StratosPHere 2

• Research type

  Research Study

• Full title

  StratosPHere 2: A response-adaptive randomised placebo controlled Phase II trial to evaluate hydroxychloroquine and phenylbutyrate in Pulmonary arterial Hypertension caused by mutations in BMPR2

• IRAS ID

  1003631

• Contact name

  Mark Toshner

• Contact email

  mrt34@medschl.cam.ac.uk

• Sponsor organisation

  Royal Papworth Hospital NHS Foundation Trust

• Research summary

  Pulmonary Arterial Hypertension (PAH) is a progressive respiratory condition that causes high blood pressure in the blood vessels that supply blood to the lungs. Current treatments help to manage the symptoms of the disease but do not treat the underlying cause.
  The condition can occur in families due to an alteration in a gene (piece of inherited material) called the bone morphogenetic protein type 2 receptor (BMPR2). Currently there are no therapies that target BMPR2 dysfunction. In a previous study we identified a set of proteins or biological markers (biomarkers) in the blood that regulate BMPR2. In this study we will be looking at two drug therapies Hydroxychloquine and Glycerol Phenylbutyrate, both of which can affect BMPR2 to see if improving the function of BMPR2 is a potential therapy area in PAH.

• REC name

  London - Surrey Borders Research Ethics Committee

• REC reference

  23/LO/0285

• Date of REC Opinion

  14 Sep 2023

• REC opinion

  Further Information Favourable Opinion