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

This study has been completed.
Sponsor:
Collaborator:
Institut de Myologie, Pitié-Salpêtrière Hospital, France
Information provided by (Responsible Party):
Mette Cathrine Oerngreen, Rigshospitalet, Denmark
ClinicalTrials.gov Identifier:
NCT00983788
First received: September 23, 2009
Last updated: May 29, 2012
Last verified: May 2012
  Purpose

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 Intervention Phase
Carnitine Palmitoyltransferase II Deficiency
Very Long Chain Acyl Coa Dehydrogenase Deficiency
Drug: Bezafibrate
Other: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by Rigshospitalet, Denmark:

Primary Outcome Measures:
  • Fatty acid oxidation [ Time Frame: Two years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Heart rate [ Time Frame: Two years ] [ Designated as safety issue: No ]

Enrollment: 12
Study Start Date: October 2009
Study Completion Date: September 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Bezafibrate Drug: Bezafibrate
3 x 200 mg/ day
Placebo Comparator: Placebo Other: Placebo
Placebo

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • CPT II or VLCAD deficiency

Exclusion Criteria:

  • Competing disorders
  • Liver or kidney disease
  • Allergy towards fibrates
  • Pregnancy and breastfeeding
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00983788

Locations
Denmark
Neuromusculare Research Unit, Rigshospitalet
Copenhagen, Denmark, 2100
Sponsors and Collaborators
Rigshospitalet, Denmark
Institut de Myologie, Pitié-Salpêtrière Hospital, France
  More Information

No publications provided

Responsible Party: Mette Cathrine Oerngreen, MD, Rigshospitalet, Denmark
ClinicalTrials.gov Identifier: NCT00983788     History of Changes
Other Study ID Numbers: EudraCT: 2008-006704-27
Study First Received: September 23, 2009
Last Updated: May 29, 2012
Health Authority: Denmark: Danish Medicines Agency

Keywords provided by Rigshospitalet, Denmark:
CPT II
VLCAD

Additional relevant MeSH terms:
Lipid Metabolism, Inborn Errors
Muscular Diseases
Mitochondrial Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Bezafibrate
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Lipid Regulating Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on August 25, 2014