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StratosPHere 2 trial of hydroxychloroquine and phenylbutyrate in BMPR2-associated pulmonary arterial hypertension

ISRCTN10304915Phase 2Recruiting

Sponsor
Papworth Hospital NHS Foundation Trust
Start
Dec 2023
Modality
Small Molecules
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Overview

Phase 2, randomised, double-blind trial in pulmonary arterial hypertension (PAH) and Heritable Pulmonary Arterial Hypertension (HPAH) comparing Standard of care + hydroxychloroquine, Standard of care + phenylbutyrate and Standard of care + placebo (3 arms). Primary endpoint: Target engagement of the BMPR2 pathway. Started Dec 2023; status: recruiting.

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Drugs

hydroxychloroquinesodium phenylbutyrate
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Conditions

pulmonary arterial hypertension (PAH)Heritable Pulmonary Arterial Hypertension (HPAH)

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