Recruiting
Phase 2
Phase 3

Zilucoplan

Sponsor:

UCB Biopharma SRL

Code:

NCT06055959

Conditions

Generalized Myasthenia Gravis

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

Interventions

Zilucoplan

Study Details

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 myasthenia gravis (gMG).

Conditions

Generalized Myasthenia Gravis

Study ID

NCT06055959

Start date

Oct 16, 2024

Status verified date

Feb, 2026

Completion date

Dec 25, 2026

Anticipated

Primary completion date

Nov 16, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

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 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 Criteria:

  • 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

Study Design

Enrollment

8 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Zilucoplan Arm

Study participants will receive zilucoplan in pre-defined dose based on their weight.

Interventions

Zilucoplan

Zilucoplan will be administered subcutaneously to pediatric study participants.

Primary outcome measure

  • Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) [ Time Frame: Week 4 (Day 29) ]
  • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) [ Time Frame: Week 4 (Day 29) ]
  • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29) [ Time Frame: Week 4 (Day 29) ]

Central Contacts and Locations

Locations

Mg0014 50168

Recruiting

Chicago, Illinois, United States, 60611

More Information

Sponsor

UCB Biopharma SRL

Last update posted

Feb 13, 2026

Last verified

Feb, 2026

Keywords

  • RA101495
  • gMG
  • generalized myasthenia gravis
  • zilucoplan
  • pediatric
  • MG0014

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by UCB Biopharma SRL on 2026-02-13.