Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Terminated · Phase 3 · NCT06617429
Conditions: Angelman Syndrome
At a glance
- Research payments reported, 2025
- $6.4M
- U.S. investigators named
- 16
- Sites paid
- 15
- Planned or actual enrollment
- 129
About the trial
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Tested
- GTX-102 (drug), Sham-LP (procedure)
- Dates
- Started 2024-12-03; completion 2026-10-30
- Results posted
- Not yet
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).
From ClinicalTrials.gov, last updated 2026-11-03. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ultragenyx Pharmaceutical Inc. reported $6.4M in research payments for this study in 2025 (133 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
- Cesar A. Ochoa-lubinoff, M.D. Pediatrics · Los Angeles, CA
- Elliott H. Sherr, M.D. Child Neurology · San Francisco, CA
- Diana J. Walleigh, MD Neurology · Aurora, CO
- Paula P. Schleifer, MD Child Neurology · Miami, FL
- 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
- Jean-baptiste Le Pichon, M.D., PH.D. Child Neurology · Topeka, KS
- Neha S. Bhatia, M.D. Medical Genetics · Boston, MA
- Kristina K. Julich, MD Neurology · Boston, MA
- Bridget L. Ostrem, M.D., PH.D. Neurology · Boston, MA
- Elizabeth R. Jalazo, M.D. Pediatrics · Chapel Hill, NC
- Edward C. Smith, MD Pediatrics · Durham, NC
- Jennifer M. Bain, M.D., PH.D. Child Neurology · New York, NY
- Andrea R. Lowden, MD Child Neurology · Irving, TX
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Rare Disease Research, LLC $828,919
- Rush University Medical Center, IL $633,473
- The Children's Mercy Hospital, MO $583,065
- Cedars-Sinai Medical Center, CA $579,166
- The University of Texas at Austin $573,703
- The Trustees of Columbia University $526,866
- Carum Research Inc. $521,526
- Boston Children's Hospital, MA $469,024
- Regents of the University of California $327,160
- UCSF Langley Porter Psychiatric Hospital and Clinics, CA $316,915
- Nicklaus Children's Hospital, FL $264,280
- The University of North Carolina at Chapel Hill $262,727
- Children's Hospital Colorado, CO $216,666
- Children's Hospital of Philadelphia, PA $184,457
- University of Michigan Health System, MI $79,823
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.