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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: 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.


Ages Eligible for Study:   Child, Adult, Senior
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)
  Contacts and Locations
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Please refer to this study by its identifier: NCT00004770

Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Rochester
Study Chair: Robert Griggs University of Rochester
  More Information Identifier: NCT00004770     History of Changes
Other Study ID Numbers: 199/11774
Study First Received: February 24, 2000
Last Updated: June 23, 2005

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 processed this record on August 23, 2017