A phase 1/2 study of GTX-102 in paediatric patients with AS
Research type
Research Study
Full title
A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)
IRAS ID
299037
Contact name
Laurent Servais
Contact email
Sponsor organisation
GeneTx Biotherapeutics, LLC
Eudract number
2021-001793-36
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Angelman Syndrome (AS) is a genetic condition that affects the nervous system and causes severe physical and learning disabilities. There are currently no approved therapies specifically indicated for the treatment of AS.
The purpose of this study is to determine the safety and tolerability of an investigational medicine, called GTX-102, in paediatric patients with AS. The study will also assess how well GTX-102 is processed in the participants body and its effectiveness.
The study consists of two phases: the Dose Escalation Phase and the Maintenance Phase. The Dose Escalation Phase is the initial part of the study where participants will receive GTX-102 monthly.
Participants will be enrolled into 1 of 2 cohorts: Cohort 4 or Cohort 5, and will receive 3-4 doses of GTX-102. After completion of the Dose Escalation Phase, participants will continue into the Maintenance Phase and will receive GTX-102 approximately every 3 months.
This is an open-label study meaning all participants will receive GTX-102.
GeneTx Biotherapeutics, LLC is sponsoring this multicenter study, with approximately 8 to 12 study sites in the United States, United Kingdom, and Canada.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
21/SC/0168
Date of REC Opinion
24 May 2021
REC opinion
Favourable Opinion