Bezafibrate Trial in CPT2 Deficiency

• Status: Unknown status
• Trial end date: 2007-07-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: Association Française contre les Myopathies (AFM), Paris; Institut National de la Santé Et de la Recherche Médicale, France
• Treatments: Bezafibrate

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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