NCT00004770

Brief Summary

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

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for not_applicable

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 1995

Completed
4.4 years until next milestone

First Submitted

Initial submission to the registry

February 24, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 25, 2000

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2001

First QC Date

February 24, 2000

Last Update Submit

June 23, 2005

Conditions

Mitochondrial Myopathy

Keywords

inborn errors of metabolismmitochondrial myopathyrare disease

Interventions

thioctic acidDRUG

Eligibility Criteria

Age0 Years+
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
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)

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

MeSH Terms

Conditions

Mitochondrial MyopathiesMetabolism, Inborn ErrorsRare Diseases

Interventions

Thioctic Acid

Condition Hierarchy (Ancestors)

Muscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Carboxylic AcidsOrganic ChemicalsThiophenesSulfur CompoundsCoenzymesEnzymes and CoenzymesFatty AcidsLipids

Study Officials

  • Robert Griggs

    University of Rochester

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
not applicable
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

October 1, 1995

Last Updated

June 24, 2005

Record last verified: 2001-12