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

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

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Overview
Principal Investigator: Harrison Farber, MD

This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate sotatercept when added to background PAH therapy in newly diagnosed intermediate- and high-risk PAH patients.

Participants enrolled in the study will have a diagnosis within 6 months of study screening of symptomatic PAH (WHO Group 1, classified as FC II or III) and presentation of idiopathic or heritable PAH, PAH associated with connective tissue diseases (CTD), drug- or toxin- induced PAH, after shunt correction PAH, or PAH presenting at least 1 year following the correction of congenital heart defects. The objective of this study is to evaluate the effects of sotatercept treatment (plus background PAH therapy) versus placebo (plus background PAH therapy) on time to clinical worsening (TTCW) in participants who are newly diagnosed with PAH and are at intermediate or high risk of disease progression.

Study details
Inclusion Criteria
  • Symptomatic PAH classified as WHO FC II or III
  • REVEAL Lite 2 risk score ≥ 6
  • Six-minute walk distance ≥ 150 m repeated twice at screening at least 4 hours apart, but no longer than 1 week apart, and both values are within 15% of each other (calculated from the highest value)
Exclusion Criteria
  • Hemoglobin at screening above gender-specific ULN, per local laboratory test
  • Baseline systolic BP < 90 mmHg at screening
  • History of pneumonectomy
Study Requirements

Patients will participate in this study for up to 56 months (approximately 4 to 5 years) including the follow-up period. The first 9 visits will be at clinic and then patients have option to do at home health visits. Blood will be drawn within 4 weeks prior to the first visit, 4 times in the first 6 months on study and then about every 3 months and at the Follow-up Visit. An estimated amount of 4.5 teaspoons/22 mL of blood will be collected at screening. An estimated amount of 12 teaspoons/58 mL of blood will be collected at visit 1. For those visits where blood is collected after visit 1, an estimated amount of 5.5 teaspoons/26.5 mL will be collected. It’s expected on average the total amount of blood collected per year is 27 teaspoons/132.5 mL.