Status:

RECRUITING

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

Lead Sponsor:

UCB Biopharma SRL

Conditions:

Generalized Myasthenia Gravis

Eligibility:

All Genders

12-17 years

Phase:

PHASE2

PHASE3

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 my...

Eligibility Criteria

Inclusion

  • United States of America (USA) specific inclusion criterion:
  • \- Participant must be 12 to \<18 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 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation
  • Global inclusion criteria:
  • 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)

Exclusion

  • 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

Key Trial Info

Start Date :

October 16 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 25 2026

Estimated Enrollment :

8 Patients enrolled

Trial Details

Trial ID

NCT06055959

Start Date

October 16 2024

End Date

December 25 2026

Last Update

January 6 2026

Active Locations (9)

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Page 1 of 3 (9 locations)

1

Mg0014 50168

Chicago, Illinois, United States, 60611

2

Mg0014 50574

Denton, Texas, United States, 76208

3

Mg0014 40144

Milan, Italy

4

Mg0014 40774

Katowice, Poland