Drug Pipeline

GTX-102 Clinical Trials

Data updated daily from ClinicalTrials.gov — last refresh 2026-06-12
3
Active Studies
1
Recruiting
2
Phase 3
1
Top Sponsors
Active Studies by Phase
Phase 3
2
Phase 2
1
Active Trials
NCT IDTitleConditionStatus
Phase 3
NCT06415344 Long-term Extension of GTX-102 in Angelman Syndrome Angelman Syndrome Enrolling by Invitation
NCT06617429 Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome… Angelman Syndrome Active, Not Recruiting
Phase 2
NCT07157254 A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type… Angelman Syndrome Recruiting
Sponsors Running GTX-102 Trials
Ultragenyx Pharmaceutical Inc (3)
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