Clinical trial · Interventional
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
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
NCT06055959CI-TRIAL-00120200ziMyGrecruitingPhase 2 / Phase 3ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Generalized Myasthenia Gravis | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Zilucoplan | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Zilucoplan Arm
- description
- Study participants will receive zilucoplan in pre-defined dose based on their weight.
- interventionNames
- Drug: Zilucoplan
Primary outcomes (3)
- measure
- Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
- timeFrame
- Week 4 (Day 29)
- description
- Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
- measure
- Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
- timeFrame
- Week 4 (Day 29)
- description
- Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 12 Years
- Maximum age
- 17 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available
No reference posted for this study.