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.

• IRAS ID

  1003773

• Contact name

  SaraBeth Hahn

• Contact email

  shahn@acceleronpharma.com

• Sponsor organisation

  Acceleron Pharma Inc.

• Eudract number

  2021-000199-12

• Clinicaltrials.gov Identifier

  NCT04811092

• Research summary

  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.

  Summary of results

  Researchers learned that compared to people who received placebo with standard treatment, people who received sotatercept with their standard treatment had a longer time between starting the trial and having any of these happen:
  • A hospital stay for more than one day because PAH got worse
  • A heart surgery to help blood flow better and lessen symptoms of PAH
  • A lung transplant
  • A shorter 6MWD and other signs that PAH got worse
  • They were no longer alive Researchers looked at any adverse reactions participants had during this trial. Of the 320 participants who were treated during the trial:
  • 5 participants (1.6%) had a serious adverse reaction
  • 1 participant (0.3%) stopped trial medicine because of a serious adverse reaction
  • No one died during the trial because of a serious adverse reaction
  

• REC name

  London - Chelsea Research Ethics Committee

• REC reference

  21/LO/0648

• Date of REC Opinion

  28 Oct 2021

• REC opinion

  Further Information Favourable Opinion