Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension

NCT ID: NCT03814317

Last Updated: 2025-06-30

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-30
• Study Completion Date: 2026-07-29

Brief Summary

This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.

Detailed Description

Pulmonary sarcoidosis-associated pulmonary hypertension is classified as WHO Group 5 pulmonary hypertension and may occur in anywhere from 5-20% of sarcoidosis patients. Inhaled treprostinil has shown clinical improvements in exercise capacity after 12 weeks of therapy in patients with WHO Group 1 pulmonary hypertension. More recently, there has been interest in using inhaled PAH-specific therapies for the treatment of pulmonary hypertension associated with interstitial lung disease.

The investigators believe that those patients with pulmonary hypertension in the setting of sarcoidosis-associated interstitial lung disease are a unique population which may potentially benefit from inhaled, targeted pulmonary arterial hypertension therapy (inhaled treprostinil) while minimizing the adverse effects associated with systemic pulmonary vasodilators. This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.

Conditions

Sarcoidosis; Precapillary Pulmonary Hypertension; Interstitial Lung Disease

Keywords

right heart catheterization (RHC); pulmonary vascular resistance (PVR); mean pulmonary arterial pressure (mPAP); pulmonary hypertension (PH); Inhaled treprostinil; Sarcoidosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Study Group

Sarcoidosis patients with interstitial lung disease and precapillary pulmonary hypertension based on right heart catheterization (RHC).

All subjects will initiate inhaled treprostinil at a dose of 3 breaths (18 mcg) four times daily. Study drug doses escalations (additional one breath four times daily) can occur every three days with a maximum dosing regimen of up to 12 breaths (72 mcg) four times daily, as clinically tolerated.

Group Type: EXPERIMENTAL

Inhaled Treprostinil

Intervention Type: DRUG

Inhaled treprostinil causes dilatation of the pulmonary arteries and may help reduce the pulmonary pressures in this studied population.

All subjects will initiate inhaled treprostinil at a dose of 3 breaths (18 mcg) four times daily. Study drug doses escalations (additional one breath four times daily) can occur every three days with a maximum dosing regimen of up to 12 breaths (72 mcg) four times daily, as clinically tolerated.

Interventions

Inhaled Treprostinil

Inhaled treprostinil causes dilatation of the pulmonary arteries and may help reduce the pulmonary pressures in this studied population.

All subjects will initiate inhaled treprostinil at a dose of 3 breaths (18 mcg) four times daily. Study drug doses escalations (additional one breath four times daily) can occur every three days with a maximum dosing regimen of up to 12 breaths (72 mcg) four times daily, as clinically tolerated.

Intervention Type: DRUG

Other Intervention Names

Tyvaso®; pulmonary arterial hypertension (PAH) therapy

Eligibility Criteria

Inclusion Criteria

• Study participant willing and able to provide informed consent
• Negative urine pregnancy test at baseline for females of childbearing potential
• Established diagnosis of sarcoidosis by ATS/ERS/WASOG 1999 Statement on of Sarcoidosis
• Presence of interstitial lung disease by Scadding Stage IV chest radiograph or extensive fibrosis on chest computed tomography
• Right heart catheterization within six months of baseline visit showing precapillary pulmonary hypertension (mPAP ≥ 25 mmHg, PCWP ≤ 15 mmHg, and PVR > 3 WU)
• Patient on stable sarcoidosis therapy for at least three months prior to screening
• If patients are on oral PAH therapy (PDE5i/SCGS and/or ERA) then dose should be stable for at least three months prior to screening
• A 6MWT within three months of screening visit of > 100 meters

Exclusion Criteria

• Pregnant patients or those who are actively lactating
• Patient not willing to use form of birth control (if applicable) during the study
• Inability to undergo 6MWT, RHC, PFTs or CMRI
• Predicted survival < 6 months
• Patient on any prostanoid or prostanoid analog therapy
• Patients with left sided heart disease as defined by either a PCWP > 15 mmHg and/or left ventricular ejection fraction < 40%
• Use of any investigational drug/device, or participation in any investigational study with therapeutic intent within 30 days prior to randomization.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

United Therapeutics

INDUSTRY

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ali Ataya, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

University of Florida, Division of Pulmonary and Critical Care Medicine

Gainesville, Florida, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Duncan R Lewis, BS

Role: CONTACT

Phone: 352-294-5195

Email: duncanlewis@ufl.edu

Ali Ataya, MD

Role: CONTACT

Phone: 352-273-8740

Email: ali.ataya@medicine.ufl.edu

Facility Contacts

Duncan R Lewis, BS

Role: primary

Other Identifiers

20192572

Identifier Type: OTHER

Identifier Source: secondary_id

OCR19684

Identifier Type: -

Identifier Source: org_study_id