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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00983788
Recruitment Status : Completed
First Posted : September 24, 2009
Last Update Posted : May 30, 2012
Groupe Hospitalier Pitie-Salpetriere
Information provided by (Responsible Party):
Mette Cathrine Oerngreen, Rigshospitalet, Denmark

Brief 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).

Condition or disease Intervention/treatment Phase
Carnitine Palmitoyltransferase II Deficiency Very Long Chain Acyl Coa Dehydrogenase Deficiency Drug: Bezafibrate Other: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Evaluation of the Effect of Bezafibrate on Muscle Metabolism During Exercise in Patients With CPTII and VLCAD Deficiency
Study Start Date : October 2009
Actual Primary Completion Date : September 2011
Actual Study Completion Date : September 2011

Arm Intervention/treatment
Experimental: Bezafibrate Drug: Bezafibrate
3 x 200 mg/ day

Placebo Comparator: Placebo Other: Placebo

Primary Outcome Measures :
  1. Fatty acid oxidation [ Time Frame: Two years ]

Secondary Outcome Measures :
  1. Heart rate [ Time Frame: Two years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • CPT II or VLCAD deficiency

Exclusion Criteria:

  • Competing disorders
  • Liver or kidney disease
  • Allergy towards fibrates
  • Pregnancy and breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00983788

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Neuromusculare Research Unit, Rigshospitalet
Copenhagen, Denmark, 2100
Sponsors and Collaborators
Rigshospitalet, Denmark
Groupe Hospitalier Pitie-Salpetriere
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Responsible Party: Mette Cathrine Oerngreen, MD, Rigshospitalet, Denmark Identifier: NCT00983788    
Other Study ID Numbers: EudraCT: 2008-006704-27
First Posted: September 24, 2009    Key Record Dates
Last Update Posted: May 30, 2012
Last Verified: May 2012
Keywords provided by Mette Cathrine Oerngreen, Rigshospitalet, Denmark:
Additional relevant MeSH terms:
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Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents