- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05590468
A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy
June 6, 2023 updated by: Eduardo N. Chini
Randomized, Double-blinded, Placebo-controlled Study Evaluating the Efficacy of Nicotinamide Riboside (NR) - a Vitamin B3 Derivative - for Treatment of Mitochondrial Myopathy Disorder
The purpose of this study is to determine the effects of Nicotinamide Riboside (NR) supplement in adult-onset symptoms of mitochondrial myopathy.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
34
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Alexandra Miller
- Phone Number: 507-293-1139
- Email: miller.alexandra@mayo.edu
Study Locations
-
-
Minnesota
-
Rochester, Minnesota, United States, 55905
- Recruiting
- Mayo Clinic in Rochester
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Nicotinamide Riboside treatment group
Subjects will receive vitamin B3 derivative Nicotinamide Riboside (NR) daily for 12 months
|
Vitamin B3 derivative; a total of 1000 mg/day in a regimen of 500mg every 12 hours by mouth (either fasting or fed)
|
Placebo Comparator: Placebo Group
Subjects will receive a placebo daily for 12 months
|
Looks exactly like the study drug, but it contains no active ingredient
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in 6-Minute Walk Test Performance
Time Frame: Baseline, 6 months, 12 months
|
The 6-minute walk test (6MWT) quantifies the distance a person can walk in six minutes.
Measured in meters.
|
Baseline, 6 months, 12 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in skeletal muscle function
Time Frame: Baseline, 6 months, 12 months
|
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.
|
Baseline, 6 months, 12 months
|
Change in skeletal muscle strength
Time Frame: Baseline, 6 months, 12 months
|
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.
|
Baseline, 6 months, 12 months
|
Change in isometric strength of trunk flexors
Time Frame: Baseline, 6 months, 12 months
|
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.
|
Baseline, 6 months, 12 months
|
Change in isometric strength of trunk extensor muscle
Time Frame: Baseline, 6 months, 12 months
|
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.
|
Baseline, 6 months, 12 months
|
Change in maximal oxygen uptake (VO2 max)
Time Frame: Baseline, 6 months, 12 months
|
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.
|
Baseline, 6 months, 12 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Eduardo Chini, MD, PhD, Mayo Clinic
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
May 26, 2023
Primary Completion (Estimated)
November 1, 2025
Study Completion (Estimated)
November 1, 2025
Study Registration Dates
First Submitted
October 19, 2022
First Submitted That Met QC Criteria
October 19, 2022
First Posted (Actual)
October 21, 2022
Study Record Updates
Last Update Posted (Actual)
June 8, 2023
Last Update Submitted That Met QC Criteria
June 6, 2023
Last Verified
June 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Nervous System Diseases
- Musculoskeletal Diseases
- Neuromuscular Diseases
- Mitochondrial Diseases
- Muscular Diseases
- Mitochondrial Myopathies
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Vasodilator Agents
- Antimetabolites
- Micronutrients
- Hypolipidemic Agents
- Lipid Regulating Agents
- Vitamins
- Vitamin B Complex
- Nicotinic Acids
- Niacinamide
- Niacin
Other Study ID Numbers
- 21-005125
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Mitochondrial Myopathy
-
Reneo Pharma LtdTerminatedPrimary Mitochondrial MyopathySpain, Australia, Belgium, Denmark, Canada, United Kingdom, France, Germany, Hungary, Italy, Netherlands, New Zealand
-
The Emmes Company, LLCUnited Mitochondrial Disease Foundation; PolG Foundation Mito AustraliaRecruitingPolG, Primary Mitochondrial MyopathyUnited States
-
Reneo Pharma LtdCompletedPrimary Mitochondrial MyopathyUnited States, Spain, Australia, France, United Kingdom, New Zealand, Denmark, Norway, Czechia, Belgium, Canada, Germany, Hungary, Italy, Netherlands
-
Stealth BioTherapeutics Inc.TerminatedPrimary Mitochondrial MyopathyUnited States, Germany, Canada, Italy, United Kingdom, Denmark, Hungary
-
Reneo Pharma LtdTerminatedPrimary Mitochondrial MyopathyUnited Kingdom
-
Astellas Pharma IncActive, not recruitingPrimary Mitochondrial MyopathyUnited States
-
Reata, a wholly owned subsidiary of BiogenAbbVieCompletedMItochondrial MyopathiesUnited States, Denmark
-
Children's Hospital of PhiladelphiaUniversity of Pennsylvania; United Mitochondrial Disease Foundation (UMDF)Recruiting
-
Stealth BioTherapeutics Inc.CompletedMitochondrial MyopathyUnited States
-
Children's Hospital of PhiladelphiaNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMitochondrial Diseases | Mitochondrial MyopathiesUnited States
Clinical Trials on Nicotinamide Riboside
-
Cambridge University Hospitals NHS Foundation TrustUniversity of Cambridge; Medical Research Council Mitochondrial Biology UnitCompletedMitochondrial Diseases | Mitochondrial Myopathies | Progressive External Ophthalmoplegia | Progressive Ophthalmoplegia | Progressive; Ophthalmoplegia, External | Mitochondria DNA Deletion | MELASUnited Kingdom
-
National Heart, Lung, and Blood Institute (NHLBI)TerminatedHeart FailureUnited States
-
National Heart, Lung, and Blood Institute (NHLBI)CompletedObesity | Psoriasis | Dyslipidemia | Atherosclerotic Cardiovascular Disease | Cardiometabolic DiseasesUnited States
-
Iowa State UniversityCompleted
-
Franklin Health ResearchActive, not recruiting
-
National Heart, Lung, and Blood Institute (NHLBI)CompletedCancer | Muscle Weakness | Skin FibroblastsUnited States
-
ChromaDex, Inc.Midwest Center for Metabolic and Cardiovascular ResearchCompletedSleep | Cognitive Function | MoodUnited States
-
Haukeland University HospitalHaraldsplass Deaconess HospitalRecruitingDementia | Alzheimer DiseaseNorway
-
Société des Produits Nestlé (SPN)Active, not recruiting
-
MediGeneCharité Research Organisation GmbHCompletedGenital Warts | Perianal WartsGermany