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

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

Tracking Information

• First Submitted Date: April 19, 2018
• First Posted Date: November 2, 2018
• Last Update Posted Date: November 27, 2019
• Actual Study Start Date: April 9, 2018
• Actual Primary Completion Date: October 1, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 1, 2018)

Heart rate [ Time Frame: 20 weeks ]

Decrease in heart rate during constant load cycling exercise.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 1, 2018)

• Peak oxygen utilization [ Time Frame: 20 weeks ]
  VO^2max (ml/min)
• 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)
• Perceived exertion [ Time Frame: 20 weeks ]
  Evaluation of perceived exertion (Borg score) during constant workload cycling
• Fatigue Severity Scale score [ Time Frame: 20 weeks ]
  Evaluation of self-rated fatigue
• SF-36 questionnaire [ Time Frame: 20 weeks ]
  Evaluation of self-rated daily function scores

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders
• Official Title: Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders: A Double-blind, Placebo-controlled, Cross Over Study
• 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.

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 Type: Interventional
• Study Phase: Not Applicable

Study Design

Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:

double-blind, randomized, placebo-controlled, cross-over study.

Masking: Double (Participant, Investigator)
Primary Purpose: Treatment

Condition

• Mitochondrial Myopathies
• Fatty Acid Oxidation Defects

Intervention

Dietary Supplement: Resveratrol

Resveratrol supplementation 1000 mg /day or placebo

Study Arms

• Placebo Comparator: Placebo
  Intervention: Dietary Supplement: Resveratrol
• Experimental: Resveratrol
  Over the counter supplement
  Intervention: Dietary Supplement: Resveratrol

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: November 1, 2018): 20
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: October 1, 2019
• Actual Primary Completion Date: October 1, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 80 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Denmark

Administrative Information

• NCT Number: NCT03728777
• Other Study ID Numbers: H-17039503
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: Nicoline Løkken, Rigshospitalet, Denmark
• Original Responsible Party: Same as current
• Current Study Sponsor: Rigshospitalet, Denmark
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: John Vissing, professor; Copenhangen Neuromuscular Center, Rigshospitalet, Denmark

• PRS Account: Rigshospitalet, Denmark
• Verification Date: November 2019