NICOFA
Research type
Research Study
Full title
A RANDOMISED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICH´S ATAXIA (NICOFA)
IRAS ID
248392
Contact name
Richard Festenstein
Contact email
Sponsor organisation
RWTH Aachen University, Center for Translational & Clinical Research Aachen (CTC-A)
Eudract number
2017-002163-17
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 8 months, 29 days
Research summary
Friedreich’s ataxia is a rare inherited disease affecting between 1 in 20,000 to 1 in 50,000 people across Europe. It usually starts in childhood and results in a progressive loss of coordination, slurred speech, difficulty swallowing and breathlessness due to heart failure. There is no cure or treatment that can slow down the disease which frequently results in severe disability by early adulthood.
The disease is inherited due to a gene mutation which partially ‘switches off’ the Frataxin gene resulting in lower levels of frataxin protein being made. Frataxin deficiency is what is causing the disease.
Our research showed that nicotinamide (vitamin B3) could be a potential treatment for restoring the frataxin protein levels. Nicotinamide interacts with the affected frataxin gene and allows it to be ‘switched on’. This was further investigated in a recent small exploratory clinical trial on 10 Friedreich’s ataxia patients that showed that nicotinamide was well tolerated by patients and its intake resulted in switching on of the frataxin gene.
This study will be carried out across several European countries and will investigate whether nicotinamide is an effective treatment for Friedreich’s ataxia. Patients will be given either placebo or nicotinamide (ratio 1:2) at a dose shown to be well tolerated and safe. Neither the study team nor the patients will know whether they are taking placebo or nicotinamide. After treatment is allocated patients will be asked to come for a safety visit after a month of taking the study drug. Then the study visits will continue at 4-monthly intervals for 1 year and then 6-monthly for another year to determine the safety of the treatment and how rapidly their disease is progressing.
This study will be the first to provide clinical evidence for the efficacy and safety of nicotinamide in patients with Friedreich´s ataxia.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0258
Date of REC Opinion
5 Nov 2019
REC opinion
Further Information Favourable Opinion