Clinical Trial

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

Study acronym: Aurora
Recruiting Phase 2
View on ClinicalTrials.gov →
Summary
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
Protocol Amendment History 16 amendments
This ClinicalTrials.gov record has been amended 16 times since 2025-08-28; most recent amendment 2026-07-24.
Status change: Not Yet Recruiting → Recruiting 2025-09-26
Trial Details
NCT Number NCT07157254
Lead Sponsor Ultragenyx Pharmaceutical Inc
Conditions Angelman Syndrome
Enrollment 60 participants
Start Date 2025-10-13
Primary Completion 2030-01 (estimated)
Study Completion 2030-01 (estimated)
Updated on ClinicalTrials.gov 2026-08-07