NCT07157254

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 1 Year – 64 Years
• Healthy volunteers: Not accepted

Summary

The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.

Detailed description

This basket study is designed to evaluate safety and efficacy of GTX-102 in participants with Angelman syndrome across genotypes and age groups. The study consists of subprotocols A, B, C and D. All subprotocols are open-label and follow the same design which includes a Screening, Loading and Maintenance period. Subprotocols A, B and C are single arm only. In subprotocol D, participants are randomized 2:1 to a GTX-102 group or a No Treatment group. The No Treatment group follows the same schedule of events as all other groups after completion of the No Treatment period. Participants from all the subprotocols have the option to continue treatment in a long-term extension study following their end of study visit.

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
OTHER	No intervention	During the no treatment period participants do not receive any study drug
DRUG	GTX-102	antisense oligonucleotide

Timeline

• Start date: 2025-10-13
• Primary completion: 2030-01-01
• Completion: 2030-01-01
• First posted: 2025-09-05
• Last updated: 2026-03-16

Locations

22 sites across 8 countries: United States, Argentina, Brazil, France, Israel, Italy, Portugal, United Kingdom

Regulatory

• FDA-regulated drug study