RECRUITINGPhase 3INTERVENTIONAL
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+)
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
United States of America (USA) specific inclusion criterion:
- Participant must be 12 to \< 18 years of age at the time of signing the willing to sign a consent form/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 Who May Qualify:
- Participant must be 6 to \< 18 years of age at the time of signing the willing to sign a consent form/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 Who May Qualify:
- 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
Who Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
COMBINATION_PRODUCT
Gefurulimab
Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
Locations (13)
Research Site
Washington D.C., District of Columbia, United States
Research Site
Norfolk, Virginia, United States
Research Site
Joinville, Brazil
Research Site
Salvador, Brazil
Research Site
São José do Rio Preto, Brazil
Research Site
São Paulo, Brazil
Research Site
São Paulo, Brazil
Research Site
Saitama-Shi, Japan
Research Site
Bydgoszcz, Poland
Research Site
Lodz, Poland
Research Site
Warsaw, Poland
Research Site
New Taipei City, Taiwan
Research Site
Taipei, Taiwan