ClinicaliQ Trial Snapshot
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) — Recruiting • Phase II • NCT07157254.
- GTX-102 injection being tested for safety and effectiveness in treating Angelman syndrome caused by different genetic mutations.
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What This Trial Is Studying
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome. Conditions: Angelman Syndrome Interventions: No intervention, GTX-102 Lead Sponsor: Ultragenyx Pharmaceutical Inc Planned Enrollment: 60 participants