Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
Assistance Publique - Hôpitaux de Paris
Usual dietary therapies of mitochondrial fatty acid oxidation disorders (FAO) are based on 3
- 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
Groupe Hospitalier Pitie-Salpetriere
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).
Serving leading biopharmaceutical companies globally:
Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors.
Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data.
The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free.
thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user.
Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.
Alerts Available With Subscription
Alerts are available for users with active subscriptions.