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

Authors

Matthew R. Lammi, LSU Health Sciences Center - New OrleansFollow
Monica Mukherjee, Johns Hopkins University, Baltimore, MA
Lesley Ann Saketkoo, New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center, New Orleans, LA
Kyle Carey, Johns Hopkins University, Baltimore, MA
Laura Hummers, Johns Hopkins University, Baltimore, MA
Steven Hsu, Johns Hopkins University, Baltimore, MA
Amita Krishnan, LSU Health Sciences Center - New OrleansFollow
Marie Sandi, LSU Health Sciences Center - New OrleansFollow
Ami A. Shah, Johns Hopkins University, Baltimore, MA
Stefan L. Zimmerman, Johns Hopkins University, Baltimore, MA
Paul M. Hassoun, Johns Hopkins University, Baltimore, MA
Steven C. Mathai, Johns Hopkins University, Baltimore, MA

Document Type

Article

Publication Date

4-30-2024

Publication Title

BMC Pulmonary Medicine

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.

First Page

211

PubMed ID

38689245

Volume

24

Issue

1

Creative Commons License

This work is licensed under a Creative Commons Attribution 4.0 International License.

Recommended Citation

Lammi, Matthew R.; Mukherjee, Monica; Saketkoo, Lesley Ann; Carey, Kyle; Hummers, Laura; Hsu, Steven; Krishnan, Amita; Sandi, Marie; Shah, Ami A.; Zimmerman, Stefan L.; Hassoun, Paul M.; and Mathai, Steven C., "Sildenafil Versus Placebo for Early Pulmonary Vascular Disease in Scleroderma (SEPVADIS): protocol for a randomized controlled trial" (2024). School of Medicine Faculty Publications. 2600.
https://digitalscholar.lsuhsc.edu/som_facpubs/2600
10.1186/s12890-024-02892-3