An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

NCT06435312 | Enrolling By Invitation Phase 3 FDA Drug

• 3 other identifiers: MG00152022-502073-42U1111-1290-3806
• Study Type: interventional
• Target: 8
• Locations: 4 countries
• Sites: 7

Brief Summary

The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

Conditions

Generalized Myasthenia Gravis

Keywords

generalized Myasthenia Gravis; gMG; RA101495; Zilucoplan; pediatric; MG0015

Outcome Measures

Primary Outcomes (4)

• Occurence of treatment emergent adverse events during the course of the study

  An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

  Baseline (Day 1) to Safety Follow-up (up to Week 60)

• Occurence of treatment-emergent serious adverse events (TESAEs)

  A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose: Results in death Is life-threatening Requires inpatient hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.

  Baseline (Day 1) to Safety Follow-up (up to Week 60)

• Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product

  An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.

  Baseline (Day 1) to Safety Follow-up (up to Week 60)

• Occurence of treatment-emergent infections

  Percentage of participants who experienced treatment-emergent infections as adverse events. An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment.

  Baseline (Day 1) to Safety Follow-up (up to Week 60)

Secondary Outcomes (5)

• Plasma concentration of Zilucoplan at Week 52

  Week 52

• Sheep red blood cell (sRBC) lysis activity at Week 52

  Week 52

• Blood complement component 5 (C5) levels at Week 52

  Week 52

• Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52

  Week 52

• Quantitative Myasthenia Gravis (QMG) score at Week 52

  Week 52

Study Arms (1)

Zilucoplan Arm

EXPERIMENTAL

Study participants will receive Zilucoplan at a pre-defined dose based on their weight.

Drug: Zilucoplan

Interventions

Zilucoplan DRUG

Zilucoplan will be administered subcutaneously to pediatric study participants

Also known as: RA101495

Zilucoplan Arm

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• \- Participant must be ≥ 12 years of age at the time of signing the Informed Consent/Assent according to local regulation.
• \- Participant must be ≥ 2 years of age at the time of signing the Informed Consent/Assent according to local regulation.
• Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion
• Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care

You may not qualify if:

• Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP
• Participant has known positive serology for muscle-specific kinase
• Participant has known hypersensitivity to any components of the IMP
• Participant has a prior history of meningococcal disease

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (7)

Mg0015 40144

Milan, Italy

Location

Mg0015 40774

Katowice, Poland

Location

Mg0015 40218

Warsaw, Poland

Location

Mg0015 20104

Seoul, South Korea

Location

Mg0015 20220

Seoul, South Korea

Location

Mg0015 40735

Glasgow, United Kingdom

Location

Mg0015 40736

London, United Kingdom

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

zilucoplan

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• UCB Cares

  001 844 599 2273 (UCB)

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2024
• First Posted: May 30, 2024
• Study Start: November 19, 2024
• Primary Completion (Estimated): October 26, 2027
• Study Completion (Estimated): November 19, 2027
• 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.

Locations

GB (2); PL (2); KR (2); IT (1)