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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 is a Phase 1 study that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 74 participants.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04259281
The ClinicalTrials.gov registry entry for NCT04259281 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 74 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which GTX-102 is the first listed.
NCT04259281 reports 0 study locations.
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.
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