Overview

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