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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02676947
• Date of registration: 21/12/2015
• Prospective Registration: Yes
• Primary sponsor: Papworth Hospital NHS Foundation Trust
• Public title: A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension TRANSFORM-UK
• Scientific title: A Therapeutic Open Label Study of Tocilizumab in the Treatment of Pulmonary Arterial Hypertension
• Date of first enrolment: January 2016
• Target sample size: 29
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02676947
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United Kingdom

Contacts

• Name: Mark Toshner, MD
• Affiliation: Papworth Hospital

Key inclusion & exclusion 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 <100x10

- Neutrophil count <2x10/L

- Left ventricular disease/dysfunction risk factors

- Myocardial infarction within 90 days prior to screening

- Female subjects who are pregnant or breastfeeding

- History of malignancies within past 5 years



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pulmonary Arterial Hypertension

Intervention(s)

Drug: Tocilizumab

Primary Outcome(s)

Pulmonary vascular resistance- dynes (cm-5) [Time Frame: Change from baseline pulmonary vascular resistance to end of study at 6 months]

Safety - Incidence and severity of adverse events [Time Frame: 6 months]

Secondary Outcome(s)

World Health Organisation functional class assessment of patient reported symptoms [Time Frame: Baseline and every month for 6 months]

Quality of Life [Time Frame: Baseline and every month for 6 months]

N-Terminal pro-B-type Natriuretic Peptide [Time Frame: Baseline and every month for 6 months]

Six minute walk test [Time Frame: Baseline and every month for 6 months]

Secondary ID(s)

PO2060

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Roche Pharma AG

National Institute for Health Research, United Kingdom