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233AS101 - Phase 1 Study in ALS

  • Research type

    Research Study

  • Full title

    A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis



  • Contact name

    Pamela Shaw

  • Contact email

  • Sponsor organisation

    Biogen Idec Research Limited

  • Eudract number


  • Duration of Study in the UK

    2 years, 1 months, 0 days

  • Research summary

    Amyotrophic lateral sclerosis (ALS), is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles (muscle action we are able to control, such as those in the arms, legs, and face).

    Although the majority of patients suffer from sporadic ALS (without family history), approximately 2%, have an inherited, or familial, form of ALS caused by a variety of genetic mutations known as superoxide dismutase 1 mutations (SOD1-ALS).

    BIIB067 is an investigational drug which reduces the levels of the toxic protein that causes the genetic mutation. The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of BIIB067, in adults with SOD1-ALS.

    The study is randomised (participants are randomly allocated to treatment), double-blind (neither the participant nor study team will know which treatment is being given) and placebo-controlled (the study drug will be compared with an inactive “placebo”). It consists of 2-parts, A (single ascending dose) and B (multiple ascending dose) and BIIB067 will be administered by intrathecal bolus injection (via an injection into the spinal canal) to up to approximately 72 adults with either sporadic ALS (Part A only) or SOD1-ALS (Parts A and B).

    Approximately 17 sites are planned in the United States, Canada, and Western Europe.
    This study is sponsored by Biogen Idec Research Limited.

  • REC name

    Yorkshire & The Humber - Leeds East Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    18 Apr 2016

  • REC opinion

    Further Information Favourable Opinion