Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Public ClinicalTrials.gov record NCT06617429. 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 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective of this study is to evaluate the effect of GTX-102 in cognitive function in participants with deletion-type Angelman Syndrome (AS).
Study identification
- NCT ID
- NCT06617429
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 129 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 17 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
- Dec 3, 2024 (Actual)
- Primary completion
- Jul 10, 2026 (Actual)
- Completion
- Nov 2027 (Estimated)
- Last update posted
- Aug 6, 2026
2024 – 2027
United States locations
- U.S. sites
- 14
- U.S. states
- 10
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Cedars Sinai | Los Angeles | California | 90048 | — |
| UCSD, Rady Children's Hospital | San Diego | California | 92123 | — |
| UCSF | San Francisco | California | 94143 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | — |
| Rare Disease Research | Atlanta | Georgia | 30329 | — |
| Rush University | Chicago | Illinois | 60612 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Children's Mercy | Kansas City | Missouri | 64108 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| UNC Chapel Hill Pediatrics | Chapel Hill | North Carolina | 27599 | — |
| Rare Disease Research | Hillsborough | North Carolina | 27278 | — |
| The University of Texas | Austin | Texas | 78723 | — |
| Carum Research Inc | Dallas | Texas | 75243 | — |
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 14 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06617429, 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 Aug 6, 2026 · Synced Oct 20, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT06617429 live on ClinicalTrials.gov.