STRATOSPHERE
Research type
Research Study
Full title
Optimal biomarkers to ascertain target engagement in therapies targeting the BMPR2 pathway in Pulmonary Arterial Hypertension (PAH)
IRAS ID
257956
Contact name
Carolyn Read
Contact email
Sponsor organisation
University of Cambridge
Duration of Study in the UK
2 years, 0 months, 3 days
Research summary
Pulmonary Arterial Hypertension is a progressive disease that has no cure. Patients die young and are limited in their daily activity. Current treatments only treat the symptoms of the disease rather than the underlying cause. At least 1 in 5 patients has a change in a gene called the bone morphogenetic type 2 protein (or BMPR2). Extensive evidence supports the concept of addressing the reduced levels of the BMPR2 protein to reverse disease. Through work already undertaken by this group, two potential therapies which increase BMPR2 have been identified for use in a future randomised control trial. In order for a clinical trial to be informative we need an accurate way of measuring the protein or the effects of the protein (known as a biomarker).
This study will use blood samples taken from 17 patients and 30 healthy participants over various time-points (2-5 visits over 5 weeks for healthy controls; 2 visits, approximately four months apart for patients). Laboratory work will help identify the best biomarkers for subsequent therapy studies. By defining the best biomarkers we can speed up the drug development in this rare disease.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
21/EE/0043
Date of REC Opinion
16 Feb 2021
REC opinion
Favourable Opinion