Clinical Trial

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

Study acronym: Aspire
Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).
Protocol Amendment History 17 amendments
This ClinicalTrials.gov record has been amended 17 times since 2024-09-24; most recent amendment 2026-05-04.
Status change: Recruiting → Active, Not Recruiting 2025-07-31
Status change: Not Yet Recruiting → Recruiting 2024-12-18
Trial Details
NCT Number NCT06617429
Lead Sponsor Ultragenyx Pharmaceutical Inc
Conditions Angelman Syndrome
Enrollment 129 participants
Start Date 2024-12-03
Primary Completion 2026-07-10 (estimated)
Study Completion 2027-11 (estimated)
Updated on ClinicalTrials.gov 2026-08-06