LTE GTX-102 in AS
Research type
Research Study
Full title
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome
IRAS ID
1009975
Contact name
Vanessa Horne
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Clinicaltrials.gov Identifier
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 open-label, long-term extension (LTE) study is to evaluate the long-term safety and efficacy of the investigational medicine, called GTX-102, in patients with AS. This LTE study will also explore how well GTX-102 works based on the outcome of various functional assessments (assessments and questionnaires aimed at evaluating cognitive function, development, and behaviours) in patients with AS.
This is an open-label study meaning all participants will receive GTX-102. Patients will receive treatment with GTX-102 via intrathecal (IT) lumbar puncture (LP) in accordance with the study protocol.
Ultragenyx Pharmaceutical Inc. is sponsoring this multicenter study, with approximately 75 patients in approximately 24 investigational study sites globally, planned to be rolled over from other GTX-102 studies into this LTE study.
Patients will be required to attend hospital visits whereby study assessments will be performed. GTX-102 is at a research stage, so it may cause side effects that are not known at this time. Patients will be monitored throughout the study; participation is voluntary and informed consent is mandatory.REC name
London - Riverside Research Ethics Committee
REC reference
24/LO/0422
Date of REC Opinion
3 Aug 2024
REC opinion
Further Information Favourable Opinion