Status:
UNKNOWN
Bezafibrate Trial in CPT2 Deficiency
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborating Sponsors:
Association Française contre les Myopathies (AFM), Paris
Institut National de la Santé Et de la Recherche Médicale, France
Conditions:
Carnitine Palmitoyl Transferase 2 Deficiency
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
The purpose of this study is to determine whether bezafibrate is effective in the treatment of the muscular adult form of carnitine palmitoyltransferase 2 deficiency
Detailed Description
Fatty acids are the main source of energy for non-glucodependent tissues during fasting and prolonged exercise. Carnitine Palmitoyltransferase (CPT) 1 and 2 are a key-enzymes in the regulation of mito...
Eligibility Criteria
Inclusion
- occurrence of at least 5 attacks of rhabdomyolysis or of severe myalgias per year, AND/OR permanent muscle weakness objectivized by muscle testing outside a rhabdomyolysis attack AND
- significant decrease in both the CPT2 activity and the rate of long-chain fatty acid oxidation measured in lymphocytes and/or in a skeletal muscle sample outside a rhabdomyolysis attack
Exclusion
- age below 18 years
- less than 5 attacks of rhabdomyolysis or severe myalgias per year AND absence of muscle impairment detected by muscle testing
- liver failure, renal failure, hyperhomocysteinemia prior to setting up the bezafibrate therapy
- treatment with another hypolipidemic drug ("statins) or with anticoagulant
- pregnancy or lactation during the period of fibrate therapy
Key Trial Info
Start Date :
June 1 2006
Trial Type :
INTERVENTIONAL
End Date :
July 1 2007
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT00336167
Start Date
June 1 2006
End Date
July 1 2007
Last Update
April 9 2007
Active Locations (1)
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1
Jean Paul Bonnefont
Paris, France, 75015