An open label study of Tocilizumab in PAH
Research type
Research Study
Full title
TRANSFORM-UK: A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension
IRAS ID
185662
Contact name
Mark Toshner
Contact email
Sponsor organisation
Papworth Hospital NHS Foundation Trust
Eudract number
2015-002799-26
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
In pulmonary arterial hypertension (PAH) raised blood pressure in the lungs leads to heart failure and early death. Patients not only have a significantly reduced life expectancy, but their quality of life is severely affected. If left untreated life expectancy is 2-3 years. Current treatments all aim to relax the vessels in the lung and lower the blood pressures, however none target the causes of the disease and currently there is no cure. Despite the availability of treatments the impact on mortality has been modest at best with one third of patients still dying within two years of diagnosis. There remains an urgent need to test new ways of treating PAH.
PAH is often associated with auto-immune diseases (when the body's own immune system attacks itself rather than fight infection). Targeting components of the immune system involved in the development of disease offer a potential new area of treatment for PAH; an example known to be involved in the progression of PAH is the protein Interleukin-6 (IL-6).Tocilizumab is a drug which blocks the action of interleukin-6 and blocking interleukin-6 has been shown to be effective in animal models of pulmonary arterial hypertension. Tocilizumab was demonstrated to be safe and effective in trials in other diseases associated with PAH, such as rheumatoid arthritis.
We propose a clinical trial of Tocilizumab in PAH to see if it is safe and reduces the blood pressure in the lungs . 21 patients will be given Tocilizumab intravenously once a month for 6 months, with close safety monitoring. The trial will be lead by Papworth Hospital and a total of 7 UK centres will take part. The trial will assess the safety of the drug and response to treatment by measuring heart function, blood pressure in the lungs, exercise capacity and quality of life measurements.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
15/EM/0401
Date of REC Opinion
24 Sep 2015
REC opinion
Further Information Favourable Opinion