The effect of Nicotinamide (Vitamin B3) in Friedreich’s ataxia

• Research type

  Research Study

• Full title

  Pharmacodynamic Studies of a Histone Deacetylase Inhibitor in FRDA

• IRAS ID

  47293

• Contact name

  Richard Festenstein

• Sponsor organisation

  Imperial College London

• Eudract number

  2011-002744-27

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Friedreich??s Ataxia is caused by abnormal DNA that might ??switch off?? a nearby gene called Frataxin, stopping it working properly. We want to look at the way this gene is ??switched off?? in blood cells taken from patients. We have recently identified a vitamin (Vitamin B3 or nicotinamide,), as a possible treatment for Friedreich??s ataxia. This is based on our finding that treatment of blood cells with Vitamin B3 results in ??switching on?? the Friedreich??s ataxia gene again. This study will measure the effect of ??switching on?? the Friedreich??s ataxia gene by giving Vitamin B3 to patients. If successful this could be the basis of a new treatment for Friedreich??s ataxia in the future.

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  11/LO/0998

• Date of REC Opinion

  1 Nov 2011

• REC opinion

  Further Information Favourable Opinion