Efficacy & Safety of DAXI for injection in Isolated Cervical Dystonia

  • Research type

    Research Study

  • Full title

    A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Trial to Evaluate the Efficacy and Safety of a Single Treatment of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia (ASPEN-1)

  • IRAS ID

    244655

  • Contact name

    Timothy Harrower

  • Contact email

    timothy.harrower@nhs.net

  • Sponsor organisation

    Revance Therapeutics Inc

  • Eudract number

    2018-000446-19

  • ISRCTN Number

    ISRCTN00000000

  • Clinicaltrials.gov Identifier

    NCT03608397

  • Clinicaltrials.gov Identifier

    N/A, N/A

  • Duration of Study in the UK

    1 years, 11 months, 0 days

  • Research summary

    Isolated Cervical Dystonia (CD) is a condition in which there are uncontrollable and often painful muscle contractions in the neck which cause awkward postures and discomfort. CD is the most common dystonia and affects an estimated 18,000 adults in the UK.

    One of the first line treatments for CD is botulinum neurotoxin (BoNT). BoNT is highly effective for the treatment of CD symptoms; however patient satisfaction is limited because of a decline in the effects of BoNT at approximately 9.5 weeks after their injection. This means that many patients have to rely on muscle relaxants and painkillers. A BoNT that is effective with a potentially longer lasting therapeutic effect, and that is well-tolerated by patients and safe, would be a welcome addition to the treatments available to physicians for management of CD.

    The study aims to compare the safety and effectiveness of a high and low dose of a new formulation of BoNT called daxibotulinumtoxinA for injection (DAXI for injection) against each other and a placebo.

    Participants in this study will be randomly assigned to one of three treatment groups;
    • High dose of DAXI for injection;
    • Low dose of DAXI for injection, and;
    • Placebo.

    As this is a double blind study, participants, the sponsor and the investigator will not know which study drug the participants will receive.

    This study will recruit participants who have CD and are between 18 and 80 years old, and be carried out at three NHS hospitals in the UK, as well as other hospitals in Europe, the USA and Canada. Participation is expected to be up to 39 weeks; 3 weeks for screening, followed by study drug administration at Baseline, then follow-up visits at Weeks 2, 4, 6, 12, and every 4 weeks thereafter to assess treatment response, tolerability, and safety up to Week 36.

  • REC name

    South West - Central Bristol Research Ethics Committee

  • REC reference

    18/SW/0216

  • Date of REC Opinion

    28 Nov 2018

  • REC opinion

    Further Information Favourable Opinion