MELAS SyndromeEarly Phase 1NCT01252979
Ketones & Mitochondrial Heteroplasmy
A Early Phase 1 study testing Medium-Chain Triglycerides in people with MELAS Syndrome and Mitochondrial Diseases, run by The University of Texas Health Science Center, Houston at 1 site in United States.
Trial summary
The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known mutation in their mtDNA at position 3243 (A>G). Subjects will be given supplemental medium chain triglycerides (MCTs) for a period of 6 months. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MELAS Syndrome, Mitochondrial Diseases
- What is being tested?
- Medium-Chain Triglycerides
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2010to Dec 2011
- Phase
- Early Phase 1
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is informed and given ample opportunity to consider his/her participation and has given his/her written consent.
- Subject is willing and able to comply with all trial requirements.
- Subject harbors the 3243 A>G mtDNA mutation at a level detectable in blood.
- Female subjects of child-bearing potential must not be pregnant. Female subjects of child-bearing potential (not surgically sterile or 2 years post-menopausal) must also agree to use appropriate contraceptive methods (abstinence, oral, injectable, implantable, or barrier) for the duration of the trial.
- Subject must not have diabetes mellitus.
Who cannot join
- Subject is currently participating or has participated within the last 2 months in any clinical trial involving treatment of mitochondrial disorders with MCT supplementation or induction of ketosis.
- Subject has a medical condition that could reasonably be exacerbated by ketone supplementation (including diabetes mellitus).
- Subject is unable to give reasonable informed consent/assent.
- Subject is a pregnant or nursing female.
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
About this study
The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known mutation in their mtDNA at position 3243 (A>G). Subjects will be given supplemental medium chain triglycerides (MCTs) for a period of 6 months. mtDNA heteroplasmy will be measured 3 months prior to treatment, at treatment initiation, and 6 months after initiation.
The primary objective of the current study is to determine if there is a shift in heteroplasmy in patients harboring the 3243 A>G mtDNA mutation to a more favorable (higher wild-type) profile while in a state of ketosis.
Read the detailed description
The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known pathogenic mutation in their mtDNA at position 3243 (A>G). Subjects will be induced in to ketosis by administration of supplemental medium chain triglycerides (MCTs) for a period of 6 months. mtDNA heteroplasmy will be assessed 3 months prior to treatment, at treatment initiation, and 6 months after initiation.
The primary objective of the current study is to determine if there is a shift in heteroplasmy in patients harboring the 3243 A>G mtDNA mutation to a more favorable (higher wild-type) genotypic profile while in a state of ketosis.
- Conditions
- MELAS Syndrome, Mitochondrial Diseases
- Being tested
- Medium-Chain Triglycerides
- Keywords
- MELAS, Ketones, Medium Chain Triglycerides, MCT oil, Mitochondrial Disorder, Heteroplasmy, 3243AG, Subject is informed and given ample opportunity to consider his/her participation and has given his/her written consent., Subject is willing and able to comply with all trial requirements., Subject harbors the 3243 A>G mtDNA mutation at a level detectable in blood., Female subjects of child-bearing potential must not be pregnant., Subjects must not have Diabetes Mellitus.
- Medical subject headings
- MELAS Syndrome, Mitochondrial Diseases, Ketosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Medium-Chain TriglyceridesDietary supplement
Subjects will take supplemental MCT oil 3 times a day for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Medium Chain Triglyceride | Experimental | Not described Receives: Dietary Supplement: Medium-Chain Triglycerides |
What is being measured
The primary outcome is the main question the study is designed to answer.
Heteroplasmy
Blood samples will be used to measure the degree of mitochondrial DNA heteroplasmy
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2010 (estimated)
- Study startDec 2010
- Primary completionDec 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MELAS SyndromeStudies of the same condition, open and under way.
- More trials run by The University of Texas Health Science Center, HoustonThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mary Kay Koenig, The University of Texas Health Science Center, Houston
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01252979
- Sponsor's protocol ID
- HSC-MS-10-0091
- Status last verified
- Feb 2012
- First submitted
- 1 Dec 2010
- Last updated
- 6 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ketones & Mitochondrial Heteroplasmy
- ClinicalTrials.gov ID
- NCT01252979
- Acronym / sponsor ID
- HSC-MS-10-0091
- Conditions
- MELAS Syndrome, Mitochondrial Diseases
- Keywords
- MELAS, Ketones, Medium Chain Triglycerides, MCT oil, Mitochondrial Disorder, Heteroplasmy, 3243AG, Subject is informed and given ample opportunity to consider his/her participation and has given his/her written consent.
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- The University of Texas Health Science Center, HoustonUniversity, hospital or other
- Study start
- Dec 2010
- Primary completion
- Dec 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 3 Dec 2010 (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.
The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known mutation in their mtDNA at position 3243 (A>G). Subjects will be given supplemental medium chain triglycerides (MCTs) for a period of 6 months. mtDNA heteroplasmy will be measured 3 months prior to treatment, at treatment initiation, and 6 months after initiation.
The primary objective of the current study is to determine if there is a shift in heteroplasmy in patients harboring the 3243 A>G mtDNA mutation to a more favorable (higher wild-type) profile while in a state of ketosis.
Detailed description
The current study is a prospective evaluation of the ability of ketosis to shift mitochondrial DNA (mtDNA) heteroplasmy in subjects harboring a known pathogenic mutation in their mtDNA at position 3243 (A>G). Subjects will be induced in to ketosis by administration of supplemental medium chain triglycerides (MCTs) for a period of 6 months. mtDNA heteroplasmy will be assessed 3 months prior to treatment, at treatment initiation, and 6 months after initiation.
The primary objective of the current study is to determine if there is a shift in heteroplasmy in patients harboring the 3243 A>G mtDNA mutation to a more favorable (higher wild-type) genotypic profile while in a state of ketosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Medium Chain Triglyceride | Experimental | Dietary Supplement: Medium-Chain Triglycerides |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Subject is informed and given ample opportunity to consider his/her participation and has given his/her written consent.
- Subject is willing and able to comply with all trial requirements.
- Subject harbors the 3243 A>G mtDNA mutation at a level detectable in blood.
- Female subjects of child-bearing potential must not be pregnant. Female subjects of child-bearing potential (not surgically sterile or 2 years post-menopausal) must also agree to use appropriate contraceptive methods (abstinence, oral, injectable, implantable, or barrier) for the duration of the trial.
- Subject must not have diabetes mellitus.
Key exclusion criteria (4 listed)
- Subject is currently participating or has participated within the last 2 months in any clinical trial involving treatment of mitochondrial disorders with MCT supplementation or induction of ketosis.
- Subject has a medical condition that could reasonably be exacerbated by ketone supplementation (including diabetes mellitus).
- Subject is unable to give reasonable informed consent/assent.
- Subject is a pregnant or nursing female.
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 |
|---|---|---|
| Medium-Chain TriglyceridesSubjects will take supplemental MCT oil 3 times a day for 6 months | Dietary supplement | Medium Chain Triglyceride |
Primary outcome
Heteroplasmy
Time frame: 9 months
Blood samples will be used to measure the degree of mitochondrial DNA heteroplasmy
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 6 Feb 2012.
Related trials for MELAS Syndrome
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Medium-Chain Triglycerides in people with MELAS Syndrome and Mitochondrial Diseases, run by The University of Texas Health Science Center, Houston at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. 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 6 Feb 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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.
What does the primary outcome measure?
Heteroplasmy (time frame: 9 months). It is what the study was designed to measure, not a finding.
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 6 Feb 2012; AzTrials last refreshed its copy on 2 Nov 2026.