Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Mitochondrial Myopathy Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004770
• Other study ID #: 199/11774
• Secondary ID: URMC-5231
• Status: Completed
• Phase: N/A
• First received: February 24, 2000
• Last updated: June 23, 2005
• Start date: October 1995

Study information

• Verified date: December 2001
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

OBJECTIVES:

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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 Design

Endpoint Classification: Efficacy Study, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mitochondrial Myopathies
• Mitochondrial Myopathy
• Muscular Diseases

Intervention

Drug:
• thioctic acid

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
National Center for Research Resources (NCRR)	University of Rochester