A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Completed · Phase 1, Phase 2 · NCT04259281

Conditions: Angelman Syndrome

Research payments reported, 2025: $234,341
U.S. investigators named: 6
Sites paid: 8
Planned or actual enrollment: 74

About the trial

Sponsor: Ultragenyx Pharmaceutical Inc
Tested: GTX-102 (drug)
Dates: Started 2020-02-24; completion 2025-01-08
Results posted: Not yet

The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).

From ClinicalTrials.gov, last updated 2026-01-09. Full record, eligibility and contacts (opens in a new tab)

Industry funding

Ultragenyx Pharmaceutical Inc. reported $234,341 in research payments for this study in 2025 (41 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

Clinicians the company named as principal investigators in its Open Payments reports.

Sites paid to run it

Rush University Medical Center, IL $60,340
Boston Children's Hospital, MA $58,437
Rare Disease Research, LLC $38,508
UCLA Dept of Pediatric Grp Practice $31,398
Ronald Reagan UCLA Medical Center, CA $19,878
The Regents of the University of California (san Diego Campus) $11,441
Weill Medical College of Cornell University $9,840
Regents of the University of California $4,499

Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.

At a glance

About the trial

Industry funding

U.S. investigators named on this study

Sites paid to run it