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

Description

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

Generalized Myasthenia Gravis, gMG

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Study Overview

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Study Details

Study overview

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.

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+)

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

Condition
Generalized Myasthenia Gravis
Intervention / Treatment

-

Contacts and Locations

Washington

Research Site, Washington, District of Columbia, United States, 20010

Norfolk

Research Site, Norfolk, Virginia, United States, 23507

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Participant must be 6 to \< 18 years of age at the time of signing the informed consent/assent.
  • * 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
  • * 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)
  • * 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

Ages Eligible for Study

6 Years to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Alexion Pharmaceuticals, Inc.,

Study Record Dates

2029-10-01