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Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

This study has been completed.
University of Rochester
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 2001


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

Condition Intervention
Mitochondrial Myopathy
Drug: thioctic acid

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 1
Study Start Date: October 1995
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.


Genders 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)
  Contacts and Locations
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Please refer to this study by its identifier: NCT00004770

Sponsors and Collaborators
University of Rochester
Study Chair: Robert Griggs University of Rochester
  More Information

No publications provided Identifier: NCT00004770     History of Changes
Other Study ID Numbers: 199/11774, URMC-5231
Study First Received: February 24, 2000
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

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

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