Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
Primary Purpose
Mitochondrial Myopathy
Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
thioctic acid
Sponsored by
About this trial
This is an interventional treatment trial for Mitochondrial Myopathy focused on measuring inborn errors of metabolism, mitochondrial myopathy, rare disease
Eligibility 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)
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004770
First Posted
February 24, 2000
Last Updated
June 23, 2005
Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Rochester
1. Study Identification
Unique Protocol Identification Number
NCT00004770
Brief Title
Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
Study Type
Interventional
2. Study Status
Record Verification Date
December 2001
Overall Recruitment Status
Completed
Study Start Date
October 1995 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Rochester
4. Oversight
5. Study Description
Brief Summary
OBJECTIVES:
I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial myopathy.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mitochondrial Myopathy
Keywords
inborn errors of metabolism, mitochondrial myopathy, rare disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Enrollment
1 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
thioctic acid
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Griggs
Organizational Affiliation
University of Rochester
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
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