Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

National Center for Research Resources (NCRR)1 enrolled

Overview

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

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

Purpose

TREATMENT

Enrollment

Conditions

Mitochondrial Myopathy

Interventions

thioctic acidDRUG

Eligibility

Sex: MALEMin age: 0 Years

Medical Language ↔ Plain English

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)

Data from ClinicalTrials.gov

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