Trials / Active Not Recruiting
Active Not RecruitingNCT06617429
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 129 (actual)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 4 Years – 17 Years
- Healthy volunteers
- Not accepted
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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | GTX-102 | antisense oligonucleotide |
| PROCEDURE | Sham-LP | Small needle prick on the lower back at the location where the LP injection is normally made |
Timeline
- Start date
- 2024-12-03
- Primary completion
- 2026-07-01
- Completion
- 2027-11-01
- First posted
- 2024-09-27
- Last updated
- 2026-02-04
Locations
28 sites across 6 countries: United States, Canada, Germany, Japan, Poland, Spain
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06617429. Inclusion in this directory is not an endorsement.