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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

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Overview

Principal Investigator: Harrison Farber, MD

This is a Phase 3, randomized, double-blinded, 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. The objective of this study is to evaluate the efficacy and safety of sotatercept treatment (plus background PAH therapy) versus placebo (plus background PAH therapy) at 24 weeks in adults with PAH. Participants enrolled in the study will have a diagnosis of PAH (Pulmonary Hypertension WHO Group 1) associated with idiopathic/heritable, drug-induced, connective tissue diseases (CTD),or post shunt correction PAH, within WHO FC II or III. Approximately 284 participants will be randomly assigned in a 1:1 ratio to the two study treatment groups (142 participants per arm).

Study details

Inclusion Criteria
  • Age ≥ 18 years
  • Symptomatic PAH classified as WHO Functional Class II or III
  • Baseline RHC performed during the Screening Period documenting a minimum pulmonary vascular resistance (PVR) of ≥ 5 Wood units (WU)
Exclusion Criteria
  • Diagnosis of pulmonary hypertension WHO Groups 2, 3, 4, or 5
  • Diagnosis of the following PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH and PAH associated with portal hypertension. Exclusions in PAH Group I should also include schistosomiasis APAH and pulmonary veno-occlusive disease
  • Hemoglobin (Hgb) at screening above gender-specific upper limit of normal (ULN), per local laboratory test
Study Requirements

Patients will be enrolled for approximately 108 weeks. Patients will come in for 9 visits over 24 weeks and then will rollover into the Long Term Follow-Up study. If a patient is unable to have a Pulmonary Function Test, then he/she will have a CT scan at Screening. A total of approximately 1 tablespoon of blood will be collected at screening. If a patient is eligible and participates in the study, a total of approximately 13 tablespoons will be collected if a patient completes treatment from Visit 1 through Visit 9. If a patient goes beyond Visit 9, a total of approximately 35 tablespoons of blood may be collected with about 3 tablespoons collected at each visit.

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