Trials / Enrolling By Invitation
Enrolling By InvitationNCT06415344
Long-term Extension of GTX-102 in Angelman Syndrome
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
- Status
- Enrolling By Invitation
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 255 (estimated)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Detailed description
This study is a phase 3, long term extension (LTE) which is open to participants rolling over from a prior GTX-102 clinical study. The LTE study will evaluate the long-term safety and efficacy of GTX-102 in participants with AS.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | GTX-102 | Antisense Oligonucleotide |
Timeline
- Start date
- 2024-07-31
- Primary completion
- 2029-02-01
- Completion
- 2029-02-01
- First posted
- 2024-05-16
- Last updated
- 2026-01-07
Locations
23 sites across 8 countries: United States, Australia, Canada, France, Germany, Israel, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06415344. Inclusion in this directory is not an endorsement.