
Katowice, Poland
Actively Recruiting
5
Mg0014 40218
Warsaw, Poland
Active, Not Recruiting
6
Mg0014 20104
Seoul, South Korea
Actively Recruiting
7
Mg0014 20220
Seoul, South Korea
Actively Recruiting
8
Mg0014 40735
Glasgow, United Kingdom
Actively Recruiting
9
Mg0014 40736
London, United Kingdom
Actively Recruiting
Research Team
U
UCB Cares
U
UCB Cares
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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