Sclero XIII

  • Research type

    Research Study

  • Full title

    Sclero XIII: A phase II ,double-blind, randomized, placebo-controlled study to investigate pharmacokinetics (PK), safety and efficacy of intravenous factor XIII treatment in patients with systemic sclerosis

  • IRAS ID

    150751

  • Contact name

    Chris Denton

  • Contact email

    c.denton@ucl.ac.uk

  • Eudract number

    2014-001101-40

  • Duration of Study in the UK

    3 years, 1 months, 1 days

  • Research summary

    This is a phase II ,double-blind, randomized, placebo-controlled study to investigate pharmacokinetics (PK), safety and efficacy of intravenous factor XIII treatment in patients with systemic sclerosis.

    Scleroderma (Systemic sclerosis) is a multisystem rheumatic disease that is characterised by progressive vascular damage e.g Raynaud’s phenomenon and digital ulcers and organ fibrosis e.g skin thickening and pulmonary fibrosis.

    The disease is associated with significant morbidity and mortality and current therapeutic options are only partially effective, including cyclophosphamide for skin or lung fibrosis and bosentan which reduces but does not heal digital ulcers. There is no cure available and there is therefore a high need for new therapeutic options.

    Administration of human factor XIII concentrate in patients with scleroderma demonstrated promising results in the 1980s and 1990s . However these studies were not performed according to current GCP guidelines and involved relatively small sample sizes.

    The main inclusion criteria are:
    1.Male and female adults aged = 18 years,
    2.Subjects with a diagnosis of limited or diffuse SSc,
    3.Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test
    4.Patients who have given their free and informed consent

  • REC name

    London - Hampstead Research Ethics Committee

  • REC reference

    15/LO/0073

  • Date of REC Opinion

    17 Feb 2015

  • REC opinion

    Further Information Favourable Opinion