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NCT04259281 · ClinicalTrials.gov registry record · Phase 1
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
A Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 74
- Enrollment target
NCT04259281: Completed Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
NCT04259281 is a Phase 1 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 74 participants, above the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (23% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04259281, a Phase 1 study, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 74 participants
- Enrollment target
Study Summary
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).
Interventions
- DRUG GTX-102
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 74 participants |
| Start Date | 2020-02-24 |
| Est. Completion | 2025-01-08 |
| Phase | Phase 1 |
What the finished NCT04259281 record still lists
NCT04259281 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 74 participants, a relatively small participant target, above the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (23% higher).
The record links to 0 conditions, and to 1 intervention - of which GTX-102 is the first listed.
NCT04259281 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04259281 about?
NCT04259281 is a clinical study titled "A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome". 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).
What is the current status of trial NCT04259281?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 74 participants. The study started on 2020-02-24. Estimated completion is 2025-01-08.
What interventions are being tested in trial NCT04259281?
The interventions under investigation include: GTX-102 (DRUG).
Who is sponsoring clinical trial NCT04259281?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04259281's enrollment target sits among peer trials
74 725th of 2000 higher than 1,269 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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
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