Can a vitamin B3 pill help people with rare muscle disease?
NCT ID NCT05590468
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether nicotinamide riboside, a form of vitamin B3, can improve walking distance and muscle function in adults with mitochondrial myopathy, a rare muscle disorder. Thirty-four participants receive either the supplement or a placebo for several months. The main goal is to see if the supplement helps them walk farther in six minutes.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- nicotinamide riboside (a form of vitamin B3)
- What this could lead to
- If it works, this could point toward a simple vitamin supplement to help manage mitochondrial myopathy symptoms.
- What could go wrong
- This is a small, early-phase trial with only 34 people. The supplement may not improve walking or muscle function, and results may not apply to everyone with the condition.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
About 34 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
May 2023
- Expected to finish
-
Nov 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Mayo Clinic in Rochester
Rochester, Minnesota, 55905, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.