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Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00004770
First Posted: February 25, 2000
Last Update Posted: December 9, 2005
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
University of Rochester
Information provided by:
Office of Rare Diseases (ORD)
  Purpose

OBJECTIVES:

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


Condition Intervention
Mitochondrial Myopathy Drug: thioctic acid

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 1
Study Start Date: October 1995
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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004770


Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Rochester
Investigators
Study Chair: Robert Griggs University of Rochester
  More Information

ClinicalTrials.gov Identifier: NCT00004770     History of Changes
Other Study ID Numbers: 199/11774
URMC-5231
First Submitted: February 24, 2000
First Posted: February 25, 2000
Last Update Posted: December 9, 2005
Last Verified: December 2001

Keywords provided by Office of Rare Diseases (ORD):
inborn errors of metabolism
mitochondrial myopathy
rare disease

Additional relevant MeSH terms:
Muscular Diseases
Mitochondrial Myopathies
Mitochondrial Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolic Diseases
Thioctic Acid
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances