NCT04797286

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
4mo left

Started Sep 2021

Longer than P75 for phase_2

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress94%
Sep 2021Sep 2026

First Submitted

Initial submission to the registry

March 11, 2021

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 15, 2021

Completed
6 months until next milestone

Study Start

First participant enrolled

September 20, 2021

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2026

Last Updated

October 15, 2025

Status Verified

October 1, 2025

Enrollment Period

5 years

First QC Date

March 11, 2021

Last Update Submit

October 13, 2025

Conditions

Keywords

SSc-MEP

Outcome Measures

Primary Outcomes (1)

  • Difference in change in distance walked in 6 minute walk test (6MWT) at 4 months

    As assessed by change in 6 minute walk distance (6MWD) in feet (from baseline to 4 months) between the sildenafil group and the placebo group.

    Baseline and 4 months

Secondary Outcomes (12)

  • Difference in change in distance walked in 6MWT at 12 months

    Baseline and 12 months

  • Difference in change in right ventricular function as assessed by cardiac MRI

    Baseline and 4 months

  • Difference in change in right ventricular function as assessed by cardiac MRI

    Baseline and 12 months

  • Difference in change in right ventricular function as assessed by invasive hemodynamics

    Baseline and 4 months

  • Difference in change in right ventricular function as assessed by invasive hemodynamics

    Baseline and 12 months

  • +7 more secondary outcomes

Study Arms (2)

Sildenafil

EXPERIMENTAL

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

Drug: Sildenafil

Placebo

PLACEBO COMPARATOR

Placebo by mouth three(3) times each day

Other: Placebo

Interventions

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.

Also known as: Revatio, Viagra
Sildenafil
PlaceboOTHER

Oral pill placebo.

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Louisiana State University

New Orleans, Louisiana, 70806, United States

NOT YET RECRUITING

Johns Hopkins

Baltimore, Maryland, 21287, United States

RECRUITING

Related Publications (1)

  • 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.

MeSH Terms

Conditions

Scleroderma, Diffuse

Interventions

Sildenafil Citrate

Condition Hierarchy (Ancestors)

Scleroderma, SystemicConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Intervention Hierarchy (Ancestors)

SulfonamidesAmidesOrganic ChemicalsSulfonesSulfur CompoundsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Study Officials

  • Stephen Mathai, MD

    Johns Hopkins University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephen Mathai, MD

CONTACT

Renee Ofori

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 11, 2021

First Posted

March 15, 2021

Study Start

September 20, 2021

Primary Completion (Estimated)

September 1, 2026

Study Completion (Estimated)

September 1, 2026

Last Updated

October 15, 2025

Record last verified: 2025-10

Data Sharing

IPD Sharing
Will not share

Locations