Pilot Compassionate Use Study of Thioctic Acid Treatment... | NCT00004770

Studies
Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

NCT00004770

Sponsor

National Center for Research Resources (NCRR)

Status

Completed

Last Update Posted

Jun 24, 2005Estimated

Enrollment

Phase

Not Applicable

Conditions

Mitochondrial Myopathy

Interventions

thioctic acid

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
URMC-5231

Status Module

Record Verification Date

Dec 2001

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 1995

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Feb 24, 2000

First Submission Date that Met QC Criteria

Feb 24, 2000

First Posted Date

Feb 25, 2000Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 23, 2005

Last Update Posted Date

Jun 24, 2005Estimated

Name	Class
University of Rochester	OTHER

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

Name	Type	Description	Arm Group Labels	Other Names
thioctic acid	Drug

Eligibility Module

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)

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

0 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Robert Griggs	University of Rochester	Study Chair

Condition Browse Module

MeSH Terms

ID	Term
D017240	Mitochondrial Myopathies
D008661	Metabolism, Inborn Errors
D035583	Rare Diseases

Ancestor Terms

ID	Term
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D009468	Neuromuscular Diseases
D009422	Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D008063	Thioctic Acid

Ancestor Terms

ID	Term
D002264	Carboxylic Acids
D009930	Organic Chemicals
D013876	Thiophenes
D013457	Sulfur Compounds

Browse Leaves

Not provided

Browse Branches

Not provided