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RecruitingInterventionalPhase 2/Phase 3

A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

NCT ID: NCT06055959Sponsor: UCB Biopharma SRLLast updated: 2026-05-08

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).

Arms & interventions

  • DrugZilucoplan

    Zilucoplan will be administered subcutaneously to pediatric study participants.

Outcome measures

Primary

  • Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)

    Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.

    Time frame: Week 4 (Day 29)

  • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)

    Samples for measurement of sRBC lysis will be collected on Day 29 predose.

    Time frame: Week 4 (Day 29)

  • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)

    Samples for measurement of C5 will be collected on Day 29 predose.

    Time frame: Week 4 (Day 29)

Secondary

  • Occurence of treatment-emergent adverse events (TEAEs) during the course of the study

    Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

  • Occurrence of treatment-emergent serious adverse events (TESAEs)

    Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

  • Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)

    Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

  • Occurrence of treatment-emergent infections

    Time frame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

  • Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)

    Time frame: Week 4 (Day 29)

  • Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).

    Time frame: Week 4 (Day 29)

  • Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)

    Time frame: Week 4 (Day 29)

  • Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)

    Time frame: Week 4 (Day 29)

  • Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)

    Time frame: Week 4 (Day 29)

Eligibility criteria

Sex: AllAge: 12 Years to 17 YearsHealthy volunteers: No
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 locations (2)

Mg0014 50168

Chicago, Illinois, 60611

Withdrawn

Mg0014 50574

Denton, Texas, 76208

Withdrawn