A Phase3 Study of Sotatercept in Participants with PAH WHO FC III or FC IV at High Risk of Mortality

  • Research type

    Research Study

  • Full title

    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sotatercept When Added to Maximum Tolerated Background Therapy in Participants With Pulmonary Arterial Hypertension (PAH) World Health Organization (WHO) Functional Class (FC) III or FC IV at High Risk of Mortality

  • IRAS ID

    1004073

  • Contact name

    SaraBeth Hahn

  • Contact email

    shahn@acceleronpharma.com

  • Sponsor organisation

    Acceleron Pharma Inc.

  • Eudract number

    2021-001498-21

  • Clinicaltrials.gov Identifier

    NCT04896008

  • Research summary

    PAH leads to increased pressure in the blood vessels of the lungs which further leads to straining of the right side of the heart. This study is for Patients diagnosed with PAH who have been determined to fall under the following two, the WHO categories and therefore require treatment for their PAH:
    • Class III: They’re comfortable at rest but have symptoms when they’re physically active which noticeably limits their physical activity.
    • Class IV: They may suffer from symptoms when at rest and will suffer from severe symptoms when doing any activity.
    Sotatercept is a new medicine that is believed to decrease the wall thickness of blood vessels in the lung. By doing so, it allows more blood into the lung blood vessels and therefore may help improve shortness of breath and increase exercise capacity.
    In this study, participants will be randomly assigned to receive sotatercept or placebo in combination with their regular PAH medication(s).
    Participant will be randomly assigned in a 1:1 ratio to one of the 2 treatment arms prior to starting the Double Blind Placebo-Controlled Treatment Period.
    • Arm 1: Placebo administered subcutaneously (SC - injection) every 21 days plus back ground PAH therapy
    • Arm 2: Sotatercept at a starting dose of 0.3 mg/kg, with a target dose of 0.7 mg/kg, administered SC (injection) every 21 days plus background PAH therapy
    Participation is for up to 49 months which includes a screening period, treatment period and Follow-up period. Participant will be asked to visit the site every 21 days. The sponsor aims to randomise 166 participants globally on this study in a 1:1 ratio to the 2study treatment groups (83 per group) and 12 participants will be in the UK. The maximum amount of blood per year on this study is112ml, and t procedures such as Right Heart Caterisation (RHC) , Electrocardiogram (ECG),Echocardiogram (ECHO), physical examinations, blood and urine tests to mention a few.

  • REC name

    London - Riverside Research Ethics Committee

  • REC reference

    21/LO/0704

  • Date of REC Opinion

    20 Jan 2022

  • REC opinion

    Further Information Favourable Opinion