Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension (SAPPHIRE)
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| ClinicalTrials.gov Identifier: NCT03814317 |
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Recruitment Status :
Recruiting
First Posted : January 24, 2019
Last Update Posted : November 15, 2021
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Sarcoidosis Precapillary Pulmonary Hypertension Interstitial Lung Disease | Drug: Inhaled Treprostinil | Phase 2 |
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 (Clinical Trial) |
| Estimated Enrollment : | 10 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Intervention 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) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label Study of Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension and Interstitial Lung Disease (SAPPHIRE) |
| Actual Study Start Date : | January 30, 2020 |
| Estimated Primary Completion Date : | October 2022 |
| Estimated Study Completion Date : | October 2022 |
| Arm | Intervention/treatment |
|---|---|
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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. |
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. Other Names:
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- PVR by Right heart catheterization (RHC) [ Time Frame: Baseline, Week 16 ]Change in RHC parameter PVR (pulmonary vascular resistance )
- mPAP by Right heart catheterization (RHC) [ Time Frame: Baseline, Week 16 ]Change in RHC parameter mPAP (mean pulmonary arterial pressure)
- Change in 6-Minute Walk Test (6MWT) [ Time Frame: Baseline, Week 8, Week 16 ]
- 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
- Change in Pulmonary Function Testing [ Time Frame: Baseline, Week 16 ]Change in FEV1 abd FVC
- Change in Brain Natriuretic Peptide (BNP) [ Time Frame: Baseline, Week 16 ]
- Change in WHO Functional Class (WHO FC) [ Time Frame: Baseline, Week 8, Week 16 ]
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| Ages Eligible for Study: | 18 Years to 99 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03814317
| Contact: Christina M Eagan, DNP | 352-273-8990 | christina.eagan@medicine.ufl.edu | |
| Contact: Rosie Kizza, RN | 352-273-7225 | rosie.kizza@medicine.ufl.edu |
| United States, Florida | |
| University of Florida, Division of Pulmonary and Critical Care Medicine | Recruiting |
| Gainesville, Florida, United States, 32610 | |
| Contact: Christina M Eagan, DNP | |
| Principal Investigator: Ali Ataya, MD | |
| Sub-Investigator: Divya C Patel, DO, MBA | |
| Principal Investigator: | Ali Ataya, MD | University of Florida |
| Responsible Party: | University of Florida |
| ClinicalTrials.gov Identifier: | NCT03814317 |
| Other Study ID Numbers: |
OCR19684 20192572 ( Other Identifier: WIRB ) |
| First Posted: | January 24, 2019 Key Record Dates |
| Last Update Posted: | November 15, 2021 |
| Last Verified: | November 2021 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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right heart catheterization (RHC) pulmonary vascular resistance (PVR) mean pulmonary arterial pressure (mPAP) |
pulmonary hypertension (PH) Inhaled treprostinil Sarcoidosis |
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Lung Diseases Hypertension, Pulmonary Lung Diseases, Interstitial Hypertension Sarcoidosis Vascular Diseases |
Cardiovascular Diseases Respiratory Tract Diseases Lymphoproliferative Disorders Lymphatic Diseases Treprostinil Antihypertensive Agents |