A Phase 3 Study of Sotatercept in Newly Diagnosed Intermediate- and High-risk PAH Patients

  • Research type

    Research Study

  • Full title

    A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate Sotatercept When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy in Newly Diagnosed Intermediate- and High-risk PAH Patients.



  • Contact name

    SaraBeth Hahn

  • Contact email


  • Sponsor organisation

    Acceleron Pharma Inc.

  • Eudract number


  • Clinicaltrials.gov Identifier


  • Research summary

    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. 56 months with a Screening Period, Double Blind Placebo-Controlled Treatment Period and a Follow Up Period.
    Each study eligible participant (Up to 662 participants) will be randomly assigned in a 1:1 ratio to 1 of the 2 treatment arms (up to 331 Participants/arm) for the duration of the DBPC 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, Six Minute Walk Test (6MWT), pulmonary function test and an Echocardiogram. This study is sponsored by Acceleron Pharma.

  • REC name

    London - Chelsea Research Ethics Committee

  • REC reference


  • Date of REC Opinion

    28 Oct 2021

  • REC opinion

    Further Information Favourable Opinion

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