Zilucoplan: Zilucoplan will be administered subcutaneously to pediatric study participants.
Study summary
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
Eligibility
Sex
ALL
Min age
12 Years
Max age
17 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) — Week 4 (Day 29) Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) — Week 4 (Day 29) Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29) — Week 4 (Day 29) Samples for measurement of C5 will be collected on Day 29 predose.
Trial sites (9)
Facility
City
Region
Status
Mg0014 50168
Chicago
Illinois
Withdrawn
Mg0014 50574
Flower Mound
Texas
Withdrawn
Mg0014 40144
Milan
Italy
Recruiting
Mg0014 40774
Katowice
Poland
Recruiting
Mg0014 40218
Warsaw
Poland
Active Not Recruiting
Mg0014 20104
Seoul
South Korea
Withdrawn
Mg0014 20220
Seoul
South Korea
Recruiting
Mg0014 40735
Glasgow
United Kingdom
Recruiting
Mg0014 40736
London
United Kingdom
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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