Clinical Trial

Long-term Extension of GTX-102 in Angelman Syndrome

Name: Long-term Extension of GTX-102 in Angelman Syndrome — clinical trial record (NCT06415344)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Enrolling by Invitation Phase 3

View on ClinicalTrials.gov →

Summary

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

Protocol Amendment History 1 change

critical Primary endpoint(s) modified 2026-07-28

Trial Details

NCT Number	NCT06415344
Lead Sponsor	Ultragenyx Pharmaceutical Inc
Conditions	Angelman Syndrome
Enrollment	255 participants
Start Date	2024-07-31
Primary Completion	2029-02 (estimated)
Study Completion	2029-02 (estimated)
Updated on ClinicalTrials.gov	2026-07-27