Ignite Creation Date:
2025-12-24 @ 5:56 PM
Ignite Modification Date:
2026-02-25 @ 6:53 PM
Study NCT ID:
NCT02201368
Status:
WITHDRAWN
Last Update Posted:
2014-07-28
First Post:
2013-03-22
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects
Sponsor:
Maria Luz Couce Pico
Organization:
Study Overview
Official Title:
Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects
Status:
WITHDRAWN
Status Verified Date:
2014-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
IMP management difficulties
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to determine if administration Triheptanoin is an effective treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults. Period of treatment and follow-up will be 16 months.
Detailed Description:
Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could cause not only a great improvement in their quality of life, also could prevent life-threatening signs, reducing symptoms and serious complications of their disease, like cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a gluconeogenic effect.
This treatment opens the door to be used in other diseases such as pyruvate carboxylase deficiency, glycogen storage disease and other diseases with energy problems.
All patients will be followed up until 16 months.
This treatment opens the door to be used in other diseases such as pyruvate carboxylase deficiency, glycogen storage disease and other diseases with energy problems.
All patients will be followed up until 16 months.
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 |
|---|---|---|---|---|
| 2007-005578-29 | EUDRACT_NUMBER | None | View |