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A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension

Descriptive Information
Brief Title † A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension
Official Title † A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension
Brief Summary An open label study to assess the safety and efficacy of tocilizumab in group 1 pulmonary arterial hypertension patients
Detailed Description 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 not 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 bodies own 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 PAH. Tocilizumab was demonstrated to be safe and effective in trials in other diseases associated with PAH, such as rheumatoid arthritis. This study is a 6 month open label phase II trial of IV Tocilizumab in 21 patients with group 1 PAH. The aim of the trial is to see if Tocilizumab is safe and whether it reduces the blood pressure in the lungs. Patients will be given Tocilizumab intravenously once a month for six months with close safety monitoring. The trial will be led by Papworth Hospital and a total of 7 UK specialist 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.
Study Phase Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Safety - Incidence and severity of adverse events
Secondary Outcome Measure † Six minute walk test
Condition † Pulmonary Arterial Hypertension
Intervention † DrugTocilizumab
Study Arms / Comparison Groups Open Label Intravenous Tocilizumab 8mg/kg monthly (up to a maximum dose 800mg) for 6 months
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 21
Start Date † January 2016
Completion Date September 30, 2017
Primary Completion Date September 2017
Eligibility Criteria † Inclusion Criteria: - Group 1 PAH due to: Idiopathic or Heritable PAH, PAH associated with connective tissue disease excluding SLE, RA and mixed CTD, Drug and Toxins - WHO functional class II-IV - Weight more than 40kg - 6 minute walk distance of 100-500 m - Haemodynamic criteria measure by RHC - Documented negative V/Q scan or pulmonary arteriogram confirming absence of chromic thromboembolic disease - Resting oxygen saturations of >85% - Lung function confirming absence of significant lung disease - Stable on unchanged PAH therapeutic regime for at least 1 month Exclusion Criteria: - Subjects on continuous infusions either intravenously or subcutaneously - Hypersensitivity to Investigational Product - Severe hepatic impairment - Severe renal impairment - Clinically significant anaemia - Blood platelets
Gender All
Ages 18 Years - 70 Years
Accepts Healthy Volunteers No
Contacts †† Louise Harlow, 01480364107, louise.harlow@papworth.nhs.uk
Location Countries † United Kingdom
Administrative Information
NCT ID † NCT02676947
Organization ID PO2060
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Papworth Hospital NHS Foundation Trust
Collaborators †† Roche Pharma AG
Investigators † Principal Investigator: Mark Toshner, MD, Papworth Hospital
Information Provided By
Verification Date April 2017
First Received Date † December 21, 2015
Last Updated Date April 13, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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