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NCT06607627 | RECRUITING | Generalized Myasthenia Gravis

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
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.

Condition or disease

Generalized Myasthenia Gravis

GMG

Intervention/treatment

Gefurulimab

Phase

PHASE3

Study Type : INTERVENTIONAL
Estimated Enrollment : 12 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : 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+)
Actual Study Start Date : 2024-12-23
Estimated Primary Completion Date : 2027-04-16
Estimated Study Completion Date : 2029-07-18

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 6 Years to 17 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis

Location Details

NCT06607627

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, District of Columbia

Research Site

Washington, District of Columbia, United States, 20010

RECRUITING

United States, Virginia

Research Site

Norfolk, Virginia, United States, 23507

RECRUITING

Brazil,

Research Site

Joinville, Brazil, 89202-451

NOT YET RECRUITING

Brazil,

Research Site

Salvador, Brazil, 41253-190

NOT YET RECRUITING

Brazil,

Research Site

Sao jose do rio preto, Brazil, 15090-000

RECRUITING

Brazil,

Research Site

São Paulo, Brazil, 05403-000

NOT YET RECRUITING

Brazil,

Research Site

São Paulo, Brazil, 0438-002

NOT YET RECRUITING

Poland,

Research Site

Bydgoszcz, Poland, 85-086

NOT YET RECRUITING

Poland,

Research Site

Warsaw, Poland, 02-097

NOT YET RECRUITING

Poland,

Research Site

Łódź, Poland, 93-338

NOT YET RECRUITING

Taiwan,

Research Site

Taipei, Taiwan, 11101

RECRUITING

Taiwan,

Research Site

Taipei, Taiwan, 23561