A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Public ClinicalTrials.gov record NCT07157254. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official 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
Study identification
- NCT ID
- NCT07157254
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Enrollment
- 60 participants
Conditions and interventions
Conditions
Interventions
- No intervention Other
- GTX-102 Drug
Other · Drug
Eligibility (public fields only)
- Age range
- 1 Year to 64 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 12, 2025
- Primary completion
- Dec 31, 2029
- Completion
- Dec 31, 2029
- Last update posted
- Jun 22, 2026
2025 – 2030
United States locations
- U.S. sites
- 8
- U.S. states
- 7
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Cedars Sinai Medical Center | Los Angeles | California | 90048 | Recruiting |
| Rush University Medical Center | Chicago | Illinois | 60612 | Recruiting |
| Clinical Trial Site | Baltimore | Maryland | 21205 | Not yet recruiting |
| Clinical Trial Site | Kansas City | Missouri | 64108 | Not yet recruiting |
| Rare Disease Research | Hillsborough | North Carolina | 27278 | Recruiting |
| Akron Children's Hospital | Akron | Ohio | 44308 | Recruiting |
| UT Health Austin | Austin | Texas | 78723 | Recruiting |
| Carum Research Inc. | Dallas | Texas | 75243 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 13 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07157254, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 22, 2026 · Synced Jul 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07157254 live on ClinicalTrials.gov.