Mitochondrial MyopathiesNCT03728777
Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders
A study testing Resveratrol in people with Mitochondrial Myopathies and Fatty Acid Oxidation Defects, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial summary
The purpose of this study is to investigate the potential beneficial effects of a daily supplement of Resveratrol (1000mg/day) on physical ability and on muscle metabolism in patients with verified mitochondrial myopathy and patients with a verified fatty acid oxidation defect of VLCAD and CPTII deficiencies. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Mitochondrial Myopathies, Fatty Acid Oxidation Defects
- What is being tested?
- Resveratrol
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteDenmark
- Study dates
- 9 Apr 2018to 1 Oct 2019
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient is willing and able to provide written informed consent prior to participation.
- Patient is ≥18 and ≤80 years of age at baseline.
- Patients have genetically verified mitochondrial disorder or a fatty acid oxidation deficiency (VLCAD/CPTII).
- Patient has a clinical presentation, signs or symptoms suggestive of myopathy (e.g., easy fatigability, exercise intolerance, muscle pain) in the opinion of the Investigator.
- Patient is ambulatory.
Who cannot join
- Patient has any prior or current medical conditions that, in the judgment of the Investigator, would prevent the patient from safely participating in and/or completing all study requirements.
- Patient has symptoms of mitochondrial myopathy due to known secondary mitochondrial dysfunction (e.g., drug-induced myopathy).
- Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments.
- Pregnancy or breastfeeding
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders: A Double-blind, Placebo-controlled, Cross Over Study
The purpose of this study is to investigate the potential beneficial effects of a daily supplement of Resveratrol (1000mg/day) on physical ability and on muscle metabolism in patients with verified mitochondrial myopathy and patients with a verified fatty acid oxidation defect of VLCAD and CPTII deficiencies. Investigators hypothesize an improved muscle metabolism, mitochondrial function, fatty acid oxidation and thus improvement of physical ability.
Read the detailed description
Study design: double-blind, randomized, placebo-controlled, cross-over study.
To ensure enough participants, and due to the risk of a heterogeneous cohort, a cross-over design is chosen, where participants are their own controls. 10 patients with mitochondrial myopathy and 10 patients with fatty acid oxidation defects will be included. Eligible patients will be randomized using a 1:1 assignment ratio to receive placebo or RSV first. Each treatment will be administered orally twice daily for 8 weeks, followed by a 4 weeks wash-out, and afterwards a new 8-week treatment period.
During the 20-week trial period, subjects will visit the trial site on five occasions, for functional assessments (cycle ergometer testing), blood sampling and questionnaires.
- Being tested
- Resveratrol
- Medical subject headings
- Mitochondrial Myopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ResveratrolDietary supplement
Resveratrol supplementation 1000 mg /day or placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Dietary Supplement: Resveratrol |
| Resveratrol | Experimental | Over the counter supplement Receives: Dietary Supplement: Resveratrol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Heart rate
Decrease in heart rate during constant load cycling exercise.
Measured: 20 weeks
Secondary outcomes (5)
Peak oxygen utilization
VO^2max (ml/min)
Measured: 20 weeks
Fatty acid oxidation
Fatty acid oxidation will be assessed by stable isotope technique (only for fatty acid oxidation defect disease subgroup)
Measured: 20 weeks
Perceived exertion
Evaluation of perceived exertion (Borg score) during constant workload cycling
Measured: 20 weeks
Fatigue Severity Scale score
Evaluation of self-rated fatigue
Measured: 20 weeks
SF-36 questionnaire
Evaluation of self-rated daily function scores
Measured: 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2018
- Study start9 Apr 2018
- Primary completion1 Oct 2019
- Study completion1 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mitochondrial MyopathiesStudies of the same condition, open and under way.
- More trials run by Copenhagen University Hospital, RigshospitaletThe 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- double-blind, randomized, placebo-controlled, cross-over study.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nicoline Løkken, Copenhagen University Hospital, Rigshospitalet
- Investigators
- John Vissing, professor, Study director, Copenhangen Neuromuscular Center, Rigshospitalet, Denmark
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03728777
- Sponsor's protocol ID
- H-17039503
- Status last verified
- Nov 2019
- First submitted
- 19 Apr 2018
- Last updated
- 27 Nov 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders: A Double-blind, Placebo-controlled, Cross Over Study
- ClinicalTrials.gov ID
- NCT03728777
- Acronym / sponsor ID
- H-17039503
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- 9 Apr 2018 (actual)
- Primary completion
- 1 Oct 2019 (actual)
- Study completion
- 1 Oct 2019 (actual)
- First posted
- 2 Nov 2018 (actual)
- 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 purpose of this study is to investigate the potential beneficial effects of a daily supplement of Resveratrol (1000mg/day) on physical ability and on muscle metabolism in patients with verified mitochondrial myopathy and patients with a verified fatty acid oxidation defect of VLCAD and CPTII deficiencies. Investigators hypothesize an improved muscle metabolism, mitochondrial function, fatty acid oxidation and thus improvement of physical ability.
Detailed description
Study design: double-blind, randomized, placebo-controlled, cross-over study.
To ensure enough participants, and due to the risk of a heterogeneous cohort, a cross-over design is chosen, where participants are their own controls. 10 patients with mitochondrial myopathy and 10 patients with fatty acid oxidation defects will be included. Eligible patients will be randomized using a 1:1 assignment ratio to receive placebo or RSV first. Each treatment will be administered orally twice daily for 8 weeks, followed by a 4 weeks wash-out, and afterwards a new 8-week treatment period.
During the 20-week trial period, subjects will visit the trial site on five occasions, for functional assessments (cycle ergometer testing), blood sampling and questionnaires.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- double-blind, randomized, placebo-controlled, cross-over study.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Dietary Supplement: Resveratrol |
| Resveratrol | Experimental | Dietary Supplement: ResveratrolOver the counter supplement |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patient is willing and able to provide written informed consent prior to participation.
- Patient is ≥18 and ≤80 years of age at baseline.
- Patients have genetically verified mitochondrial disorder or a fatty acid oxidation deficiency (VLCAD/CPTII).
- Patient has a clinical presentation, signs or symptoms suggestive of myopathy (e.g., easy fatigability, exercise intolerance, muscle pain) in the opinion of the Investigator.
- Patient is ambulatory.
Key exclusion criteria (4 listed)
- Patient has any prior or current medical conditions that, in the judgment of the Investigator, would prevent the patient from safely participating in and/or completing all study requirements.
- Patient has symptoms of mitochondrial myopathy due to known secondary mitochondrial dysfunction (e.g., drug-induced myopathy).
- Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments.
- Pregnancy or breastfeeding
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 |
|---|---|---|
| ResveratrolResveratrol supplementation 1000 mg /day or placebo | Dietary supplement | Placebo, Resveratrol |
Primary outcome
Heart rate
Time frame: 20 weeks
Decrease in heart rate during constant load cycling exercise.
5 secondary outcomes
Peak oxygen utilization
Time frame: 20 weeks
Fatty acid oxidation
Time frame: 20 weeks
Perceived exertion
Time frame: 20 weeks
Fatigue Severity Scale score
Time frame: 20 weeks
SF-36 questionnaire
Time frame: 20 weeks
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 27 Nov 2019.
- Individual participant dataSharing planned: Undecided
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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.
John Vissing, professor study director
Copenhangen Neuromuscular Center, Rigshospitalet, Denmark
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Resveratrol in people with Mitochondrial Myopathies and Fatty Acid Oxidation Defects, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
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 27 Nov 2019. 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 Denmark. 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. Some participants in this study may receive a placebo. 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.
What does the primary outcome measure?
Heart rate (time frame: 20 weeks). 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 27 Nov 2019.