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

May 7, 2026 updated by: UCB Biopharma SRL

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

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

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

8

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Milan, Italy
        • Mg0015 40144
  • Poland
      • Katowice, Poland
        • Mg0015 40774
      • Warsaw, Poland
        • Mg0015 40218
  • South Korea
      • Seoul, South Korea
        • Mg0015 20104
      • Seoul, South Korea
        • Mg0015 20220
  • United Kingdom
      • Glasgow, United Kingdom
        • Mg0015 40735
      • London, United Kingdom
        • Mg0015 40736

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

United States of America (USA) specific inclusion criterion:

- Participant must be ≥ 12 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 years of age at the time of signing the Informed Consent/Assent according to local regulation.

Global specific inclusion criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Zilucoplan Arm
Study participants will receive Zilucoplan at a pre-defined dose based on their weight.
Drug: Zilucoplan
Zilucoplan will be administered subcutaneously to pediatric study participants
Other Names:
  • RA101495

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurence of treatment emergent adverse events during the course of the study
Time Frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)
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)
Time Frame: Baseline (Day 1) to Safety Follow-up (up to Week 60)

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

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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma concentration of Zilucoplan at Week 52
Time Frame: Week 52
Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52.
Week 52
Sheep red blood cell (sRBC) lysis activity at Week 52
Time Frame: Week 52
Blood samples for measurement of sRBC lysis will be collected at Week 52.
Week 52
Blood complement component 5 (C5) levels at Week 52
Time Frame: Week 52
Blood samples for measurement of C5 will be collected at Week 52.
Week 52
Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52
Time Frame: Week 52
The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability.
Week 52
Quantitative Myasthenia Gravis (QMG) score at Week 52
Time Frame: Week 52
QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.
Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

UCB Biopharma SRL

Investigators

  • Study Director: UCB Cares, 001 844 599 2273 (UCB)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 19, 2024

Primary Completion (Estimated)

October 26, 2027

Study Completion (Estimated)

November 19, 2027

Study Registration Dates

First Submitted

May 23, 2024

First Submitted That Met QC Criteria

May 23, 2024

First Posted (Actual)

May 30, 2024

Study Record Updates

Last Update Posted (Actual)

May 8, 2026

Last Update Submitted That Met QC Criteria

May 7, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MG0015
  • 2022-502073-42 (Registry Identifier: EU Clinical Trials)
  • U1111-1290-3806 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing 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.

IPD Sharing 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.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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