RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

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+)

Quick Facts

Study Start:2024-11-13
Study Completion:2029-10-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06607627

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.

Ages Eligible for Study:6 Years to 17 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Participant must be 6 to \< 18 years of age at the time of signing the informed consent/assent.
  2. * Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
  3. * Positive serological test for autoantibodies against AChR
  4. * 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)
  1. * History of thymectomy, or any other thymic surgery within 12 months prior to Screening
  2. * Untreated thymic malignancy, carcinoma, or thymoma
  3. * History of Neisseria meningitidis infection
  4. * Pregnancy, breastfeeding, or intention to conceive during the course of the study

Contacts and Locations

Study Contact

Alexion Pharmaceuticals, Inc. (Sponsor)
CONTACT
1-855-752-2356
clinicaltrials@alexion.com

Study Locations (Sites)

Research Site
Washington, District of Columbia, 20010
United States
Research Site
Norfolk, Virginia, 23507
United States

Collaborators and Investigators

Sponsor: Alexion Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-11-13
Study Completion Date2029-10-01

Study Record Updates

Study Start Date2024-11-13
Study Completion Date2029-10-01

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Generalized Myasthenia Gravis
  • gMG