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NCT07157254 · ClinicalTrials.gov registry record · Phase 2
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
A Phase 2 study of Angelman Syndrome, sponsored by Ultragenyx Pharmaceutical.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 60
- Enrollment target
- 20
- Study locations
NCT07157254: Recruiting Phase 2 study of Angelman Syndrome, sponsored by Ultragenyx Pharmaceutical.
NCT07157254 is a Phase 2 study of Angelman Syndrome that is actively recruiting participants, run by Ultragenyx Pharmaceutical. The registered enrollment target is 60 participants, below the 121-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (50% lower). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07157254, a Phase 2 study of Angelman Syndrome, is actively recruiting participants, sponsored by Ultragenyx Pharmaceutical.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 60 participants
- Enrollment target
- 20
- Study locations
Study Summary
The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
Conditions Studied
Interventions
- OTHER No intervention
- DRUG GTX-102
Study Locations (20)
Other
- Clinical Trial Site - Marseille
- Clinical Trial Site - Paris
- Clinical Trial Site - Ramat Gan
- Azienda Ospedaliera Universitaria Meyer IRCCS - Florence
- Fondazione IRCCS Istituto Neurologico C. Besta - Milan
- Clinical Trial Site - Rome
- Hospital de Santa Maria - Lisbon
- Hospital Santa Joao - Porto
Texas
- UT Health Austin - Austin
- Carum Research Inc. - Dallas
California
- Clinical Trial Site - Los Angeles
Illinois
- Rush University Medical Center - Chicago
Maryland
- Clinical Trial Site - Baltimore
Missouri
- Clinical Trial Site - Kansas City
North Carolina
- Rare Disease Research - Hillsborough
Ohio
- Akron Children's Hospital - Akron
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 60 participants |
| Start Date | 2025-10-13 |
| Est. Completion | 2030-01 |
| Phase | Phase 2 |
What NCT07157254 shows while recruiting
NCT07157254 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 60 participants, a relatively small participant target, below the 121-participant average among 11 other Angelman Syndrome trials with a reported enrollment target (50% lower).
The record links to 1 condition, with Angelman Syndrome appearing as the primary indexed condition, and to 2 interventions - of which No intervention is the first listed.
NCT07157254 names 20 study sites across 12 states, led by Other, Texas, California.
Frequently Asked Questions
What is clinical trial NCT07157254 about?
NCT07157254 is a clinical study titled "A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)". The main goal of the study is to evaluate the safety and efficacy of GTX-102 in participants with Angelman syndrome.
What is the current status of trial NCT07157254?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 60 participants. The study started on 2025-10-13. Estimated completion is 2030-01.
What conditions does trial NCT07157254 study?
This clinical trial studies the following conditions: Angelman Syndrome.
What interventions are being tested in trial NCT07157254?
The interventions under investigation include: No intervention (OTHER), GTX-102 (DRUG).
Who is sponsoring clinical trial NCT07157254?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07157254 being conducted?
This trial has 20 study locations across California, Illinois, Maryland, Missouri, North Carolina. 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 NCT07157254's enrollment target sits among peer trials
60 10th of 11 higher than 2 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
-
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
COMPLETED · Phase 2
-
Angelman Syndrome Natural History Study
RECRUITING
-
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
RECRUITING · Phase 3
-
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
-
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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