Overview

Effect of Nicotinamide in Friedreich's Ataxia

Status:
Unknown status
Trial end date:
2017-12-01
Target enrollment:
Participant gender:
Summary
The purpose of the interventional study is to determine whether Nicotinamide is effective at upregulating the Frataxin (FXN) gene in patients with Friedreich's ataxia (FRDA) where this gene is abnormally 'switched off'. The purpose of the non-interventional study is to investigate the use of novel, highly-sensitive technology to capture clinical deficit and measure subtle changes in the activities of daily living and to correlate functional changes to levels of expression of Frataxin protein and the epigenetic structure of the Frataxin gene over a 9-12 month period without nicotinamide. Healthy volunteers will be included as comparators in this part of the study.
Phase:
Phase 2
Details
Lead Sponsor:
Imperial College London
Treatments:
Histone Deacetylase Inhibitors
Niacin
Niacinamide
Nicotinic Acids