CWoW-A Phase 3 Study of Sotatercept for the Treatment of PAH
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare the Efficacy and Safety of Sotatercept Versus Placebo When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy for the Treatment of PAH
Acceleron Pharma Inc.
This is a clinical research study of a new treatment called Sotatercept versus Placebo given in combination with background Pulmonary Arterial Hypertension (PAH) therapy for the treatment of PAH. PAH applies to a group of diseases causing a progressive increase in pulmonary vascular resistance (PVR), resulting in right ventricular dysfunction and ultimately failure as well as premature death. PAH is a progressive, fatal disease that causes marked limitations in physical activity and quality of life, even when treated with approved therapies. This Phase 3 study is supported by data from the PULSAR study (Phase 2, NCT03496207), in which participants taking any approved single or combination therapy for PAH were randomised to receive additional Sotatercept or placebo for 24 weeks. The PULSAR study demonstrated a statistically significant improvement in its primary endpoint, Pulmonary Vascular Resistance (PVR). The study duration for a given participant will be approx.. 108 weeks with a Screening Period, Double Blind Placebo-Controlled Treatment Period , A Long Term Double-Blind (LTDB) Treatment Period and a Follow Up Period.
Each study eligible participant (Approximately 284 participants) will be randomly assigned in a 1:1 ratio to 1 of the 2 treatment arms (142 Participants/arm) for the duration of the DBPC and LTDB Treatment Periods.
• Arm 1: Placebo administered subcutaneously (SC) every 21 days plus background PAH therapy
• Arm 2: Sotatercept at a starting dose of 0.3 mg/kg with a target dose of 0.7 mg/kg administered subcutaneously (SC) every 21 days plus background PAH therapy.
The study involves procedures including physical examinations, vital signs, ECG, Assessment of heart pumping rate and measurement of pressure in heart and lungs via a Right Heart Catheterisation process, Six Minute Walk Test (6MWT) , pulmonary function test, if required a chest CT-scan maybe performed and an Echocardiogram. This study is sponsored by Acceleron Pharma.
South West - Central Bristol Research Ethics Committee
Date of REC Opinion
23 Mar 2021
Further Information Favourable Opinion