Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
Completed
Assistance Publique - Hôpitaux de Paris
N/A
2006-06-01
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.
Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects
Completed
Groupe Hospitalier Pitie-Salpetriere
Phase 2
2009-10-01
The investigators propose to evaluate the effect of bezafibrate on metabolism during exercise
in 22 adult patients affected with carnitine palmitoyltransferase II (CPTII) or very-long
chain acyl-CoA-dehydrogenase (VLCAD) deficiencies. This study will be an 9-month, randomized,
double-blind, placebo-controlled crossover trial. The trial will be conducted in two centers:
Institut de Myologie, Pitié-Salpêtrière Hospital in France, and Rigshospitalet, University of
Copenhagen, in Denmark. The main criteria for assessing the potential effect of this drug
will be the fat oxidation rate studied during a moderate workload on cycle ergometer, after
infusion of stable isotopes (palmitate and glucose tracers).
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