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Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension (SAPPHIRE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03814317
Recruitment Status : Recruiting
First Posted : January 24, 2019
Last Update Posted : November 15, 2021
Sponsor:
Collaborator:
United Therapeutics
Information provided by (Responsible Party):
University of Florida

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.

Condition or disease Intervention/treatment Phase
Sarcoidosis Precapillary Pulmonary Hypertension Interstitial Lung Disease Drug: Inhaled Treprostinil Phase 2

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.

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




Primary Outcome Measures :
  1. PVR by Right heart catheterization (RHC) [ Time Frame: Baseline, Week 16 ]
    Change in RHC parameter PVR (pulmonary vascular resistance )

  2. mPAP by Right heart catheterization (RHC) [ Time Frame: Baseline, Week 16 ]
    Change in RHC parameter mPAP (mean pulmonary arterial pressure)


Secondary Outcome Measures :
  1. Change in 6-Minute Walk Test (6MWT) [ Time Frame: Baseline, Week 8, Week 16 ]
  2. 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

  3. Change in Pulmonary Function Testing [ Time Frame: Baseline, Week 16 ]
    Change in FEV1 abd FVC

  4. Change in Brain Natriuretic Peptide (BNP) [ Time Frame: Baseline, Week 16 ]
  5. Change in WHO Functional Class (WHO FC) [ Time Frame: Baseline, Week 8, Week 16 ]


Information from the National Library of Medicine

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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
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.

Information from the National Library of Medicine

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


Contacts
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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

Locations
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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         
Sponsors and Collaborators
University of Florida
United Therapeutics
Investigators
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Principal Investigator: Ali Ataya, MD University of Florida
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University of Florida:
right heart catheterization (RHC)
pulmonary vascular resistance (PVR)
mean pulmonary arterial pressure (mPAP)
pulmonary hypertension (PH)
Inhaled treprostinil
Sarcoidosis
Additional relevant MeSH terms:
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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