Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004770
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
University of Rochester
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:


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

Condition or disease Intervention/treatment Phase
Mitochondrial Myopathy Drug: thioctic acid Not Applicable

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.

Study Type : Interventional  (Clinical Trial)
Enrollment : 1 participants
Primary Purpose: Treatment
Study Start Date : October 1995

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No


--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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004770

Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Rochester
Study Chair: Robert Griggs University of Rochester Identifier: NCT00004770     History of Changes
Other Study ID Numbers: 199/11774
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2001

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
mitochondrial myopathy
rare disease

Additional relevant MeSH terms:
Muscular Diseases
Mitochondrial Myopathies
Mitochondrial Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolic Diseases
Thioctic Acid
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Vitamin B Complex
Growth Substances