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

NCT ID: NCT03728777

Last Updated: 2019-11-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-04-09
• Study Completion Date: 2019-10-01

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.

Conditions

Mitochondrial Myopathies; Fatty Acid Oxidation Defects

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Placebo

Group Type: PLACEBO_COMPARATOR

Resveratrol

Intervention Type: DIETARY_SUPPLEMENT

Resveratrol supplementation 1000 mg /day or placebo

Resveratrol

Over the counter supplement

Group Type: EXPERIMENTAL

Resveratrol

Intervention Type: DIETARY_SUPPLEMENT

Resveratrol supplementation 1000 mg /day or placebo

Interventions

Resveratrol

Resveratrol supplementation 1000 mg /day or placebo

Intervention Type: DIETARY_SUPPLEMENT

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Rigshospitalet, Denmark

OTHER

Sponsor Role: lead

Responsible Party

Nicoline Løkken

Medical Doctor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

John Vissing, professor

Role: STUDY_DIRECTOR

Copenhangen Neuromuscular Center, Rigshospitalet, Denmark

Locations

Copenhagen Neuromuscular Center

Copenhagen, , Denmark

Countries

Denmark

Other Identifiers

H-17039503

Identifier Type: -

Identifier Source: org_study_id