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Clinical trial

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

NCT07157254 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT07157254
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 2
Overall status
Active, not recruiting
Start date
October 13, 2025
Completion date
January 31, 2030
Enrollment
60 (estimated)
Sites
21

Conditions

As the registry lists them; a condition with its own page is linked.

angelman syndrome