Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

National Center for Research Resources (NCRR)

Status: Completed

Conditions: Mitochondrial Myopathy

Treatments: Drug: thioctic acid

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00004770

URMC-5231

199/11774

Details and patient eligibility

About

OBJECTIVES:

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

Full description

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.

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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)