Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: Male

Summary

OBJECTIVES:

I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial myopathy.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Center for Research Resources (NCRR)
• Collaborator: University of Rochester
• Treatments: Thioctic Acid

Criteria

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)