A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia… (NCT06055959) | Clinical Trial Compass
RecruitingPhase 2/3
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
United States, Italy, Poland8 participantsStarted 2024-10-16
Plain-language 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).
Who can participate
Age range12 Years – 17 Years
SexALL
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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 jeo…
What they're measuring
1
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Timeframe: Week 4 (Day 29)
2
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Timeframe: Week 4 (Day 29)
3
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)