Ignite Creation Date:
2025-12-25 @ 2:10 AM
Ignite Modification Date:
2026-02-25 @ 10:04 PM
Study NCT ID:
NCT02214160
Status:
COMPLETED
Last Update Posted:
2023-08-01
First Post:
2014-08-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
Sponsor:
Ultragenyx Pharmaceutical Inc
Organization:
Study Overview
Official Title:
An Open-label Long-Term Safety and Efficacy Extension Study in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies
Status:
COMPLETED
Status Verified Date:
2023-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
True
If Expanded Access, NCT#:
NCT03773770
Has Expanded Access, NCT# Status:
AVAILABLE
Acronym:
None
Brief Summary:
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events associated with LC-FAOD.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2016-000322-19 | EUDRACT_NUMBER | None | View |