Long-term Extension of GTX-102 in Angelman Syndrome
Terminated · Phase 3 · NCT06415344
Conditions: Angelman Syndrome
At a glance
- Research payments reported, 2025
- $1.5M
- U.S. investigators named
- 7
- Sites paid
- 9
- Planned or actual enrollment
- 66
About the trial
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Tested
- GTX-102 (drug)
- Dates
- Started 2024-07-31; completion 2026-10-30
- Results posted
- Not yet
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
From ClinicalTrials.gov, last updated 2026-11-03. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ultragenyx Pharmaceutical Inc. reported $1.5M in research payments for this study in 2025 (86 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Lynne M. Bird, M.D. Medical Genetics · San Diego, CA
- Rajsekar R. Rajaraman, M.D. Child Neurology · Los Angeles, CA
- Scott M. Batchelor, M.D. Pediatrics · Atlanta, GA
- Han C. Phan, MD Child Neurology · Atlanta, GA
- Elizabeth Berry-kravis, M.D. Child Neurology · Chicago, IL
- Neha S. Bhatia, M.D. Medical Genetics · Boston, MA
- Srishti Nangia, M.D. Child Neurology · New York, NY
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Rush University Medical Center, IL $552,481
- Boston Children's Hospital, MA $402,152
- Rare Disease Research, LLC $147,395
- Ronald Reagan UCLA Medical Center, CA $135,173
- Weill Medical College of Cornell University $133,558
- Regents of the University of California $61,255
- UCLA Dept of Pediatric Grp Practice $56,516
- Seattle Children's Hospital, WA $16,996
- The Children's Mercy Hospital, MO $5,632
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.