Ignite Creation Date:
2025-12-24 @ 12:54 PM
Ignite Modification Date:
2025-12-27 @ 9:33 PM
Study NCT ID:
NCT02599961
Status:
TERMINATED
Last Update Posted:
2020-06-11
First Post:
2015-11-03
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Study Overview
Official Title:
An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects With Glucose Transporter Type 1 Deficiency Syndrome
Status:
TERMINATED
Status Verified Date:
2020-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Study was halted prematurely due to lack of efficacy
Has Expanded Access:
True
If Expanded Access, NCT#:
NCT03773770
Has Expanded Access, NCT# Status:
AVAILABLE
Acronym:
None
Brief Summary:
The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.
Detailed Description:
The study will enroll up to 40 pediatric, adolescent and adult Glut 1 DS participants who have completed the UX007G-CL201 (NCT019933186) study and, at the discretion of the Sponsor, additional participants from other clinical studies, investigator sponsored trials (ISTs), or expanded access/compassionate use treatment.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2015-000389-69 | EUDRACT_NUMBER | None | View |