Clinical trial · Interventional
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)
- 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 primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
Conditions
Conditions (2)
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 | — |
| gMG | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Gefurulimab | Combination Product | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Gefurulimab
- description
- On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.
- interventionNames
- Combination Product: Gefurulimab
Primary outcomes (2)
- measure
- Maximum Observed Serum Concentration (Cmax) of Gefurulimab
- timeFrame
- Day 1 predose through Week 18 predose
- measure
- Serum Free Complement Component 5 (C5) Concentration
- timeFrame
- Day 1 predose through Week 18 predose
Secondary outcomes (8)
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 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. * All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration. Rest of World (ROW) specific inclusion criteria: * Participant must be 6 to \< 18 years of age at the time of signing the informed consent/assent. * All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs \< 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available) Global inclusion criteria: * Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV * Positive serological test for autoantibodies against AChR Exclusion Criteria: * History of thymectomy, or any other thymic surgery within 12 months prior to Screening * Untreated thymic malignancy, carcinoma, or thymoma * History of Neisseria meningitidis infection * Pregnancy, breastfeeding, or intention to conceive during the course of the study
References
Publications (0)
Data not yet available