Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

OBJECTIVES:

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

Completed

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Key information

Conditions

Mitochondrial Myopathy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Mitochondrial Diseases Mitochondrial Myopathies Muscular Diseases Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

0 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Not applicable

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Thioctic Acid

Drug

Sponsors and collaborators

Lead sponsor

National Center for Research Resources (NCRR)

Nih

Collaborators

University of Rochester

Registry information

Important dates

Study start: 1995
First posted: Feb 25, 2000
Registry last updated: Jun 24, 2005

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed