Bezafibrate Trial in CPT2 Deficiency (NCT00336167) | Clinical Trial Compass
UnknownPhase 3
Bezafibrate Trial in CPT2 Deficiency
France12 participantsStarted 2006-06
Plain-language 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
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
Rate of 3H-palmitate oxidation in the patients'lymphocytes and skeletal muscle