Sildenafil for Early Pulmonary Vascular Disease in Scleroderma

NCT ID: NCT04797286

Last Updated: 2025-10-15

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-09-20
• Study Completion Date: 2026-09-01

Brief Summary

This is a Phase II randomized, double-blind, placebo-controlled trial of sildenafil in men and women with Scleroderma with mildly elevated pulmonary pressures (SSc-MEP) to determine whether sildenafil may be an effective treatment for SSc-MEP.

Detailed Description

Data shows that sildenafil (SIL) is an effective therapy in SSc-PAH. SIL has been safely used in many patients with various vascular and cardiovascular diseases over the past three decades. Randomized controlled trial data for SIL shows improvement in 6MWD, hemodynamics, and even evidence of cardiac remodeling in PAH and SSc-PAH patients. Based upon these data, SIL may be an effective therapy in SSc-MEP. This study will help determine whether sildenafil could be a good treatment for patients with scleroderma that have mildly elevated pulmonary pressures.

Conditions

Scleroderma; Mildly Elevated Pulmonary Pressures

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Sildenafil

Sildenafil 20 mg by mouth three(3) times each day

Group Type: EXPERIMENTAL

Sildenafil

Intervention Type: DRUG

Sildenafil 20 mg three times a day. This is the approved dose for the treatment of pulmonary arterial hypertension. It is being studied in this trial with a population who has mildly elevated pulmonary pressures.

Placebo

Placebo by mouth three(3) times each day

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Oral pill placebo.

Interventions

Sildenafil

Sildenafil 20 mg three times a day. This is the approved dose for the treatment of pulmonary arterial hypertension. It is being studied in this trial with a population who has mildly elevated pulmonary pressures.

Intervention Type: DRUG

Placebo

Oral pill placebo.

Intervention Type: OTHER

Other Intervention Names

Revatio, Viagra

Eligibility Criteria

Inclusion Criteria

• Previous documentation of mean pulmonary artery pressure between 21 and 24 mm Hg with a pulmonary capillary wedge pressure (or left ventricular end-diastolic pressure) ≤ 15 mm Hg within six months before study entry.
• Diagnosis of SSc according to 2013 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) classification criteria.
• Pulmonary function tests with forced expiratory volume in one second/forced vital capacity (FEV1/FVC) >50% AND either a) total lung capacity (TLC) or forced vital capacity (FVC) > 70% predicted or b) TLC or FVC between 60% and 70% predicted with no more than mild interstitial lung disease on computerized tomography scan of the chest on studies obtained within 6 months of enrollment.
• Ventilation perfusion scan or computed tomography with intravenous contrast (CT angiogram) without evidence of chronic thromboembolism at anytime before study entry.
• Ability to perform six minute walk testing without significant limitations in musculoskeletal function or coordination.
• Informed consent.

Exclusion Criteria

• World Health Organization (WHO) Class IV functional status.
• Systolic blood pressure less than 90 mmHg at screening visit prior to enrollment.
• Clinically significant untreated sleep apnea.
• Left-sided valvular disease (more than moderate mitral valve stenosis or insufficiency or aortic stenosis or insufficiency), pulmonary artery or valve stenosis, or ejection fraction < 45% on most recent echocardiography (within 1 year).
• Use of Pulmonary Arterial Hypertension (PAH) therapy (prostacyclin analogues, endothelin-1 receptor antagonists,phosphodiesterase-5 inhibitors, riociguat, selexipag) within the past 3 months.
• Hospitalized or acutely ill.
• Renal failure (creatinine above 2.0) at screening visit.
• Enrollment in a clinical trial or concurrent use of another investigational drug (non FDA approved) or device therapy within 30 days of screening visit.
• Age < 18.
• Currently pregnant.
• Current use of nitrates.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Louisiana State University Health Sciences Center in New Orleans

OTHER

Sponsor Role: collaborator

United States Department of Defense

FED

Sponsor Role: collaborator

Johns Hopkins University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Stephen Mathai, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Locations

Louisiana State University

New Orleans, Louisiana, United States

Site Status: NOT_YET_RECRUITING

Johns Hopkins

Baltimore, Maryland, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Stephen Mathai, MD

Role: CONTACT

smathai4@jhmi.edu

4106146311

Renee Ofori

Role: CONTACT

rofori2@jhu.edu

410-614-6311

Facility Contacts

Matthew Lammi, MD

Role: primary

504-568-4634

Marie Sandi

Role: backup

mchild@lsuhsc.edu

Stephen Mathai, MD

Role: primary

smathai4@jhmi.edu

410-614-6311

Dezeray Dutton

Role: backup

dcephas1@jhu.edu

4435078222

References

Lammi MR, Mukherjee M, Saketkoo LA, Carey K, Hummers L, Hsu S, Krishnan A, Sandi M, Shah AA, Zimmerman SL, Hassoun PM, Mathai SC. Sildenafil Versus Placebo for Early Pulmonary Vascular Disease in Scleroderma (SEPVADIS): protocol for a randomized controlled trial. BMC Pulm Med. 2024 Apr 30;24(1):211. doi: 10.1186/s12890-024-02892-3.

Reference Type: DERIVED

PMID: 38689245 (View on PubMed)

Other Identifiers

IRB00265164

Identifier Type: -

Identifier Source: org_study_id