Dados do Trabalho

Título

TRIAL IN PROGRESS: PROSERA, A PHASE 3 STUDY OF THE EFFICACY AND SAFETY OF SERALUTINIB IN ADULTS WITH PULMONARY ARTERIAL HYPERTENSION (PAH)

Introdução

Seralutinib is a highly potent tyrosine kinase inhibitor (TKI) targeting PDGFRα/ß, CSF1R, and c-KIT pathways that drive PAH pathology. Seralutinib is the first TKI specifically formulated for inhalation for deep lung deposition and reduced systemic exposure. The phase 2 TORREY study of seralutinib in patients with PAH on standard of care therapy met its primary endpoint of significant reduction in pulmonary vascular resistance (PVR), with favorable tolerability (NCT04456998). Significant improvements in NT-proBNP and right heart function by echocardiography were observed.

Objetivo

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Método

PROSERA is a global phase 3, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of inhaled seralutinib in adults with WHO Group 1 Pulmonary Hypertension, Functional Class (FC) II/III, PVR ≥400 dyne*s/cm5, 6-minute walk distance (6MWD) 150–475 m, either REVEAL Lite 2 Risk Score ≥5 or NT-proBNP ≥300 ng/L or PVR ≥800 dyne*s/cm5, and on at least one stable PAH background therapy, including parenteral prostacyclins (NCT05934526). 350 patients will be randomized to inhaled seralutinib 90 mg or placebo by dry powder inhaler BID for ≤48 weeks. The primary endpoint is 6MWD change from baseline (BL) to Week 24 (W24). Key secondary endpoints (from BL) are time to first clinical worsening event through Week 48 (W48), proportion of patients with clinical improvement (W24), change in NT-proBNP (W24) and proportion of patients with ≥1-point decrease in REVEAL Lite 2 Risk Score (W24). Other secondary endpoints (from BL) include proportion of patients with each clinical worsening outcome (through W48), proportion of patients who improve in WHO FC or maintain WHO FC II (through W48) and change in health-related quality of life (PAH-SYMPACT, EQ-5D-5L; W24). Patients completing the study on blinded treatment may be eligible to enroll in a separate open-label extension study. A functional respiratory imaging substudy will examine seralutinib’s potential effect on pulmonary vascular remodeling.

Resultados

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Conclusão

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