Long-Term Safety & Efficacy of DAXI for injection in Isolated-CD

  • Research type

    Research Study

  • Full title

    A Phase 3, Open-Label, Multi-Center Trial to Evaluate the Long-Term Safety and Efficacy of Repeat Treatments of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia (ASPEN-OLS)

  • IRAS ID

    247695

  • Contact name

    Timothy Harrower

  • Contact email

    timothy.harrower@nhs.net

  • Sponsor organisation

    Revance Therapeutics Inc

  • Eudract number

    2018-000447-11

  • ISRCTN Number

    ISRCTN00000000

  • Clinicaltrials.gov Identifier

    NCT03617367

  • Duration of Study in the UK

    2 years, 9 months, 4 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.

    This study aims to evaluate the immunogenicity (the ability to provoke an immune response) of the components of a new formulation of BoNT called daxibotulinumtoxinA (DAXI) and its long-term safety after multiple injections.

    This is a non-randomised, open label study; this means the participants, sponsor and investigator will know which dosage each participant will receive. An initial dose of 125U or 250U in Cycle 1 (determined by the investigator) will be given. The dosage may remain unchanged, increase or decrease in later cycles.
    This study will recruit 350 adults (18-80 years old) who have CD across 80 sites in the USA, Canada and Europe. This includes participants who took part in the first phase 3 study (comparing the efficacy of DAXI against a placebo). Participation will be up to 55 weeks; 3 weeks of screening, DAXI will be administered at baseline, then follow-up visits will occur at the clinic at Weeks 4, 6, 12, and every 4 weeks thereafter until Week 52 or the end of study visit.

  • REC name

    South West - Central Bristol Research Ethics Committee

  • REC reference

    18/SW/0221

  • Date of REC Opinion

    28 Nov 2018

  • REC opinion

    Further Information Favourable Opinion