Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension

Interstitial Lung Disease Clinical Trial

— SAPPHIRE  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03814317
• Other study ID #: OCR19684
• Secondary ID: 20192572
• Status: Recruiting
• Phase: Phase 2
• Start date: January 30, 2020
• Est. completion date: October 29, 2023

Study information

• Verified date: May 2023
• Source: University of Florida
• Contact: Christina M Eagan, DNP
• Phone: 352-273-8990
• Email: christina.eagan[@]medicine.ufl.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: October 29, 2023
• Est. primary completion date: October 29, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hypertension
• Hypertension, Pulmonary
• Interstitial Lung Disease
• Lung Diseases
• Lung Diseases, Interstitial
• Precapillary Pulmonary Hypertension
• Sarcoidosis

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
United States	University of Florida, Division of Pulmonary and Critical Care Medicine	Gainesville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Florida	United Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	PVR by Right heart catheterization (RHC)	Change in RHC parameter PVR (pulmonary vascular resistance )	Baseline, Week 16
Primary	mPAP by Right heart catheterization (RHC)	Change in RHC parameter mPAP (mean pulmonary arterial pressure)	Baseline, Week 16
Secondary	Change in 6-Minute Walk Test (6MWT)	change in walk test distance during the study	Baseline, Week 8, Week 16
Secondary	Change in Cardiac MRI parameters	Change in Right ventricle ejection fraction, Right ventricular end diastolic ventricle index, right ventricular systolic index	Baseline, Week 16
Secondary	Change in Pulmonary Function Testing	Change in FEV1 abd FVC	Baseline, Week 16
Secondary	Change in Brain Natriuretic Peptide (BNP)	change in BNP level during the study	Baseline, Week 16
Secondary	Change in WHO Functional Class (WHO FC)	change in WHO FC status during the study	Baseline, Week 8, Week 16