Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension

NCT03814317 | Completed Phase 2 DMC FDA Drug

• 2 other identifiers: OCR1968420192572
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Sarcoidosis; Interstitial Lung Disease; Precapillary Pulmonary Hypertension

Keywords

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

Outcome Measures

Primary Outcomes (2)

• PVR by Right heart catheterization (RHC)

  Change in RHC parameter PVR (pulmonary vascular resistance )

  Baseline, Week 16

• mPAP by Right heart catheterization (RHC)

  Change in RHC parameter mPAP (mean pulmonary arterial pressure)

  Baseline, Week 16

Secondary Outcomes (5)

• Change in 6-Minute Walk Test (6MWT)

  Baseline, Week 8, Week 16

• Change in Cardiac MRI parameters

  Baseline, Week 16

• Change in Pulmonary Function Testing

  Baseline, Week 16

• Change in Brain Natriuretic Peptide (BNP)

  Baseline, Week 16

• Change in WHO Functional Class (WHO FC)

  Baseline, Week 8, Week 16

Study Arms (1)

Study Group

EXPERIMENTAL

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.

Drug: Inhaled Treprostinil

Interventions

Inhaled Treprostinil 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.

Also known as: Tyvaso®, pulmonary arterial hypertension (PAH) therapy

Study Group

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Florida: lead
• United Therapeutics: collaborator

Study Sites (1)

University of Florida, Division of Pulmonary and Critical Care Medicine

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Sarcoidosis; Lung Diseases, Interstitial; Hypertension, Pulmonary

Interventions

treprostinil; hydralazine 4-anisaldehyde hydrazone; Therapeutics

Condition Hierarchy (Ancestors)

Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases; Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Ali Ataya, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: January 4, 2019
• First Posted: January 24, 2019
• Study Start: January 30, 2020
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: February 6, 2026

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)