Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension (SAPPHIRE)

May 17, 2023 updated by: University of Florida

An Open-Label Study of Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension and Interstitial Lung Disease (SAPPHIRE)

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

Study Overview

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.

Study Type

Interventional

Enrollment (Anticipated)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

    • Florida
      • Gainesville, Florida, United States, 32610
        • Recruiting
        • University of Florida, Division of Pulmonary and Critical Care Medicine
        • Contact:
          • Christina M Eagan, DNP
        • Principal Investigator:
          • Ali Ataya, MD
        • Sub-Investigator:
          • Divya C Patel, DO, MBA

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 99 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.

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.

Other Names:
  • Tyvaso®
  • pulmonary arterial hypertension (PAH) therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
PVR by Right heart catheterization (RHC)
Time Frame: Baseline, Week 16
Change in RHC parameter PVR (pulmonary vascular resistance )
Baseline, Week 16
mPAP by Right heart catheterization (RHC)
Time Frame: Baseline, Week 16
Change in RHC parameter mPAP (mean pulmonary arterial pressure)
Baseline, Week 16

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Cardiac MRI parameters
Time Frame: Baseline, Week 16
Change in Right ventricle ejection fraction, Right ventricular end diastolic ventricle index, right ventricular systolic index
Baseline, Week 16
Change in Pulmonary Function Testing
Time Frame: Baseline, Week 16
Change in FEV1 abd FVC
Baseline, Week 16
Change in 6-Minute Walk Test (6MWT)
Time Frame: Baseline, Week 8, Week 16
change in walk test distance during the study
Baseline, Week 8, Week 16
Change in Brain Natriuretic Peptide (BNP)
Time Frame: Baseline, Week 16
change in BNP level during the study
Baseline, Week 16
Change in WHO Functional Class (WHO FC)
Time Frame: Baseline, Week 8, Week 16
change in WHO FC status during the study
Baseline, Week 8, Week 16

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Collaborators

Investigators

  • Principal Investigator: Ali Ataya, MD, University of Florida

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 30, 2020

Primary Completion (Anticipated)

October 29, 2023

Study Completion (Anticipated)

October 29, 2023

Study Registration Dates

First Submitted

January 4, 2019

First Submitted That Met QC Criteria

January 22, 2019

First Posted (Actual)

January 24, 2019

Study Record Updates

Last Update Posted (Actual)

May 19, 2023

Last Update Submitted That Met QC Criteria

May 17, 2023

Last Verified

May 1, 2023

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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