A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
Public ClinicalTrials.gov record NCT04259281. 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 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective of the study is to evaluate the safety and tolerability of multiple-ascending doses of GTX-102 administered by intrathecal (IT) injection to participants with Angelman Syndrome (AS).
Study identification
- NCT ID
- NCT04259281
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 74 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
- Feb 23, 2020
- Primary completion
- Jan 7, 2025
- Completion
- Jan 7, 2025
- Last update posted
- Jan 8, 2026
2020 – 2025
United States locations
- U.S. sites
- 6
- U.S. states
- 5
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA Medical Center | Los Angeles | California | 90095 | — |
| Rady Children's Hospital | San Diego | California | 92123 | — |
| Rare Disease Research | Atlanta | Georgia | 30318 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Weill Cornell Medicine | New York | New York | 10065 | — |
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 19 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04259281, 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 Jan 8, 2026 · Synced Sep 27, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04259281 live on ClinicalTrials.gov.