Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects

• Status: Completed
• Trial end date: 2011-09-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rigshospitalet, Denmark
• Collaborator: Groupe Hospitalier Pitie-Salpetriere
• Treatments: Bezafibrate

Criteria

Inclusion Criteria:

• CPT II or VLCAD deficiency

Exclusion Criteria:

• Competing disorders

• Liver or kidney disease

• Allergy towards fibrates

• Pregnancy and breastfeeding