Generalized Myasthenia Gravis, gMG
Conditions
Keywords
Acetylcholine Receptor antibodies, AChR+, Generalized Myasthenia Gravis, gMG, ALXN1720, anti-acetylcholine receptor antibody-positive, acetylcholine receptor, AChR, myasthenia gravis, MG, complement component 5, C5, VHH antibody, pediatric
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.
Interventions
COMBINATION_PRODUCTGefurulimab
Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 17 Years
Healthy 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. * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
Countries
Brazil, Japan, Poland, Taiwan, United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed