PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Phase 3
Recruiting
- Conditions
- Generalized Myasthenia GravisgMG
- Interventions
- Combination Product: Gefurulimab
- Registration Number
- NCT06607627
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 12
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Gefurulimab Gefurulimab On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.
- Primary Outcome Measures
Name Time Method Maximum Observed Serum Concentration (Cmax) of Gefurulimab Day 1 predose through Week 18 predose Serum Free Complement Component 5 (C5) Concentration Day 1 predose through Week 18 predose
- Secondary Outcome Measures
Name Time Method Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) Day 1 through Week 134 Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18 Baseline through Week 18 Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18 Baseline through Week 18 Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18 Baseline through Week 18 Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18 Baseline through Week 18 Number of Participants With Anti-drug Antibodies (ADAs) Baseline through Week 134 Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses Baseline through Week 134 Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status Baseline through Week 134
Trial Locations
- Locations (1)
Research Site
🇨🇳Taipei, Taiwan