GTX-102
Sponsors
Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc
Conditions
Angelman Syndrome
Phase 1
Phase 2
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Active, not recruitingNCT07157254
Start: 2025-10-13End: 2030-01-01Target: 60Updated: 2026-09-09
A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects with Deletion- or Nondeletion-type Angelman Syndrome
Not yet recruitingCTIS2024-519393-39-00
Target: 19Updated: 2025-12-19
Phase 3
Long-term Extension of GTX-102 in Angelman Syndrome
Active, not recruitingNCT06415344
Start: 2024-07-31End: 2029-02-01Target: 255Updated: 2026-09-09
A Long-Term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome
RecruitingCTIS2024-510917-14-00
Start: 2024-09-03Target: 38Updated: 2025-11-14
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Active, not recruitingNCT06617429
Start: 2024-12-03End: 2027-11-01Updated: 2026-08-06
A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects with Angelman Syndrome
RecruitingCTIS2024-512600-19-00
Start: 2025-04-14Target: 40Updated: 2025-09-19