Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy



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

Study Type

  • Study Type: Interventional
  • Study Design
    • Primary Purpose: Treatment

Detailed 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.


  • Drug: thioctic acid

Participating in This Clinical Trial


–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)

Gender Eligibility: Male

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • National Center for Research Resources (NCRR)
  • Collaborator
    • University of Rochester
  • Overall Official(s)
    • Robert Griggs, Study Chair, University of Rochester

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