Evaluating Zilucoplan for Pediatric Myasthenia Gravis
A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
This study is testing a new medication called zilucoplan to see how it works and if it's safe for children aged 2 to 18 with generalized myasthenia gravis.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 8 (estimated) |
| Ages | 12 Years to 17 Years |
| Sex | All |
| Sponsor | UCB Pharma Industry-sponsored |
| Locations | 9 sites (Chicago, Illinois and 8 other locations) |
| Trial ID | NCT06055959 on ClinicalTrials.gov |
What this trial studies
This study evaluates the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity, and activity of zilucoplan in pediatric participants diagnosed with generalized myasthenia gravis (gMG). It includes participants aged 2 to 18 years, with specific inclusion criteria based on age and severity of their condition. The study aims to gather comprehensive data on how zilucoplan affects children with gMG, potentially leading to improved treatment options.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients aged 2 to 18 years with a confirmed diagnosis of generalized myasthenia gravis.
Not a fit: Patients with mild forms of myasthenia gravis or those not meeting the specific inclusion criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for children suffering from generalized myasthenia gravis.
How similar studies have performed: Other studies have shown promise in treating generalized myasthenia gravis with similar approaches, but this specific evaluation of zilucoplan in pediatric patients is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: United States of America (USA) specific inclusion criterion: \- Participant must be 12 to \<18 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 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation Global inclusion criteria: * Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening * Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening * Participants with gMG, including: * An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to \<18 years of age at Screening * Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to \<12 years of age at Screening (does not apply to US) * Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s) Exclusion Criteria: * Participant has known positive serology for muscle-specific kinase * Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study * Participant has had a thymectomy within 6 months prior to Baseline * Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator * Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline
Where this trial is running
Chicago, Illinois and 8 other locations
- Mg0014 50168 — Chicago, Illinois, United States (Withdrawn)
- Mg0014 50574 — Denton, Texas, United States (Withdrawn)
- Mg0014 40144 — Milan, Italy (Recruiting)
- Mg0014 40774 — Katowice, Poland (Recruiting)
- Mg0014 40218 — Warsaw, Poland (Active_not_recruiting)
- Mg0014 20104 — Seoul, South Korea (Recruiting)
- Mg0014 20220 — Seoul, South Korea (Recruiting)
- Mg0014 40735 — Glasgow, United Kingdom (Recruiting)
- Mg0014 40736 — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: UCB Cares
- Email: ucbcares@ucb.com
- Phone: +18445992273 (USA)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.