AFQ056 Proof of Concept study in patients with Huntington's disease

• Research type

  Research Study

• Full title

  A multi-centre, randomized, double-blind, placebo-controlled, parallel-group, multiple oral dose titration Proof of Concept study in patients with Huntington's disease to assess the efficacy, safety and tolerability of AFQ056 in reducing chorea

• IRAS ID

  30162

• Contact name

  Anne Rosser

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2009-011743-39

• ISRCTN Number

  N/A

• Research summary

  This study is designed to identify if the study drug AFQ056 is safe and has beneficial effects in people with Huntington's disease suffering from chorea (involuntary movements). AFQ056 works against certain signal receptors (mGlu5 receptor) in the brain, present in regions that control movements. This study is being conducted for 3 reasons: 1) investigate the effectiveness of AFQ056 as a drug that reduces chorea in Huntington??s disease, 2) confirm this drug is safe and 3) study how the human body tolerates this drug. Patients who join in this study have ??equal? or 50 % chance of being treated with either AFQ056 or the placebo (. Patients will be asked to take capsules 2 times a day for 32 days.This is a ??titration study?. Patients will start on a low dose of AFQ056 or placebo (25 mg twice daily). The dose will be slowly increased (every 4th day) until it reaches the maximum (150mg twice-daily). Should side-effects occur, the doctor can reduce the dose.A sub-population of patients will be asked to give extra blood samples and Cerebro Spinaflud (CSF) samples for the optional biomarker studies. The goals of this are to (1) find biomarkers that will identify persons with Huntington??s disease who will have the best possible response to AFQ056 and to (2) identify persons who will have fewer side effects in order to maximize their benefit from AFQ056.About 60 patients will join in this study at centres in UK and Germany.This is the first time that AFQ056 will be studied in patients with Huntington??s disease. However, this is not the first time that AFQ056 has been given to people. To date 230 subjects have been treated with AFQ056. No serious or significant safety concerns have been observed in these subjects.

• REC name

  West Midlands - Coventry & Warwickshire Research Ethics Committee

• REC reference

  09/H1211/84

• Date of REC Opinion

  15 Dec 2009

• REC opinion

  Further Information Favourable Opinion