Status:

COMPLETED

Nicotinamide Riboside and Mitochondrial Biogenesis

Lead Sponsor:

Cambridge University Hospitals NHS Foundation Trust

Collaborating Sponsors:

University of Cambridge

Medical Research Council Mitochondrial Biology Unit

Conditions:

Mitochondrial Diseases

Mitochondrial Myopathies

Eligibility:

All Genders

18-70 years

Phase:

NA

Brief 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 producti...

Detailed Description

Mitochondria are the primary source of energy within the cell, in the form of adenosine triphosphate (ATP). Mitochondrial ATP production is tightly regulated according to the energy requirements of th...

Eligibility Criteria

Inclusion

  • Clinical and genetic diagnosis of:
  • Progressive external ophthalmoplegia (PEO), caused by a single deletion of mitochondrial DNA
  • or
  • Mitochondrial disease caused by the m.3243A\>G or A\>T mutation in mitochondrial DNA
  • Age 18-70 years
  • Women of child-bearing age only if they are not pregnant or breast feeding at the inclusion into the study and agree not to become pregnant during the study. Female participants will undergo a pregnancy test before study commencement.
  • Signed informed patient consent

Exclusion

  • Clinically significant liver disease (e.g., cirrhosis or a history of hepatitis)
  • Presence of significant other neurological disorders (such as multiple sclerosis, Parkinson's disease) or major co-morbidities (such as definite cognitive impairment, psychiatric disease, heart or lung failure, orthopaedic or rheumatological disorders)
  • Females who are at risk of pregnancy (i.e., not using regular contraception), who are pregnant, lactating or planning pregnancy
  • Females taking the combined oral contraceptive pill (as oestrogens can induce mitochondrial biogenesis and interfere with study results)
  • For MRI - medical contraindication e.g., pacemaker, deep brain stimulator, aneurysm clip or significant claustrophobia
  • For biopsy - varicose veins overlying biopsy site, clinically significant lower limb oedema, active lower limb infection, use of anticoagulants or antiplatelet agents that cannot be discontinued, known bleeding diathesis

Key Trial Info

Start Date :

December 8 2017

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

December 31 2022

Estimated Enrollment :

13 Patients enrolled

Trial Details

Trial ID

NCT03432871

Start Date

December 8 2017

End Date

December 31 2022

Last Update

July 20 2023

Active Locations (1)

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1

Cambridge University Hospitals NHS Foundation Trust

Cambridge, United Kingdom, CB20QQ