- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004770
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.
Study Overview
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.
Study Type
Enrollment
Phase
- Not Applicable
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Collaborators
Investigators
- Study Chair: Robert Griggs, University of Rochester
Study record dates
Study Major Dates
Study Start
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Nervous System Diseases
- Musculoskeletal Diseases
- Neuromuscular Diseases
- Mitochondrial Diseases
- Muscular Diseases
- Mitochondrial Myopathies
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Protective Agents
- Micronutrients
- Vitamins
- Antioxidants
- Vitamin B Complex
- Thioctic Acid
Other Study ID Numbers
- 199/11774
- URMC-5231
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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