Long-term Extension of GTX-102 in Angelman Syndrome
Public ClinicalTrials.gov record NCT06415344. 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 Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Study identification
- NCT ID
- NCT06415344
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 255 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- 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
- Jul 30, 2024
- Primary completion
- Jan 31, 2029
- Completion
- Jan 31, 2029
- Last update posted
- Sep 8, 2026
2024 – 2029
United States locations
- U.S. sites
- 6
- U.S. states
- 5
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, Los Angeles (UCLA) | Los Angeles | California | 90095 | — |
| University of California, San Diego - Rady Children's Hospital | San Diego | California | 92123 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Weill Cornell Medicine | New York | New York | 10021 | — |
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 17 non-U.S. sites.
Recently updated Angelman Syndrome trials
Other public records listing Angelman Syndrome. Sorted by last update posted; newest first. Not a recommendation.
- NCT07605429: BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. Recruiting
- NCT07157254: A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) Recruiting
- NCT07181837: A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome Recruiting
- NCT06139172: Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) Active, not recruiting
- NCT03655223: Early Check: Expanded Screening in Newborns Active, not recruiting
- NCT07417137: A Natural History Study of Angelman Syndrome Recruiting
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06415344, 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 Sep 8, 2026 · Synced Sep 11, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06415344 live on ClinicalTrials.gov.