- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03814317
Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension (SAPPHIRE)
An Open-Label Study of Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension and Interstitial Lung Disease (SAPPHIRE)
Study Overview
Status
Intervention / Treatment
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
Enrollment (Anticipated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Christina M Eagan, DNP
- Phone Number: 352-273-8990
- Email: christina.eagan@medicine.ufl.edu
Study Contact Backup
- Name: Rosie Kizza, RN
- Phone Number: 352-273-7225
- Email: rosie.kizza@medicine.ufl.edu
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
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
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
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:
|
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
Sponsor
Collaborators
Investigators
- Principal Investigator: Ali Ataya, MD, University of Florida
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Respiratory Tract Diseases
- Immune System Diseases
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Hypersensitivity
- Hypersensitivity, Delayed
- Hypertension
- Lung Diseases
- Hypertension, Pulmonary
- Lung Diseases, Interstitial
- Sarcoidosis
- Antihypertensive Agents
- Treprostinil
Other Study ID Numbers
- OCR19684
- 20192572 (Other Identifier: WIRB)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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