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Clinical Trial ● Currently Recruiting Phase II NCT07157254

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

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) — Recruiting • Phase II • NCT07157254.

📅 04 Jun 2026 ⏱ 1 min read
Currently Recruiting
This trial is actively seeking participants in the UK. Discuss eligibility with your patient before referring.
Status
Currently Recruiting
Phase
Phase II
NCT ID
NCT07157254
Start
2025-10-13
Completion
2030-01
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.

Verify eligibility, endpoints and current status on the original ClinicalTrials.gov registry before acting on this summary.

Use This Page For
  • Quick orientation before opening the registry record.
  • Checking recruitment status, phase and sponsor at a glance.
  • Connecting this trial to nearby guidelines, Drug Science and education.
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

Full Trial Details
View this trial on the source registry
Eligibility criteria, protocol, and results when available
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