Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
OBJECTIVES:

 I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial
 myopathy.
PROTOCOL OUTLINE: The patient receives one thioctic acid tablet 3 times a day. If patient
 experiences no side effects after 1 week, 2 tablets are administered and given 3 times a day
 over 3 months for compassionate use.

 The patient is followed at weeks 3, 8, and 12.
PROTOCOL ENTRY CRITERIA:

 --Disease Characteristics--

 -  Long standing ophthalmoparesis and fatiguable weakness of the limbs

 -  Mild ataxia and no central nervous system involvement

 -  History of mitochondrial DNA deletion and a measurable biochemical defect of the
 respiratory chain

 -  Steady deterioration in skeletal muscle mass and power over 5 years

 --Prior/Concurrent Therapy--

 -  Previous participation in studies of muscle disease natural history (CRC Protocol
 183A)