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Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension (SAPPHIRE)

Primary Purpose

Sarcoidosis, Precapillary Pulmonary Hypertension, Interstitial Lung Disease

Status

Recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Inhaled Treprostinil

About this trial

This is an interventional treatment trial for Sarcoidosis focused on measuring right heart catheterization (RHC), pulmonary vascular resistance (PVR), mean pulmonary arterial pressure (mPAP), pulmonary hypertension (PH), Inhaled treprostinil, Sarcoidosis

Eligibility Criteria

18 Years - 99 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

University of Florida, Division of Pulmonary and Critical Care MedicineRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Study Group

Arm Description

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.

Outcomes

Primary Outcome Measures

PVR by Right heart catheterization (RHC)

Change in RHC parameter PVR (pulmonary vascular resistance )

mPAP by Right heart catheterization (RHC)

Change in RHC parameter mPAP (mean pulmonary arterial pressure)

Secondary Outcome Measures

Change in 6-Minute Walk Test (6MWT)

change in walk test distance during the study

Change in Cardiac MRI parameters

Change in Right ventricle ejection fraction, Right ventricular end diastolic ventricle index, right ventricular systolic index

Change in Pulmonary Function Testing

Change in FEV1 abd FVC

Change in Brain Natriuretic Peptide (BNP)

change in BNP level during the study

Change in WHO Functional Class (WHO FC)

change in WHO FC status during the study

Full Information

NCT ID

NCT03814317

First Posted

January 4, 2019

Last Updated

May 17, 2023

Sponsor

University of Florida

Collaborators

United Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT03814317

Brief Title

Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension

Acronym

SAPPHIRE

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2023

Overall Recruitment Status

Recruiting

Study Start Date

January 30, 2020 (Actual)

Primary Completion Date

October 29, 2023 (Anticipated)

Study Completion Date

October 29, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

University of Florida

Collaborators

United Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Model Description

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.

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Study Group

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Inhaled Treprostinil

Other Intervention Name(s)

Tyvaso®, pulmonary arterial hypertension (PAH) therapy

Intervention Description

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.

Primary Outcome Measure Information:

Title

PVR by Right heart catheterization (RHC)

Description

Change in RHC parameter PVR (pulmonary vascular resistance )

Time Frame

Baseline, Week 16

Title

mPAP by Right heart catheterization (RHC)

Description

Change in RHC parameter mPAP (mean pulmonary arterial pressure)

Time Frame

Baseline, Week 16

Secondary Outcome Measure Information:

Title

Change in 6-Minute Walk Test (6MWT)

Description

change in walk test distance during the study

Time Frame

Baseline, Week 8, Week 16

Title

Change in Cardiac MRI parameters

Description

Change in Right ventricle ejection fraction, Right ventricular end diastolic ventricle index, right ventricular systolic index

Time Frame

Baseline, Week 16

Title

Change in Pulmonary Function Testing

Description

Change in FEV1 abd FVC

Time Frame

Baseline, Week 16

Title

Change in Brain Natriuretic Peptide (BNP)

Description

change in BNP level during the study

Time Frame

Baseline, Week 16

Title

Change in WHO Functional Class (WHO FC)

Description

change in WHO FC status during the study

Time Frame

Baseline, Week 8, Week 16

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

99 Years

Accepts Healthy Volunteers

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Christina M Eagan, DNP

Phone

352-273-8990

christina.eagan@medicine.ufl.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Rosie Kizza, RN

Phone

352-273-7225

rosie.kizza@medicine.ufl.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ali Ataya, MD

Organizational Affiliation

University of Florida

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Florida, Division of Pulmonary and Critical Care Medicine

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32610

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Christina M Eagan, DNP

First Name & Middle Initial & Last Name & Degree

Ali Ataya, MD

First Name & Middle Initial & Last Name & Degree

Divya C Patel, DO, MBA

12. IPD Sharing Statement

Plan to Share IPD

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Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension

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