Safety of a selective inhibitor of SirT1 in Huntington’s Disease 2.0

• Research type

  Research Study

• Full title

  A Double-blind, Placebo-controlled Study in Huntington’s Disease Patients to Determine the Safety and Tolerability of SEN0014196

• IRAS ID

  80884

• Contact name

  David Craufurd

• Sponsor organisation

  Siena Biotech SpA

• Eudract number

  2011-001131-23

• Clinicaltrials.gov Identifier

  NCT01521585.

• Research summary

  Huntington's disease is an inheritable disorder which causes a wide range of symptoms affecting the central nervous system. Currently, there is no available treatment to stop or reverse the disease.The disease is characterised by a mutated gene which leads to production of a faulty protein, mutant huntingtin. SEN0014196 may increase the clearance of mutant huntingtin protein and consequently reduce the damage caused by the faulty gene. The aim of this study is to assess the safety and tolerability of 12 weeks treatment with SEN0014196 in patients with Huntington??s disease. Blood samples will also be taken to establish how best to give the drug in future studies (pharmacokinetics) and to see if the drug is having an effect on the faulty huntingtin protein(pharmacodynamics).Approximately 144 patients will be randomised into the study by clinics in the UK, Germany and Italy. Patients will be allocated randomly (by chance) to receive a once daily dose of 50 mg or 200 mg SEN0014196 or placebo (dummy drug).Following screening, eligible consenting patients will attend the clinic for assessments at 4-weekly intervals during the 12 week treatment period, and 4 weeks after completing the study treatment.Study procedures include confirmation of Huntington??s disease using genetic testing; physical and neurological examinations; electrocardiograms (ECGs); measurement of blood pressure, heart rate, respiratory rate and temperature; alcohol breath tests; blood sampling for safety, pharmacokinetic and pharmacodynamic measurements; and urine sampling for safety tests, and where appropriate, urine pregnancy tests.The patients?? functional status and physical and mental health will be assessed throughout the study using Global Clinical Impression (patient and clinician-based); the United Huntington??s Disease Rating Scale; UHDRS Total Motor Scale; Functional Assessment; Independence Scale Assessment; Problem Behaviours Assessment; and a cognitive battery (Circle-Tracing test; Symbol Digit Modalities Test; Stroop Word Test; Verbafluncy; Mini Mental State Examination).

• REC name

  East of England - Essex Research Ethics Committee

• REC reference

  11/EE/0266

• Date of REC Opinion

  24 Oct 2011

• REC opinion

  Further Information Favourable Opinion