StratosPHere (Non-interventional Study)

NCT05767918 | Completed

• 1 other identifier: P02739
• Study Type: observational
• Target: 65
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Pulmonary Arterial Hypertension; Idiopathic Pulmonary Arterial Hypertension; Pulmonary Hypertension; Respiratory Disease

Keywords

Bone morphogenetic type 2 protein; BMPR2; Biomarker

Outcome Measures

Primary Outcomes (1)

• Biomarker(s) identification

  identify one or more biomarkers of increased BMPR2 expression or BMPR2 target engagement that fit a criterion of being reproducible and repeatable. We will define the acceptable level of variability of these in order to be used in the subsequent phase 2a trial that will be powered to detect a 30% increase in BMPR2, and or BMPR2 target engagement with an 80% power in the known patient population.

  2 years

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

PAH patients and healthy controls

You may qualify if:

• Healthy volunteers
• Individual must be 18 years of age or over
• Weigh \> 40kg at screening visit / BMI under 35
• Healthy and well at each visit
• PAH participants
• Must have a diagnosis of IPAH or HPAH with a WHO functional class I-IV
• Must be on stable or unchanged PAH therapeutics for at least one month prior to screening visit

You may not qualify if:

• Health volunteers
• Known hepatitis B, HIV or tuberculosis
• Clinically severe anaemia or bleeding disorders
• PAH participants
• Other forms of PH besides IPAH or HPAH
• Patients on TNF antagonists of other biological treatments
• Known hepatitis B, HIV or tuberculosis
• Clinically severe anaemia or bleeding disorders
• Female participants who are pregnant or breast feeding

Sponsors & Collaborators

• Papworth Hospital NHS Foundation Trust: lead
• Medical Research Council: collaborator
• University of Cambridge: collaborator

Study Sites (1)

University of Cambridge Heart and Lung Research Institute

Cambridge, England, CB2 0QQ, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum and plasma samples - Peripheral blood mononuclear cells (PBMCs) were isolated from whole blood samples and the live cells cryogenically frozen pending analysis. Blood samples destined for RNA analysis were collected into Tempus Blood RNA tubes and stored at -80˚C pending RNA extraction and further analysis.

MeSH Terms

Conditions

Pulmonary Arterial Hypertension; Familial Primary Pulmonary Hypertension; Hypertension, Pulmonary; Respiration Disorders

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Mark Toshner Associate Professor MD FRCP

  University of Cambridge

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 2, 2023
• First Posted: March 14, 2023
• Study Start: April 14, 2021
• Primary Completion: May 1, 2022
• Study Completion: September 30, 2022
• Last Updated: March 14, 2023

Record last verified: 2023-03

Locations

GB (1)