A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy

What Was Tested

In this phase 2 trial, researchers set out to test NAD+.

The purpose of this study is to determine the effects of Nicotinamide Riboside (NR) supplement in adult-onset symptoms of mitochondrial myopathy.

It had two arms (groups): Nicotinamide Riboside treatment group (experimental) and Placebo Group (placebo comparator).

Subjects will receive vitamin B3 derivative Nicotinamide Riboside (NR) daily for 12 months.

Subjects will receive a placebo daily for 12 months.

The registry classes the interventions as dietary supplement and drug.

Who Took Part

The registry lists 34 planned participants were the target.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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. Written, informed consent to participate in the study.

The full record lists 6 inclusion and 13 exclusion criteria; among the exclusions: 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.

How the Study Was Run

Design: randomized, parallel-group and double-blind.

Random assignment meant chance, not the researchers, decided who got NAD+ and who got the comparison, which limits bias.

The NAD+ group and the comparison group ran side by side rather than switching.

As a double-blind NAD+ study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant and investigator.

It started in 2023-05-26 with completion listed for 2026-11.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Changes in 6-Minute Walk Test Performance (time frame: Baseline, 6 months, 12 months).

It also tracked 5 secondary outcomes, for example Change in skeletal muscle function, Change in skeletal muscle strength, Change in isometric strength of trunk flexors, Change in isometric strength of trunk extensor muscle and Change in maximal oxygen uptake (VO2 max).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of NAD+ in 34 people.

Its size is small, so on its own it can only give an early look.

Its status is active not recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the NAD+ compound page, all human trials.