Back to resultsA Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy
Primary Purpose
Mitochondrial Myopathy
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Nicotinamide Riboside
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Mitochondrial Myopathy
Eligibility Criteria
Inclusion Criteria:
- Biochemically and/or genetically confirmed or confirmed primary mitochondrial myopathy based on published diagnostic criteria.
- Biochemically confirmed mitochondrial disorder would mean that the patient meets clinical criteria and has either biopsy or biochemical testing that supports the diagnosis.
- Confirmed mitochondrial disorder means that the patient meets published clinical criteria for the diagnosis and has also had confirmatory genetic testing for the disorder type.
- Agreed to avoid vitamin supplementation or nutritional products with vitamin B3 forms 14 days prior to the enrollment and during the study in order to not exceed 200 mg/day of vitamin B3 derivatives intake.
- Female of childbearing potential agreed to use effective contraception throughout the study.
- Written, informed consent to participate in the study.
Exclusion Criteria:
- Unwilling to comply with the follow-up schedule.
- Inability or refusal to give informed consent by the patient or a legally authorized representative.
- Known pregnancy or breastfeeding.
- Concurrent participation in another investigational drug study or within washout period of treatment.
- Presence of other medical symptoms or condition, which may interfere with interpretation of outcome measures as determined by the study PI.
Clinical / Laboratory Exclusion Criteria:
- Estimated Glomerular Filtration Rate (eGFR) < 30 mL/min.
- Patients in permanent Renal Replacement Therapy.
- Serum alkaline phosphatase 50% above normal limit.
- Serum aspartate transaminase 50% above normal limit.
- Serum Thyroxine (T4) 50% above or below normal limit.
- Serum Thyroid Stimulating Hormone (TSH) 50% above or below normal limit.
- Severe anemia with Hb < 7g/dL.
- Severe leukocytosis with WBC > 15,000/mm^3.
Sites / Locations
- Mayo Clinic in RochesterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Nicotinamide Riboside treatment group
Placebo Group
Arm Description
Subjects will receive vitamin B3 derivative Nicotinamide Riboside (NR) daily for 12 months
Subjects will receive a placebo daily for 12 months
Outcomes
Primary Outcome Measures
Changes in 6-Minute Walk Test Performance
The 6-minute walk test (6MWT) quantifies the distance a person can walk in six minutes. Measured in meters.
Secondary Outcome Measures
Change in skeletal muscle function
Measured using the Short Physical Performance Battery (SPPB) score based on timed measures of standing balance, walking speed and ability to rise from a chair. Each of the 3 performance measures will be assigned a score ranging from 0 to 4, with 4 indicating the highest level of performance and 0 the inability to complete the test. A summary score (range 0-12) will be subsequently calculated by adding the 3 scores.
Change in skeletal muscle strength
Measured using the strength one repetition maximum (1RM leg press) defined as the maximal weight an individual can lift for only one repetition with correct technique.
Change in isometric strength of trunk flexors
Assessed by generated maximum force against a stationary dynamometer reported in kilograms of force. Trunk flexion strength tested in the supine position with knees slightly bent, arms at side and head in midline. The base of the dynamometer will be placed on the middle of the sternum and the patient will be instructed to exert isometric force by lifting both scapula off the plinth.
Change in isometric strength of trunk extensor muscle
Assessed by generated maximum force against a stationary dynamometer reported in kilograms of force. Trunk extensor muscle strength tested in prone position. The base of the dynamometer will be placed at the level of the T4 spine and the patient instructed to generate an isometric force by lifting the chest off the plinth. After sufficient practice in the standardized positions, 5 trials will be recorded in each position.
Change in maximal oxygen uptake (VO2 max)
Maximal oxygen uptake (VO2 max) test measures the maximum rate of oxygen consumption during incremental exercise (exercise of increasing intensity) and reflects the cardiorespiratory fitness of an individual. Reported in ml/kg/min.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05590468
Brief Title
A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy
Official Title
Randomized, Double-blinded, Placebo-controlled Study Evaluating the Efficacy of Nicotinamide Riboside (NR) - a Vitamin B3 Derivative - for Treatment of Mitochondrial Myopathy Disorder
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 26, 2023 (Actual)
Primary Completion Date
November 2025 (Anticipated)
Study Completion Date
November 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Eduardo N. Chini
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to determine the effects of Nicotinamide Riboside (NR) supplement in adult-onset symptoms of mitochondrial myopathy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mitochondrial Myopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
34 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Nicotinamide Riboside treatment group
Arm Type
Experimental
Arm Description
Subjects will receive vitamin B3 derivative Nicotinamide Riboside (NR) daily for 12 months
Arm Title
Placebo Group
Arm Type
Placebo Comparator
Arm Description
Subjects will receive a placebo daily for 12 months
Intervention Type
Dietary Supplement
Intervention Name(s)
Nicotinamide Riboside
Intervention Description
Vitamin B3 derivative; a total of 1000 mg/day in a regimen of 500mg every 12 hours by mouth (either fasting or fed)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Looks exactly like the study drug, but it contains no active ingredient
Primary Outcome Measure Information:
Title
Changes in 6-Minute Walk Test Performance
Description
The 6-minute walk test (6MWT) quantifies the distance a person can walk in six minutes. Measured in meters.
Time Frame
Baseline, 6 months, 12 months
Secondary Outcome Measure Information:
Title
Change in skeletal muscle function
Description
Measured using the Short Physical Performance Battery (SPPB) score based on timed measures of standing balance, walking speed and ability to rise from a chair. Each of the 3 performance measures will be assigned a score ranging from 0 to 4, with 4 indicating the highest level of performance and 0 the inability to complete the test. A summary score (range 0-12) will be subsequently calculated by adding the 3 scores.
Time Frame
Baseline, 6 months, 12 months
Title
Change in skeletal muscle strength
Description
Measured using the strength one repetition maximum (1RM leg press) defined as the maximal weight an individual can lift for only one repetition with correct technique.
Time Frame
Baseline, 6 months, 12 months
Title
Change in isometric strength of trunk flexors
Description
Assessed by generated maximum force against a stationary dynamometer reported in kilograms of force. Trunk flexion strength tested in the supine position with knees slightly bent, arms at side and head in midline. The base of the dynamometer will be placed on the middle of the sternum and the patient will be instructed to exert isometric force by lifting both scapula off the plinth.
Time Frame
Baseline, 6 months, 12 months
Title
Change in isometric strength of trunk extensor muscle
Description
Assessed by generated maximum force against a stationary dynamometer reported in kilograms of force. Trunk extensor muscle strength tested in prone position. The base of the dynamometer will be placed at the level of the T4 spine and the patient instructed to generate an isometric force by lifting the chest off the plinth. After sufficient practice in the standardized positions, 5 trials will be recorded in each position.
Time Frame
Baseline, 6 months, 12 months
Title
Change in maximal oxygen uptake (VO2 max)
Description
Maximal oxygen uptake (VO2 max) test measures the maximum rate of oxygen consumption during incremental exercise (exercise of increasing intensity) and reflects the cardiorespiratory fitness of an individual. Reported in ml/kg/min.
Time Frame
Baseline, 6 months, 12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Biochemically and/or genetically confirmed or confirmed primary mitochondrial myopathy based on published diagnostic criteria.
Biochemically confirmed mitochondrial disorder would mean that the patient meets clinical criteria and has either biopsy or biochemical testing that supports the diagnosis.
Confirmed mitochondrial disorder means that the patient meets published clinical criteria for the diagnosis and has also had confirmatory genetic testing for the disorder type.
Agreed to avoid vitamin supplementation or nutritional products with vitamin B3 forms 14 days prior to the enrollment and during the study in order to not exceed 200 mg/day of vitamin B3 derivatives intake.
Female of childbearing potential agreed to use effective contraception throughout the study.
Written, informed consent to participate in the study.
Exclusion Criteria:
Unwilling to comply with the follow-up schedule.
Inability or refusal to give informed consent by the patient or a legally authorized representative.
Known pregnancy or breastfeeding.
Concurrent participation in another investigational drug study or within washout period of treatment.
Presence of other medical symptoms or condition, which may interfere with interpretation of outcome measures as determined by the study PI.
Clinical / Laboratory Exclusion Criteria:
Estimated Glomerular Filtration Rate (eGFR) < 30 mL/min.
Patients in permanent Renal Replacement Therapy.
Serum alkaline phosphatase 50% above normal limit.
Serum aspartate transaminase 50% above normal limit.
Serum Thyroxine (T4) 50% above or below normal limit.
Serum Thyroid Stimulating Hormone (TSH) 50% above or below normal limit.
Severe anemia with Hb < 7g/dL.
Severe leukocytosis with WBC > 15,000/mm^3.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Alexandra Miller
Phone
507-293-1139
Email
miller.alexandra@mayo.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Eduardo Chini, MD, PhD
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic in Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
https://www.mayo.edu/research/clinical-trials
Description
Mayo Clinic Clinical Trials
Learn more about this trial
A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy
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