Huntington’s Disease Clinical Trials Corner: February 2018

Filipe B Rodrigues, Edward J Wild, Filipe B Rodrigues, Edward J Wild

Abstract

In the second edition of the Huntington’s Disease Clinical Trials Corner we list all currently registered and ongoing clinical trials, summarise the top-line results of the recently-announced IONIS-HTTRX trial (NCT02519036), expand on Wave Life Sciences’ PRECISION-HD1 (NCT03225833) and PRECISION-HD2 (NCT03225846), and cover one recently finished trial: the FIRST-HD deutetrabenazine trial (NCT01795859).

References

    1. Ionis Pharmaceuticals I. Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-HTTRx in PatientsWith Early Manifest Huntington’s Disease. ; 2015.
    1. Wave Life Sciences. Safety and Tolerability of WVE-120101 in Patients With Huntington’s Disease. ; 2017.
    1. Wave Life Sciences. Safety and Tolerability of WVE-120102 in Patients With Huntington’s Disease. ; 2017.
    1. Teva Pharmaceutical Industries. First Time Use of SD-809 in Huntington Disease. ; 2013.
    1. Huntington Study Group. Effect of deutetrabenazine on chorea among patients with huntington disease: A randomized clinical trial. Jama. 2016;316(1):40–50.
    1. Rodrigues FB, Wild EJ. Clinical trials corner: September 2017. J Huntingtons Dis. 2017;6(3):255–63.
    1. Ionis Pharmaceuticals I. Ionis Pharmaceuticals Licenses IONIS-HTT Rx to Partner Following Successful Phase 1/2a Study in Patients with Huntington’s Disease 2017. [Available from:
    1. The Guardian. Excitement as trial shows Huntington’s drug could slow progress of disease 2017. [Available from:
    1. CNN. Drug trial shows promising results to fight Huntington’s disease 2017. [Available from:
    1. HDSA. Breaking news: update on the status of the IONIS-HTTRX program and its future 2017. [Available from:
    1. Ionis Pharmaceuticals I. Study in Huntington’s Disease Patients Who Participated in Prior Investigational Studies of ISIS 443139. ; 2017.
    1. Wild EJ, Tabrizi SJ. Therapies targeting DNA and RNA in Huntington’s disease. Lancet Neurol. 2017;16(10):837–47.
    1. Kay C, Collins JA, Skotte NH, Southwell AL, Warby SC, Caron NS, et al. Huntingtin haplotypes provide prioritized target panels for allele-specific silencing in huntington disease patients of european ancestry. Molecular Therapy. 2015;23(11):1759–71.
    1. Saudou F, Humbert S. The biology of huntingtin. Neuron. 2016;89(5):910–26.
    1. Iwamoto N, Butler DCD, Svrzikapa N, Mohapatra S, Zlatev I, Sah DWY, et al. Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides. Nature biotechnology. 2017;35(9):845–51.
    1. Frank S, Testa CM, Stamler D, Kayson E, Davis C, Edmondson MC, et al. Effect of deutetrabenazine on chorea among patients with Huntington disease: A randomized clinical trial. Jama. 2016;316(1):40–50.
    1. Auspex Pharmaceuticals I. Alternatives for Reducing Chorea in HD. 2013.
    1. Frank S, Stamler D, Kayson E, Claassen DO, Colcher A, Davis C, et al. Safety of converting from tetrabenazine to deutetrabenazine for the treatment of chorea. JAMA Neurol. 2017;74(8):977–82.
    1. Rodrigues FB, Duarte GS, Costa J, Ferreira JJ, Wild EJ. Tetrabenazine versus deutetrabenazine for Huntington’s disease: Twins or distant cousins? Mov Disord Clin Pract 2017;4(4):582–5.
    1. Claassen DO, Carroll B, De Boer LM, Wu E, Ayyagari R, Gandhi S, et al. Indirect tolerability comparison of Deutetrabenazine and Tetrabenazine for Huntington disease. J Clin Mov Disord. 2017;4:3.
    1. Rodrigues FB, Duarte GS, Costa J, Ferreira JJ, Wild EJ. Meta-research metrics matter: Letter regarding article “indirect tolerability comparison of Deutetrabenazine and Tetrabenazine for Huntington disease”. J Clin Mov Disord. 2017;4(9):19.

Source: PubMed

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