Skip to main content
OpenTrials
Enrolling by Invitation

NCT Number: NCT06435312

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

Enrolling by Invitation

Interested in participating?

Request Info

Key information

Conditions

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

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Mg0015 40144, Milan, Italy

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Zilucoplan

Drug

Zilucoplan will be administered subcutaneously to pediatric study participants

Other names: RA101495

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

Sponsors and collaborators

Lead sponsor

UCB Biopharma SRL

Industry

Registry information

Official study title

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

Acronym: ziMyG+

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
May 30, 2024
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.