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BMC Pulmonary Medicine
Published in: BMC Pulmonary Medicine 1/2024

Open Access 01-12-2024 | Sildenafil | Study Protocol

Sildenafil Versus Placebo for Early Pulmonary Vascular Disease in Scleroderma (SEPVADIS): protocol for a randomized controlled trial

Authors: Matthew R. Lammi, Monica Mukherjee, Lesley Ann Saketkoo, Kyle Carey, Laura Hummers, Steven Hsu, Amita Krishnan, Marie Sandi, Ami A. Shah, Stefan L. Zimmerman, Paul M. Hassoun, Steven C. Mathai

Published in: BMC Pulmonary Medicine | Issue 1/2024

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Abstract

Background

Pulmonary hypertension (PH) is a leading cause of death in patients with systemic sclerosis (SSc). An important component of SSc patient management is early detection and treatment of PH. Recently the threshold for the diagnosis of PH has been lowered to a mean pulmonary artery pressure (mPAP) threshold of > 20 mmHg on right heart catheterization (RHC). However, it is unknown if PH-specific therapy is beneficial in SSc patients with mildly elevated pressure (SSc-MEP, mPAP 21–24 mmHg).

Methods

The SEPVADIS trial is a randomized, double-blind, placebo-controlled phase 2 trial of sildenafil in SSc-MEP patients with a target enrollment of 30 patients from two academic sites in the United States. The primary outcome is change in six-minute walk distance after 16 weeks of treatment. Secondary endpoints include change in pulmonary arterial compliance by RHC and right ventricular function by cardiac magnetic resonance imaging at 16 weeks. Echocardiography, serum N-terminal probrain natriuretic peptide, and health-related quality of life is being measured at 16 and 52 weeks.

Discussion

The SEPVADIS trial will be the first randomized study of sildenafil in SSc-MEP patients. The results of this trial will be used to inform a phase 3 study to investigate the efficacy of treating patients with mild elevations in mPAP.

Trial registration

ClinicalTrials.gov Identifier NCT04797286.
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Metadata
Title
Sildenafil Versus Placebo for Early Pulmonary Vascular Disease in Scleroderma (SEPVADIS): protocol for a randomized controlled trial
Authors
Matthew R. Lammi
Monica Mukherjee
Lesley Ann Saketkoo
Kyle Carey
Laura Hummers
Steven Hsu
Amita Krishnan
Marie Sandi
Ami A. Shah
Stefan L. Zimmerman
Paul M. Hassoun
Steven C. Mathai
Publication date
01-12-2024
Publisher
BioMed Central
Published in
BMC Pulmonary Medicine / Issue 1/2024
Electronic ISSN: 1471-2466
DOI
https://doi.org/10.1186/s12890-024-02892-3

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