Ignite Creation Date:
2024-05-19 @ 5:34 PM
Last Modification Date:
2024-10-26 @ 3:29 PM
Study NCT ID:
NCT06415344
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-05-16
First Post:
2024-05-10
Brief Title:
Long-term Extension of GTX-102 in Angelman Syndrome
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Ultragenyx Pharmaceutical Inc
Study Overview
Official Title:
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 is a Phase 3 long term extension LTE study to evaluate the safety and efficacy of GTX-102 in participants with AS
Participants may remain in the study until GTX-102 is approved andor becomes available in their geographical region or the sponsor stops the study
Participants may remain in the study until GTX-102 is approved andor becomes available in their geographical region or the sponsor stops the study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2024-510917-14-00 | CTIS | None | None |