Mitochondrial MyopathyNCT00004770
Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
A study testing thioctic acid in people with Mitochondrial Myopathy, run by National Center for Research Resources (NCRR).
Trial summary
OBJECTIVES: I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial myopathy. Read more about this study
- Who is it for?
- Ages 0 years and older, men only
- Conditions the study is for
- Mitochondrial Myopathy
- What is being tested?
- thioctic acid
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- Oct 1995to not given
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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)
About this study
OBJECTIVES:
- 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.
- Conditions
- Mitochondrial Myopathy
- Being tested
- thioctic acid
- Keywords
- inborn errors of metabolism, mitochondrial myopathy, rare disease
- Medical subject headings
- Mitochondrial Myopathies, Metabolism, Inborn Errors, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
thioctic acidDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startOct 1995
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial MyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Enrolment
- 1 participants
Sponsor and investigators
- Lead sponsor
- National Center for Research Resources (NCRR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- University of Rochester
- Investigators
- Robert Griggs, Study chair, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004770
- Sponsor's protocol ID
- 199/11774
- Other IDs
- URMC-5231
- Status last verified
- Dec 2001
- First submitted
- 24 Feb 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
- ClinicalTrials.gov ID
- NCT00004770
- Acronym / sponsor ID
- 199/11774
- Conditions
- Mitochondrial Myopathy
- Keywords
- inborn errors of metabolism, mitochondrial myopathy, rare disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants
- Lead sponsor
- National Center for Research Resources (NCRR)US National Institutes of Health
- Collaborators
- University of Rochester
- Study start
- Oct 1995
- First posted
- 25 Feb 2000 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES:
- 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.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 0 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| thioctic acid | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Griggs study chair
University of Rochester
Questions about this trial
What is this clinical trial studying?
A study testing thioctic acid in people with Mitochondrial Myopathy, run by National Center for Research Resources (NCRR).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jun 2005. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2005.