Ignite Creation Date:
2024-05-05 @ 4:49 PM
Last Modification Date:
2024-10-26 @ 9:25 AM
Study NCT ID:
NCT00328159
Status:
COMPLETED
Last Update Posted:
2011-02-17
First Post:
2006-05-18
Brief Title:
Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
Sponsor:
Assistance Publique - Hôpitaux de Paris
Organization:
Assistance Publique - Hôpitaux de Paris
Study Overview
Official Title:
Dietary Therapy of Mitochondrial Fatty Acids Oxidation A Clinical Study of Treatment With Odd Carbons Medium-chain Fatty Acids
Status:
COMPLETED
Status Verified Date:
2007-03
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:
Usual dietary therapies of mitochondrial fatty acid oxidation disorders FAO are based on 3 strategies
limitation of lipid intake in the diet
supplementation of the diet with medium-chain triglycerides MCT for patients affected with disorders of long-chain FAO
some specific supplementations for example L-carnitine
These strategies are often ineffective The aim of the present study is to evaluate new therapeutic ways based on the underlying energetic defect observed in these disorders The long-term goal is to develop efficient therapies of these disorders
limitation of lipid intake in the diet
supplementation of the diet with medium-chain triglycerides MCT for patients affected with disorders of long-chain FAO
some specific supplementations for example L-carnitine
These strategies are often ineffective The aim of the present study is to evaluate new therapeutic ways based on the underlying energetic defect observed in these disorders The long-term goal is to develop efficient therapies of these disorders
Detailed Description:
The main specific aim of this study will be to determine the efficiency of odd-chain MCT TRIHEPTANOIN Tri-C7 and its metabolites BETA-HYDROXYPENTANOATE BHP and BETA-KETOPENTANOATE BKP as potential treatments by orale or enteral routes These compounds are efficiently used for energy production despite long-chain FAO enzyme defects They use alternative metabolic pathways and have anaplerotic effects due to propionyl-CoA production by the thiolytic cleavage of odd carbon ketone bodies
The efficiency of these compounds will be compared with conventional diet MCT for each patient Because of frequent phenotypic variations observed for each of these diseases each patient will be his own control
The same protocol study will be followed in 2 centers Dallas USA main investigator Dr CR Roe and Paris France main investigator Dr G TOUATI It is planned to include 80 patients 60 in Dallas 20 in Paris during the next 2 years The patients will be affected with 6 proven defects that are specific defects of long-chain FAO carnitine palmitoyltransferase 1 CPT1 carnitine-acylcarnitine translocase CAT carnitine palmitoyltransferase 2 CPT2 very-long chain acyl-CoA dehydrogenase VLCAD L-3-hydroxy-acyl-CoA dehydrogenase LCHAD or trifunctional protein MTP
The used methodology will be a control randomized study to compare the efficiency of 2 diet therapies TRIHEPTANOIN versus conventional MCT The studied parameters will depend on each disease and will depend on the affected organs in each patient Main studied clinical parameters will be survival rate number of metabolic acute decompensation frequency and severity of hypoglycemias frequency and severity of rhabdomyolyses evolution of cardiac or hepatic manifestations muscular strength and quality of life Main studied biological parameters will be TRIHEPTANOIN use during meal tests modifications of plasma acylcarnitines profile modifications of urinary organic acids blood measurements of CPK and transaminases Cardiac echographies will be performed for the follow-up of cardiomyopathies ergometric testing and strength tests will be performed for disorders that affect muscular function
The efficiency of these compounds will be compared with conventional diet MCT for each patient Because of frequent phenotypic variations observed for each of these diseases each patient will be his own control
The same protocol study will be followed in 2 centers Dallas USA main investigator Dr CR Roe and Paris France main investigator Dr G TOUATI It is planned to include 80 patients 60 in Dallas 20 in Paris during the next 2 years The patients will be affected with 6 proven defects that are specific defects of long-chain FAO carnitine palmitoyltransferase 1 CPT1 carnitine-acylcarnitine translocase CAT carnitine palmitoyltransferase 2 CPT2 very-long chain acyl-CoA dehydrogenase VLCAD L-3-hydroxy-acyl-CoA dehydrogenase LCHAD or trifunctional protein MTP
The used methodology will be a control randomized study to compare the efficiency of 2 diet therapies TRIHEPTANOIN versus conventional MCT The studied parameters will depend on each disease and will depend on the affected organs in each patient Main studied clinical parameters will be survival rate number of metabolic acute decompensation frequency and severity of hypoglycemias frequency and severity of rhabdomyolyses evolution of cardiac or hepatic manifestations muscular strength and quality of life Main studied biological parameters will be TRIHEPTANOIN use during meal tests modifications of plasma acylcarnitines profile modifications of urinary organic acids blood measurements of CPK and transaminases Cardiac echographies will be performed for the follow-up of cardiomyopathies ergometric testing and strength tests will be performed for disorders that affect muscular function
Study Oversight
Has Oversight DMC:
None
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?:
None