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

Mitochondrial Myopathies Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03728777
• Other study ID #: H-17039503
• Status: Completed
• Phase: N/A
• Start date: April 9, 2018
• Est. completion date: October 1, 2019

Study information

• Verified date: November 2019
• Source: Rigshospitalet, Denmark
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: October 1, 2019
• Est. primary completion date: October 1, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Fatty Acid Oxidation Defects
• Mitochondrial Myopathies
• Muscular Diseases

Intervention

Dietary Supplement:
• Resveratrol
Resveratrol supplementation 1000 mg /day or placebo

Locations

Country	Name	City	State
Denmark	Copenhagen Neuromuscular Center	Copenhagen

Sponsors (1)

Lead Sponsor	Collaborator
Rigshospitalet, Denmark

Country where clinical trial is conducted

Denmark, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Heart rate	Decrease in heart rate during constant load cycling exercise.	20 weeks
Secondary	Peak oxygen utilization	VO^2max (ml/min)	20 weeks
Secondary	Fatty acid oxidation	Fatty acid oxidation will be assessed by stable isotope technique (only for fatty acid oxidation defect disease subgroup)	20 weeks
Secondary	Perceived exertion	Evaluation of perceived exertion (Borg score) during constant workload cycling	20 weeks
Secondary	Fatigue Severity Scale score	Evaluation of self-rated fatigue	20 weeks
Secondary	SF-36 questionnaire	Evaluation of self-rated daily function scores	20 weeks