Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Angelman Syndrome
In brief
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).
Key facts
- Study ID
- NCT06617429
- Run by
- Ultragenyx Pharmaceutical Inc
- People needed
- 129
- Starts
- 2024-12-03
- Expected to finish
- 2027-11-01
- Last updated by the study team
- 2026-08-06
Who can join
Age: 4 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Cedars Sinai — Los Angeles, California, United States
- UCSD, Rady Children's Hospital — San Diego, California, United States
- UCSF — San Francisco, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Nicklaus Children's Hospital — Miami, Florida, United States
- Rare Disease Research — Atlanta, Georgia, United States
- Rush University — Chicago, Illinois, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Children's Mercy — Kansas City, Missouri, United States
- Columbia University Medical Center — New York, New York, United States
- UNC Chapel Hill Pediatrics — Chapel Hill, North Carolina, United States
- Rare Disease Research — Hillsborough, North Carolina, United States
- The University of Texas — Austin, Texas, United States
- Carum Research Inc — Dallas, Texas, United States
- McGill University Health Centre — Montreal, Quebec, Canada
- British Columbia Children's Hospital — Vancouver, Canada
- Universitaetsklinikum Hamburg-Eppendorf — Hamburg, Germany
- University of Leipzig — Leipzig, Germany
- Haunersche Kinderklinik — Munich, Germany
- Nagoya City University Graduate School of Medical Sciences — Nagoya, Aichi-ken, Japan
- Osaka City General Hospital — Osaka, Japan
- Hokkaido University Hospital — Sapporo, Japan
- Medical University of Gdańsk — Gdansk, Poland
- Polish Mothers Memorial Institute — Lodz, Poland
- Hospital Universitario Parc Tauli — Barcelona, Spain
Full record on ClinicalTrials.gov
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