Using sildenafil to treat early pulmonary vascular disease in scleroderma

Sildenafil for Early Pulmonary Vascular Disease in Scleroderma

This study is testing if sildenafil can help people with scleroderma and mildly high lung pressure feel better and improve their lung function.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Johns Hopkins University (Academic / other)
• Locations: 2 sites (New Orleans, Louisiana and 1 other locations)
• Trial ID: NCT04797286 on ClinicalTrials.gov

What this trial studies

This Phase II trial investigates the effectiveness of sildenafil in treating patients with scleroderma who have mildly elevated pulmonary pressures. The study is randomized, double-blind, and placebo-controlled, aiming to assess improvements in pulmonary function and overall health. Participants will receive either sildenafil or a placebo, and their responses will be monitored through various clinical assessments. The goal is to determine if sildenafil can provide significant benefits for this specific patient population.

Who should consider this trial

Good fit: Ideal candidates include individuals diagnosed with scleroderma who have documented mildly elevated pulmonary artery pressures.

Not a fit: Patients with severe pulmonary hypertension or significant interstitial lung disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve pulmonary function and quality of life for patients with scleroderma and mildly elevated pulmonary pressures.

How similar studies have performed: Previous studies have shown sildenafil to be effective in treating pulmonary arterial hypertension, suggesting potential success in this novel application.

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.

Where this trial is running

New Orleans, Louisiana and 1 other locations

• Louisiana State University — New Orleans, Louisiana, United States (Not_yet_recruiting)
• Johns Hopkins — Baltimore, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: Stephen Mathai, MD — Johns Hopkins University
• Study coordinator: Stephen Mathai, MD

Conditions Scleroderma; Mildly Elevated Pulmonary Pressures; SSc-MEP