A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
ziMyG
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
3 other identifiers
interventional
8
5 countries
9
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2024
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 20, 2023
CompletedFirst Posted
Study publicly available on registry
September 28, 2023
CompletedStudy Start
First participant enrolled
October 16, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 16, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 25, 2026
April 24, 2026
April 1, 2026
2.1 years
September 20, 2023
April 23, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Week 4 (Day 29)
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Week 4 (Day 29)
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
Samples for measurement of C5 will be collected on Day 29 predose.
Week 4 (Day 29)
Secondary Outcomes (9)
Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of treatment-emergent serious adverse events (TESAEs)
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)
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of treatment-emergent infections
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)
Week 4 (Day 29)
- +4 more secondary outcomes
Study Arms (1)
Zilucoplan Arm
EXPERIMENTALStudy participants will receive zilucoplan in pre-defined dose based on their weight.
Interventions
Zilucoplan will be administered subcutaneously to pediatric study participants.
Eligibility Criteria
You may qualify if:
- \- Participant must be 12 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation
- \- Participant must be 2 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
Mg0014 50168
Chicago, Illinois, 60611, United States
Mg0014 50574
Flower Mound, Texas, 75028, United States
Mg0014 40144
Milan, Italy
Mg0014 40774
Katowice, Poland
Mg0014 40218
Warsaw, Poland
Mg0014 20104
Seoul, South Korea
Mg0014 20220
Seoul, South Korea
Mg0014 40735
Glasgow, United Kingdom
Mg0014 40736
London, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
UCB Cares
001 844 599 2273
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 20, 2023
First Posted
September 28, 2023
Study Start
October 16, 2024
Primary Completion (Estimated)
November 16, 2026
Study Completion (Estimated)
December 25, 2026
Last Updated
April 24, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
- Time Frame
- Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
- Access Criteria
- Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.