Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

This study has been completed.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	National Center for Research Resources (NCRR) University of Rochester
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004770

Purpose

OBJECTIVES:

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

Condition	Intervention
Mitochondrial Myopathy	Drug: thioctic acid

Genetics Home Reference related topics: mitochondrial neurogastrointestinal encephalopathy disease

MedlinePlus related topics: Muscle Disorders

Drug Information available for: Thioctic Acid

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Efficacy Study

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.

Eligibility

Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

--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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004770

Sponsors and Collaborators

National Center for Research Resources (NCRR)

University of Rochester

Investigators

Study Chair:

Robert Griggs

University of Rochester

More Information

No publications provided

Study ID Numbers:	199/11774, URMC-5231
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004770 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

inborn errors of metabolism
mitochondrial myopathy
rare disease

Study placed in the following topic categories:

Antioxidants
Metabolic Diseases
Vitamin B Complex
Rare Diseases
Trace Elements
Mitochondrial Diseases
Metabolism, Inborn Errors
Muscular Diseases

Musculoskeletal Diseases
Neuromuscular Diseases
Vitamins
Mitochondrial Myopathies
Micronutrients
Metabolic Disorder
Thioctic Acid

Additional relevant MeSH terms:

Antioxidants
Metabolic Diseases
Vitamin B Complex
Molecular Mechanisms of Pharmacological Action
Growth Substances
Nervous System Diseases
Physiological Effects of Drugs
Mitochondrial Diseases
Protective Agents

Pharmacologic Actions
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Vitamins
Mitochondrial Myopathies
Micronutrients
Thioctic Acid

ClinicalTrials.gov processed this record on May 07, 2009