Nicotinamide Riboside and Mitochondrial Biogenesis

Summary

Mitochondria are important parts of the cell that are responsible for producing energy. The amount of energy they produce depends on how much energy the body needs to function and this energy production can be severely impaired in people with mitochondrial disease. Symptoms of mitochondrial disease vary widely but usually involve the brain, nerves and muscles, as these are tissues that need a lot of energy. Mitochondrial disorders affect 1 in 5000 of the UK population and there is currently no cure.

Some scientists think that increasing the number of mitochondria in the body (mitochondrial biogenesis) might be an effective treatment for the symptoms of mitochondrial disease. Studies carried out in mice have shown that a type of B-vitamin called Nicotinamide Riboside (NR) is able to increase the number of mitochondria, leading to increased energy and a reduction in the symptoms of mitochondrial disease.

The aim of this study is to investigate if the same B vitamin, Nicotinamide Riboside, can increase energy production and reduce symptoms in humans with mitochondrial disease.

The study will consist of two parts:

Part 1: Participants will be given a single oral dose of Nicotinamide Riboside and the levels of NR in their bloodstream will be measured at regular intervals. This will involve a single overnight stay and simple blood tests.

Part 2: This requires 6 separate visits from each participant. Each participant will undergo a series of standard tests including a muscle biopsy and an MRI scan, then they will take a course of Nicotinamide Riboside (twice daily for 4 weeks). After 4 weeks of treatment, the participants will undergo the same tests again to see if there have been any changes in response to the treatment.

Conditions

• Mitochondrial Diseases
• Mitochondrial Myopathies
• Progressive External Ophthalmoplegia
• Progressive Ophthalmoplegia
• Progressive; Ophthalmoplegia, External
• Mitochondria DNA Deletion
• MELAS

Interventions

DIETARY_SUPPLEMENT

Nicotinamide Riboside

Nicotinamide Riboside is a member of the Vitamin B family which acts as a precursor to NAD+, an enzyme involved in energy production.

Sponsors & Collaborators

• University of Cambridge

  collaborator OTHER

• Medical Research Council Mitochondrial Biology Unit

  collaborator UNKNOWN

• Cambridge University Hospitals NHS Foundation Trust

  lead OTHER

Principal Investigators

• Patrick Chinnery, Prof. · University of Cambridge

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

18 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-12-08

Primary Completion

2022-12-31

Completion

2022-12-31

Countries

• United Kingdom

Study Locations