Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

1995-10-31

Brief Summary

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OBJECTIVES:

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

Detailed Description

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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.

Conditions

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Mitochondrial Myopathy

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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thioctic acid

Intervention Type DRUG

Eligibility Criteria

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Inclusion 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)

Minimum Eligible Age

0 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Principal Investigators

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Robert Griggs

Role: STUDY_CHAIR

University of Rochester

Other Identifiers

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URMC-5231

Identifier Type: -

Identifier Source: secondary_id

199/11774