What side effects are associated with this type of intervention?

Nicotinamide Riboside (NR), a supplement aimed at increasing NAD+ levels to enhance mitochondrial function, has been studied for its effects on mitochondrial myopathy. Known side effects of NR are generally mild and may include nausea, fatigue, headaches, and gastrointestinal discomfort. However, studies have shown that NR did not significantly affect the molecular phenotype of muscle or sustainably increase the NAD pool in tissues. Other treatments aimed at enhancing mitochondrial function, such as high-dose vitamins and antioxidants, may also cause side effects like gastrointestinal issues, dizziness, and potential toxicity with excessive use.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for mitochondrial myopathy that function by increasing NAD+ levels similar to Nicotinamide Riboside (NR). However, treatments such as Coenzyme Q10 and other supplements aimed at improving mitochondrial function are commonly used. These treatments focus on enhancing mitochondrial energy production and reducing oxidative stress, although their efficacy varies and they are not specifically approved by the FDA for mitochondrial myopathy.

What other types of research exist?

Promising alternatives to Nicotinamide Riboside for mitochondrial myopathy patients may include Coenzyme Q10 (ubiquinol-10), which has been studied for its role in improving oxidative imbalance and mitochondrial function. Additionally, other NAD+ precursors like Nicotinamide Mononucleotide (NMN) could be considered, as they also aim to enhance mitochondrial function by increasing NAD+ levels.

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Vitamin B3 Derivative

Vitamin B3 Derivative for Mitochondrial Myopathy

Phase 2

Recruiting

Led By Eduardo Chini, MD, PhD

Research Sponsored by Eduardo N. Chini

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Biochemically and/or genetically confirmed or confirmed primary mitochondrial myopathy based on published diagnostic criteria

Be older than 18 years old

Must not have

Inability or refusal to give informed consent by the patient or a legally authorized representative

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, 6 months, 12 months

Summary

This trial is testing a vitamin B3 supplement called Nicotinamide Riboside (NR) in adults who have muscle problems due to mitochondrial myopathy. The supplement aims to help their muscle cells produce more energy. Researchers hope this will improve symptoms in these patients. Nicotinamide riboside (NR), a form of vitamin B3, has been shown to boost NAD(+) levels and improve mitochondrial function.

Who is the study for?

This trial is for adults with confirmed mitochondrial myopathy who agree to use effective contraception and avoid other vitamin B3 supplements. It's not for pregnant or breastfeeding individuals, those in another drug study, or with conditions that could affect results.

What is being tested?

The study tests the effects of Nicotinamide Riboside (NR), a Vitamin B3 derivative, on adult-onset symptoms of mitochondrial myopathy compared to a placebo (a substance with no therapeutic effect).

What are the potential side effects?

Potential side effects may include mild gastrointestinal issues like nausea or indigestion due to high doses of vitamin B3 derivatives but specific side effects related to NR will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My muscle weakness is confirmed to be due to mitochondrial disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I cannot or will not give my consent to participate.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, 6 months, 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, 6 months, 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 6 months, 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Changes in 6-Minute Walk Test Performance

Secondary study objectives

Change in isometric strength of trunk extensor muscle

Change in isometric strength of trunk flexors

Change in maximal oxygen uptake (VO2 max)

+2 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Nicotinamide Riboside treatment groupExperimental Treatment1 Intervention

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

Group II: Placebo GroupPlacebo Group1 Intervention

Subjects will receive a placebo daily for 12 months

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Nicotinamide Riboside

2022

N/A

~160

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Nicotinamide Riboside (NR) is a precursor to Nicotinamide Adenine Dinucleotide (NAD+), a crucial coenzyme in cellular energy production. By increasing NAD+ levels, NR enhances mitochondrial function, which is often impaired in Mitochondrial Myopathy. This improvement in mitochondrial efficiency can lead to better energy production and reduced muscle fatigue, which are significant issues for patients with this condition. Other treatments, such as Coenzyme Q10, work similarly by supporting the electron transport chain, thereby improving ATP production and overall cellular energy. These treatments are vital as they target the root cause of energy deficits in Mitochondrial Myopathy, potentially improving quality of life and muscle function for patients.

Nicotinamide pretreatment protects cardiomyocytes against hypoxia-induced cell death by improving mitochondrial stress.