Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
Dietary Therapy of Mitochondrial Fatty Acids Oxidation. A Clinical Study of Treatment With Odd Carbons Medium-chain Fatty Acids
1 other identifier
interventional
20
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Jun 2006
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 18, 2006
CompletedFirst Posted
Study publicly available on registry
May 19, 2006
CompletedStudy Start
First participant enrolled
June 1, 2006
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2010
CompletedFebruary 17, 2011
March 1, 2007
4 years
May 18, 2006
February 16, 2011
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Strength tests
24 months
Biological parameters (acylcarnitines profile, modifications of urinary organic acids)
24 months
Clinical parameters (echocardiography)
24 months
Secondary Outcomes (4)
Ergometric testing
24 months
Hepatic functions
24 months
Hypoglycaemia
24 months
Rhabdomyolyses
24 months
Interventions
Eligibility Criteria
You may qualify if:
- Patient with an enzyme deficiency of 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).
- Covered by health and social insurance
- Written informed consent
You may not qualify if:
- Patient affected with FAO dysfunction secondary to other cause (e.g. mitochondrial respiratory chain disorders)
- Patient with suspected FAO disorder that has not been proven (by enzymatic or molecular test)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Necker University Hospital - Metabolism Unit
Paris, 75743, France
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Guy Touati, PU-PH
Assistance Publique - Hôpitaux de Paris
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
Study Record Dates
First Submitted
May 18, 2006
First Posted
May 19, 2006
Study Start
June 1, 2006
Primary Completion
June 1, 2010
Study Completion
June 1, 2010
Last Updated
February 17, 2011
Record last verified: 2007-03