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NCT06617429 · ClinicalTrials.gov registry record · Phase 3
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
A Phase 3 study of Angelman Syndrome, sponsored by Ultragenyx Pharmaceutical.
- Active
- Registry status
- Phase 3
- Development phase
- 129
- Enrollment target
- 20
- Study locations
NCT06617429 is a Phase 3 study of Angelman Syndrome that is active but no longer recruiting, run by Ultragenyx Pharmaceutical. The registered enrollment target is 129 participants, above the 115-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (12% higher). The trial reports 20 study locations across 13 states.
The verdict
NCT06617429, a Phase 3 study of Angelman Syndrome, is active but no longer recruiting, sponsored by Ultragenyx Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 129 participants
- Enrollment target
- 20
- Study locations
Study Summary
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).
Conditions Studied
Interventions
- DRUG GTX-102
- PROCEDURE Sham-LP
Study Locations (20)
Other
- British Columbia Children's Hospital - Vancouver
- Universitaetsklinikum Hamburg-Eppendorf - Hamburg
- University of Leipzig - Leipzig
- Haunersche Kinderklinik - Munich
California
- Cedars Sinai - Los Angeles
- UCSD, Rady Children's Hospital - San Diego
- UCSF - San Francisco
North Carolina
- UNC Chapel Hill Pediatrics - Chapel Hill
- Rare Disease Research - Hillsborough
Texas
- The University of Texas - Austin
- Carum Research Inc - Dallas
Colorado
- Children's Hospital Colorado - Aurora
Florida
- Nicklaus Children's Hospital - Miami
Georgia
- Rare Disease Research - Atlanta
Illinois
- Rush University - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 129 participants |
| Start Date | 2024-12-03 |
| Est. Completion | 2027-11 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06617429
The ClinicalTrials.gov registry entry for NCT06617429 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 129 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 115-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (12% higher). The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Angelman Syndrome appearing as the primary indexed condition, and to 2 interventions - of which GTX-102 is the first listed.
NCT06617429 reports 20 study locations spanning 13 distinct geographic areas - top geographies include Other, California, North Carolina.
Frequently Asked Questions
What is clinical trial NCT06617429 about?
NCT06617429 is a clinical study titled "Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)". 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).
What is the current status of trial NCT06617429?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 129 participants. The study started on 2024-12-03. Estimated completion is 2027-11.
What conditions does trial NCT06617429 study?
This clinical trial studies the following conditions: Angelman Syndrome.
What interventions are being tested in trial NCT06617429?
The interventions under investigation include: GTX-102 (DRUG), Sham-LP (PROCEDURE).
Who is sponsoring clinical trial NCT06617429?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06617429 being conducted?
This trial has 20 study locations across California, Colorado, Florida, Georgia, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.