A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Zilucoplan.
- Who it may be relevant to
- Registry conditions: Generalized Myasthenia Gravis. Basic parameters: 12 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Italy, Poland, South Korea, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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).
Interventions
- Drug Zilucoplan
Zilucoplan will be administered subcutaneously to pediatric study participants.
Primary outcome measures
- Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
- Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
- Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
Secondary outcome measures (9)
- Occurence of treatment-emergent adverse events (TEAEs) during the course of the study [Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
- Occurrence of treatment-emergent serious adverse events (TESAEs) [Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
- Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP) [Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
- Occurrence of treatment-emergent infections [Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
- Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
- Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29). [Time frame: Week 4 (Day 29)]
- Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
- Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
- Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29) [Time frame: Week 4 (Day 29)]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Mg0014 50168 — Chicago
- Mg0014 50574 — Flower Mound
Poland · 2 centers
- Mg0014 40774 — Katowice
- Mg0014 40218 — Warsaw
South Korea · 2 centers
- Mg0014 20104 — Seoul
- Mg0014 20220 — Seoul
United Kingdom · 2 centers
- Mg0014 40735 — Glasgow
- Mg0014 40736 — London
Italy · 1 center
- Mg0014 40144 — Milan
Identifiers
NCT: NCT06055959 · MG0014 · U1111-1290-3349 · 2022-502072-23