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Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders

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ClinicalTrials.gov Identifier: NCT03728777
Recruitment Status : Completed
First Posted : November 2, 2018
Last Update Posted : November 27, 2019
Information provided by (Responsible Party):
Nicoline Løkken, Rigshospitalet, Denmark

Brief 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. Investigators hypothesize an improved muscle metabolism, mitochondrial function, fatty acid oxidation and thus improvement of physical ability.

Condition or disease Intervention/treatment Phase
Mitochondrial Myopathies Fatty Acid Oxidation Defects Dietary Supplement: Resveratrol Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: double-blind, randomized, placebo-controlled, cross-over study.
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders: A Double-blind, Placebo-controlled, Cross Over Study
Actual Study Start Date : April 9, 2018
Actual Primary Completion Date : October 1, 2019
Actual Study Completion Date : October 1, 2019

Arm Intervention/treatment
Placebo Comparator: Placebo Dietary Supplement: Resveratrol
Resveratrol supplementation 1000 mg /day or placebo

Experimental: Resveratrol
Over the counter supplement
Dietary Supplement: Resveratrol
Resveratrol supplementation 1000 mg /day or placebo

Primary Outcome Measures :
  1. Heart rate [ Time Frame: 20 weeks ]
    Decrease in heart rate during constant load cycling exercise.

Secondary Outcome Measures :
  1. Peak oxygen utilization [ Time Frame: 20 weeks ]
    VO^2max (ml/min)

  2. Fatty acid oxidation [ Time Frame: 20 weeks ]
    Fatty acid oxidation will be assessed by stable isotope technique (only for fatty acid oxidation defect disease subgroup)

  3. Perceived exertion [ Time Frame: 20 weeks ]
    Evaluation of perceived exertion (Borg score) during constant workload cycling

  4. Fatigue Severity Scale score [ Time Frame: 20 weeks ]
    Evaluation of self-rated fatigue

  5. SF-36 questionnaire [ Time Frame: 20 weeks ]
    Evaluation of self-rated daily function scores

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patient is willing and able to provide written informed consent prior to participation.
  2. Patient is ≥18 and ≤80 years of age at baseline.
  3. Patients have genetically verified mitochondrial disorder or a fatty acid oxidation deficiency (VLCAD/CPTII).
  4. 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.
  5. Patient is ambulatory.

Exclusion Criteria:

  1. 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.
  2. Patient has symptoms of mitochondrial myopathy due to known secondary mitochondrial dysfunction (e.g., drug-induced myopathy).
  3. Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments.
  4. Pregnancy or breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03728777

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Copenhagen Neuromuscular Center
Copenhagen, Denmark, 2100
Sponsors and Collaborators
Rigshospitalet, Denmark
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Study Director: John Vissing, professor Copenhangen Neuromuscular Center, Rigshospitalet, Denmark
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Responsible Party: Nicoline Løkken, Medical Doctor, Rigshospitalet, Denmark
ClinicalTrials.gov Identifier: NCT03728777    
Other Study ID Numbers: H-17039503
First Posted: November 2, 2018    Key Record Dates
Last Update Posted: November 27, 2019
Last Verified: November 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Diseases
Mitochondrial Myopathies
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Mitochondrial Diseases
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Enzyme Inhibitors
Platelet Aggregation Inhibitors