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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: Active Phase 3 study of Angelman Syndrome, sponsored by Ultragenyx Pharmaceutical.
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. According to ClinicalTrials.gov, the official US trial registry.
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 |
What the registry record for NCT06617429 still lists
NCT06617429 is an interventional study that assigns participants to a tested intervention. The registered 129 participants enrollment target is mid-sized for trials with a published cap, above the 115-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (12% higher).
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 names 20 study sites across 13 states, led by 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
Similar trials for Angelman Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06617429's enrollment target sits among peer trials
129 4th of 11 higher than 8 of 11 other Angelman Syndrome trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Angelman Syndrome trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
RECRUITING · Phase 3
-
Angelman Syndrome Natural History Study
RECRUITING
-
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
RECRUITING · Phase 1
-
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
RECRUITING · NA
-
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
RECRUITING · Phase 2
-
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
RECRUITING · Phase 1
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