Tocilizumab (TCZ) for the treatment of systemic sclerosis

• Research type

  Research Study

• Full title

  A Phase II/III, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Tocilizumab versus Placebo in Patients with Systemic Sclerosis

• IRAS ID

  89259

• Contact name

  Christopher Denton

• Sponsor organisation

  F. Hoffmann-La Roche Ltd

• Eudract number

  2011-001460-22

• ISRCTN Number

  n/a

• Research summary

  Systemic sclerosis (SSc) is a type of autoimmune disorder where the immune system mistakenly attacks and destroys healthy body tissue. It causes hardening and thickening of the skin, and can also affect blood vessels and internal organs. SSc is a rare disease with approximately 80,000 sufferers in Europe. In the UK, there are approximately 100 cases per million. Overall, the disease has a 10 year survival rate of approximately 70%. There is no cure for SSc and no treatment has been shown to modify overall disease progression across all affected organs. Evidence suggests that IL-6 (a protein released by cells of the immune system) may be involved in SSc; therefore blocking the IL-6 pathway may be a treatment option. Tocilizumab (TCZ) is medication that reduces the action of IL-6. It is approved in Europe for the treatment of rheumatoid arthritis but has not yet been formally studied in SSc. The purpose of the study is to assess the efficacy and safety of TCZ compared to placebo (dummy drug). Patients will be randomly assigned in a 1:1 ratio to receive weekly injections of 162mg TCZ or placebo, for 48 weeks. After this, all patients will receive TCZ for a further 48 weeks. Patients will attend study visits weekly for the first 4 weeks and then every 8 weeks. Procedures during the visits include physical examination, vital signs, ECG, chest x-ray, skin biopsy, blood and urine tests, SSc assessments and questionnaires. After the study, patients will be treated according to standard of care at the discretion of the study doctor. This study is sponsored by F. Hoffman-La Roche. Approximately 86 patients will participate in this study in Europe and North America, with approximately 30 patients from 7 hospitals in the UK.

• REC name

  London - Brent Research Ethics Committee

• REC reference

  11/LO/2005

• Date of REC Opinion

  19 Jan 2012

• REC opinion

  Further Information Favourable Opinion