SarcoidosisPhase 2SAPPHIRENCT03814317
Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension
A Phase 2 study testing Inhaled Treprostinil in people with Sarcoidosis and Precapillary Pulmonary Hypertension, run by University of Florida at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Sarcoidosis, Precapillary Pulmonary Hypertension, Interstitial Lung Disease
- What is being tested?
- Inhaled Treprostinil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jan 2020to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Florida, Division of Pulmonary and Critical Care Medicine
Gainesville, Florida, 32610
About this study
Official title: An Open-Label Study of Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension and Interstitial Lung Disease (SAPPHIRE)
This study aims to evaluate the efficacy and safety of inhaled treprostinil in subjects with sarcoidosis-associated interstitial lung disease and pulmonary hypertension.
Read the 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.
- Being tested
- Inhaled Treprostinil
- Keywords
- right heart catheterization (RHC), pulmonary vascular resistance (PVR), mean pulmonary arterial pressure (mPAP), pulmonary hypertension (PH), Inhaled treprostinil, Sarcoidosis
- Medical subject headings
- Sarcoidosis, Lung Diseases, Interstitial, Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inhaled TreprostinilDrug
Also called Tyvaso®, pulmonary arterial hypertension (PAH) therapy
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.
| Study group | Role | What they receive |
|---|---|---|
| Study Group | Experimental | 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. Receives: Drug: Inhaled Treprostinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
PVR by Right heart catheterization (RHC)
Change in RHC parameter PVR (pulmonary vascular resistance )
Measured: Baseline, Week 16
mPAP by Right heart catheterization (RHC)
Change in RHC parameter mPAP (mean pulmonary arterial pressure)
Measured: Baseline, Week 16
Secondary outcomes (5)
Change in 6-Minute Walk Test (6MWT)
change in walk test distance during the study
Measured: Baseline, Week 8, Week 16
Change in Cardiac MRI parameters
Change in Right ventricle ejection fraction, Right ventricular end diastolic ventricle index, right ventricular systolic index
Measured: Baseline, Week 16
Change in Pulmonary Function Testing
Change in FEV1 abd FVC
Measured: Baseline, Week 16
Change in Brain Natriuretic Peptide (BNP)
change in BNP level during the study
Measured: Baseline, Week 16
Change in WHO Functional Class (WHO FC)
change in WHO FC status during the study
Measured: Baseline, Week 8, Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2019
- Study start30 Jan 2020
- Primary completion31 Dec 2025
- Study completion31 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SarcoidosisStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- United Therapeutics
- Responsible party
- Sponsor
- Investigators
- Ali Ataya, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03814317
- Sponsor's protocol ID
- OCR19684
- Other IDs
- 20192572
- Acronym
- SAPPHIRE
- Status last verified
- Jun 2025
- First submitted
- 4 Jan 2019
- Last updated
- 6 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study of Inhaled Treprostinil in Sarcoidosis Patients With Pulmonary Hypertension and Interstitial Lung Disease (SAPPHIRE)
- ClinicalTrials.gov ID
- NCT03814317
- Acronym / sponsor ID
- SAPPHIRE / OCR19684
- Keywords
- right heart catheterization (RHC), pulmonary vascular resistance (PVR), mean pulmonary arterial pressure (mPAP), pulmonary hypertension (PH), Inhaled treprostinil, Sarcoidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- United Therapeutics
- Study start
- 30 Jan 2020 (actual)
- Primary completion
- 31 Dec 2025 (actual)
- Study completion
- 31 Dec 2025 (actual)
- First posted
- 24 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| Study Group | Experimental | Drug: Inhaled TreprostinilSarcoidosis 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. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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)
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Inhaled TreprostinilAlso: Tyvaso®, pulmonary arterial hypertension (PAH) therapyInhaled 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. | Drug | Study Group |
Primary outcome
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)
5 secondary outcomes
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 Pulmonary Function Testing
Time frame: Baseline, Week 16
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ali Ataya, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Inhaled Treprostinil in people with Sarcoidosis and Precapillary Pulmonary Hypertension, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PVR by Right heart catheterization (RHC) (time frame: Baseline, Week 16). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2026.