I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial myopathy.
- Study Type: Interventional
- Study Design
- Primary Purpose: Treatment
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.
- Drug: thioctic acid
Participating in This Clinical Trial
PROTOCOL ENTRY CRITERIA:
- 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
- Previous participation in studies of muscle disease natural history (CRC Protocol 183A)
Gender Eligibility: Male
Minimum Age: N/A
Maximum Age: N/A
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- National Center for Research Resources (NCRR)
- University of Rochester
- Overall Official(s)
- Robert Griggs, Study Chair, University of Rochester
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