A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Last updated: March 12, 2025
Sponsor: UCB Biopharma SRL
Overall Status: Active - Recruiting

Phase

2/3

Condition

Neuropathy

Myasthenia Gravis Generalised

Sarcopenia

Treatment

Zilucoplan

Clinical Study ID

NCT06055959
MG0014
2022-502072-23
U1111-1290-3349
2022-502072-23-00
  • Ages 12-17
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

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 Informedconsent/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 Informedconsent/assent according to local regulation

Global inclusion criteria:

  • Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by aprior positive serologic test result to acetylcholine receptor (AChR) prior toScreening

  • Participant meets the criteria as defined by the Myasthenia Gravis Foundation ofAmerica (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 adolescentsfrom 12 years to <18 years of age at Screening

  • Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2years to <12 years of age at Screening (does not apply to US)

  • Documented vaccination against meningococcal infections within 3 years prior tostudy start. If not fully vaccinated, participants must receive appropriateprophylactic antibiotic treatment until at least 2 weeks after the initial dose ofvaccine(s)

Exclusion

Exclusion Criteria:

  • Participant has known positive serology for muscle-specific kinase

  • Participant has any medical or psychiatric condition that, in the opinion of theInvestigator, could jeopardize or would compromise the participant's ability toparticipate 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 judgementof the Investigator

  • Current or recent systemic infection within 2 weeks prior to Baseline or infectionrequiring intravenous antibiotics within 4 weeks prior to Baseline

Study Design

Total Participants: 8
Treatment Group(s): 1
Primary Treatment: Zilucoplan
Phase: 2/3
Study Start date:
October 16, 2024
Estimated Completion Date:
December 25, 2026

Connect with a study center

  • Mg0014 40144

    Milano,
    Italy

    Active - Recruiting

  • Mg0014 20104

    Seoul,
    Korea, Republic of

    Active - Recruiting

  • Mg0014 20220

    Seoul,
    Korea, Republic of

    Active - Recruiting

  • Mg0014 40774

    Katowice,
    Poland

    Active - Recruiting

  • Mg0014 40218

    Warszawa,
    Poland

    Active - Recruiting

  • Mg0014 40735

    Glasgow,
    United Kingdom

    Active - Recruiting

  • Mg0014 40736

    London,
    United Kingdom

    Active - Recruiting

  • Mg0014 50168

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Mg0014 50574

    Denton, Texas 76208
    United States

    Active - Recruiting

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