A8241022 - PF-02545920 IN SUBJECTS WITH HUNTINGTON'S DISEASE

  • Research type

    Research Study

  • Full title

    An open label extension study to investigatethe long term safety, tolerability and efficacy of PF-02545920 in subjects with Huntington's disease who previously completed study A8241021

  • IRAS ID

    174190

  • Contact name

    David Craufurd

  • Contact email

    david.craufurd@manchester.ac.uk

  • Sponsor organisation

    Pfizer Inc

  • Eudract number

    2014-001291-56

  • Clinicaltrials.gov Identifier

    118,646, US IND

  • Duration of Study in the UK

    2 years, 0 months, days

  • Research summary

    Huntington's disease (HD) is a neurodegenerative genetic disorder that damages specific areas of the brain, results in movement difficulties and leads to mental and cognitive decline. Symptoms of the disease become noticeable in mid-adult life. The disease can affect both men and women. The disease is caused by an autosomal dominant mutation in either of an individual's two copies of a gene called ‘Huntingtin’, which means a child of an affected person has a 50% chance of inheriting the disease. HD is ultimately fatal and there is currently no effective disease-modifying treatment.

    The purpose of this research study is to assess long-term safety and tolerability of 20mg BID (twice a day) of PF0254920 in patients with HD who completed the previous study A8241021.

    There will be about 260 people enrolled in this study. This study is being done at about 40 different research sites in 5 countries.

    This study is an open label, extension study, to investigate the long-term safety, tolerability and efficacy of PF02545920. This means that both doctors and participants know which treatment is being administrated. All participants who proceed from the previous study A84241021 will receive PF 02545920 20mg BID. Those who started with PF02545920 5mg BID will be titrated, starting with 5 mg BID and increase by 5 mg Bid every 7 days, up to a 20 mg BID to be reached at day 22, and maintained until month 12.

    Participants will be in the study for 12 months. They will need to visit the research site at least 8 times during the study. Participants at selected sites will participate in the Magnetic Resonance Imaging (MRI) sub-study. At these sites subjects will undergo brain MRI scans, one at visit 6 and one at visit 8

  • REC name

    Yorkshire & The Humber - Leeds East Research Ethics Committee

  • REC reference

    15/YH/0127

  • Date of REC Opinion

    24 Apr 2015

  • REC opinion

    Further Information Favourable Opinion