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CompletedNCT00004770Updated Jun 24, 2005

Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

An interventional study of thioctic acid in Mitochondrial Myopathy, sponsored by National Center for Research Resources (NCRR). Completed. Open to male participants aged 0 Years and older. Per ClinicalTrials.gov, last updated 2005-06-24.

Sponsored by National Center for Research Resources (NCRR) · Not applicable, Interventional, and Treatment

Phase
Not applicable
Study type
Interventional
Enrollment
1
Ages
0 Years and older
Sex
Male
01

Study summary

OBJECTIVES:

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

Read the 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.

02

Conditions studied

  • Mitochondrial Myopathy

Keywords

  • inborn errors of metabolism
  • mitochondrial myopathy
  • rare disease
03

In context

Muscular Diseases

280 studies on the registry are indexed under Muscular Diseases; 63 are open to participants now.

This study's enrollment of 1 is below the median of 34 across 157 interventional studies indexed under Muscular Diseases.

Browse Muscular Diseases studies →

Lead sponsor

National Center for Research Resources (NCRR) is the lead sponsor of 265 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
0 Years and older
Sexes eligible
Male
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)
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Enrollment
1 participant

Interventions

  • Drugthioctic acid
06

Study locations

No study locations are listed for this record.

07

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 24, 2005, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
08

Registry details

Key details

Study ID
NCT00004770
Lead sponsor
National Center for Research Resources (NCRR)
Collaborators
University of Rochester
First posted
Feb 25, 2000
Start date
Oct 1995
Last update
Jun 24, 2005

Study contacts

Robert Griggs
study chair · University of Rochester
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Dec 2001. You cannot join it, but the record below documents what was studied.

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