Pulmonary Arterial Hypertension (PAH)Phase 2NCT07177703
Efficacy and Safety of Treprostinil in Intermediate-Risk Pulmonary Arterial Hypertension
A Phase 2 study testing Treprostinil in people with Pulmonary Arterial Hypertension (PAH), run by Shanghai Zhongshan Hospital.
Trial summary
Pulmonary Arterial Hypertension (PAH) is a serious condition that puts strain on the right side of the heart. While oral medications can help, many patients with intermediate-risk PAH may not see enough improvement, and their heart function can continue to decline. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Pulmonary Arterial Hypertension (PAH)
- What is being tested?
- Treprostinil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- No sites listed yet
- Study dates
- 31 Oct 2025to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed the informed consent form.
- Age between 18 and 75 years, inclusive.
- Diagnosed with Pulmonary Arterial Hypertension (PAH, WHO Group 1) of the following etiologies:Idiopathic;Heritable;Drug or toxin-induced (excluding amphetamine-induced PAH);Associated with repaired congenital systemic-to-pulmonary shunts (repair performed ≥1 year prior);Associated with connective tissue disease;Associated with HIV infection;
- Has undergone a right heart catheterization (RHC) within 6 months of the baseline visit.
- Baseline RHC demonstrates the following hemodynamics:Mean pulmonary arterial pressure (mPAP) > 20 mmHg;Pulmonary artery wedge pressure (PAWP) ≤ 15 mmHg;Pulmonary vascular resistance (PVR) > 2 Wood units;
- WHO Functional Class II, III, or IV.
- Risk stratification according to 2022 ESC/ERS guidelines is either intermediate-risk at initial diagnosis or low- to intermediate-risk during follow-up.
- Patient is on a stable dose of an endothelin receptor antagonist (ERA) and/or a phosphodiesterase-5 inhibitor (PDE-5i) or a soluble guanylate cyclase (sGC) stimulator for at least 30 days prior to baseline; OR, if treatment-naïve, is willing to initiate one of these therapies in addition to the study drug.
Who cannot join
- Age < 18 years.
- Diagnosis of Pulmonary Hypertension belonging to WHO Groups 2, 3, 4, or 5.
- Patients undergoing dialysis.
- Severe hepatic impairment (Child-Pugh Class C).
- Use of any prostacyclin-pathway drugs (epoprostenol, treprostinil, iloprost, beraprost, or selexipag) within 30 days prior to the baseline visit.
- Presence of implanted metallic devices that would interfere with Cardiac - - - Magnetic Resonance (CMR) imaging, such as defibrillators, neurostimulators, cochlear implants, permanent infusion devices, implanted pumps, or certain plates/screws.
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- In the investigator's judgment, the patient is unable to complete or comply with study requirements.
- Refusal to sign the informed consent form or refusal to participate in follow-up visits.
- Participation in any other clinical trial involving an investigational drug, device, or procedure within 30 days prior to the baseline visit.
About this study
Official title: A Prospective Single-arm Exploratory Study on the Efficacy and Safety of Treprostinil Injection in the Treatment of Patients With Intermediate-risk Pulmonary Arterial Hypertension
Pulmonary Arterial Hypertension (PAH) is a serious condition that puts strain on the right side of the heart. While oral medications can help, many patients with intermediate-risk PAH may not see enough improvement, and their heart function can continue to decline.
This study aims to find out if adding an injectable medication, Treprostinil, to a patient's current oral PAH therapy can improve heart function and overall health. This is a single-arm study, which means all participants will receive the study treatment.
Read more
The main goal is to measure the change in the amount of blood the right side of the heart pumps with each beat (Right Ventricular Stroke Volume, or RVSV) after 3 months of treatment. This will be measured using a specialized heart scan called Cardiac Magnetic Resonance Imaging (CMR). Researchers will also assess changes in exercise ability (with a 6-minute walk test), blood markers, and patient symptoms.
Participants will be in the main part of the study for 3 months, with follow-up for a total of 24 months to monitor the long-term effects and safety of the treatment.
Read the detailed description
Background and Rationale:
Pulmonary Arterial Hypertension (PAH) is a progressive vasculopathy leading to increased pulmonary vascular resistance, right ventricular (RV) failure, and mortality. While guidelines recommend initial combination oral therapy, a substantial number of patients, particularly those at intermediate-risk, do not achieve a low-risk status. Subclinical RV deterioration can occur even in seemingly stable patients, suggesting that more intensive therapeutic strategies may be warranted to improve long-term outcomes.
Parenteral prostacyclin analogues, like Treprostinil, are potent therapies recommended for high-risk PAH. However, there is a lack of prospective data on their efficacy in inducing positive RV remodeling and improving clinical outcomes when initiated in patients with intermediate-risk PAH who are already on background oral therapies. This study aims to address this evidence gap.
Study Design and Objectives:
This is a prospective, single-center, single-arm, open-label, exploratory study designed to evaluate the efficacy and safety of adding subcutaneous Treprostinil to standard-of-care oral therapy for patients with intermediate-risk PAH.
The primary objective is to assess the change from baseline in Right Ventricular Stroke Volume (RVSV) at 3 months, as measured by Cardiac Magnetic Resonance (CMR). The hypothesis is that the addition of Treprostinil will lead to a significant improvement in RVSV, reflecting favorable RV remodeling.
Secondary objectives include evaluating changes from baseline in:
WHO Functional Class (FC) 6-minute walk distance (6MWD) N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels Other CMR-derived parameters of RV structure and function (e.g., RV ejection fraction [RVEF], RV end-systolic volume index [RVESVI]) Risk stratification scores Incidence of clinical worsening events over the follow-up period. Exploratory objectives include the assessment of changes in other echocardiographic parameters, health-related quality of life, and a panel of serum biomarkers associated with inflammation and cardiac remodeling (e.g., TNF-α, IL-6, MMP2, TIMP1).
Methodology:
A total of 32 eligible patients with intermediate-risk PAH will be enrolled. Participants must be on a stable dose of at least one oral PAH-targeted therapy (e.g., an endothelin receptor antagonist and/or a phosphodiesterase-5 inhibitor/soluble guanylate cyclase stimulator) or be treatment-naïve and willing to start oral therapy concurrently.
All participants will initiate subcutaneous Treprostinil, with the dose titrated according to tolerability and clinical response, following the product's prescribing information.
Comprehensive assessments, including CMR, 6MWD, WHO FC, and laboratory tests, will be performed at baseline and at 3 months. Further follow-up assessments will occur at 1 month and continue for a total of 24 months to evaluate long-term safety and outcomes. Safety will be monitored throughout the study by recording all adverse events and laboratory abnormalities.
- Conditions
- Pulmonary Arterial Hypertension (PAH)
- Being tested
- Treprostinil
- Keywords
- PAH, Treprostinil
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreprostinilDrug
Treprostinil is initiated at a dose of 1.25 ng/kg/min. The dose is then titrated upwards based on individual patient tolerability and clinical response. The planned titration schedule is an increase of 1.25 ng/kg/min weekly for the first four weeks of treatment, followed by weekly increases of 2.5 ng/kg/min thereafter, as tolerated.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Treatment is added to the participant's stable background regimen of standard-of-care oral PAH therapies (e.g., endothelin receptor antagonists, phosphodiesterase-5 inhibitors, and/or soluble guanylate cyclase stimulators).
Treprostinil is initiated at a dose of 1.25 ng/kg/min. The dose is then titrated upwards based on individual patient tolerability and clinical response. The planned titration schedule is an increase of 1.25 ng/kg/min weekly for the first four weeks of treatment, followed by weekly increases of 2.5 ng/kg/min thereafter, as tolerated. Receives: Drug: Treprostinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Right Ventricular Stroke Volume (RVSV)
Change in Right Ventricular Stroke Volume (RVSV) from baseline, as measured by Cardiac Magnetic Resonance (CMR). RVSV is the volume of blood pumped from the right ventricle of the heart during one contraction.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2025
- Study start31 Oct 2025 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Arterial Hypertension (PAH)Studies of the same condition, open and under way.
- More trials run by Shanghai Zhongshan HospitalThe 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
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Zhongshan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Junbo Ge, Shanghai Zhongshan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07177703
- Sponsor's protocol ID
- Tre-ImPAH
- Status last verified
- Sep 2025
- First submitted
- 10 Sep 2025
- Last updated
- 17 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Single-arm Exploratory Study on the Efficacy and Safety of Treprostinil Injection in the Treatment of Patients With Intermediate-risk Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT07177703
- Acronym / sponsor ID
- Tre-ImPAH
- Conditions
- Pulmonary Arterial Hypertension (PAH)
- Keywords
- PAH, Treprostinil
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Shanghai Zhongshan HospitalUniversity, hospital or other
- Study start
- 31 Oct 2025 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 17 Sep 2025 (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.
Pulmonary Arterial Hypertension (PAH) is a serious condition that puts strain on the right side of the heart. While oral medications can help, many patients with intermediate-risk PAH may not see enough improvement, and their heart function can continue to decline.
This study aims to find out if adding an injectable medication, Treprostinil, to a patient's current oral PAH therapy can improve heart function and overall health. This is a single-arm study, which means all participants will receive the study treatment.
The main goal is to measure the change in the amount of blood the right side of the heart pumps with each beat (Right Ventricular Stroke Volume, or RVSV) after 3 months of treatment. This will be measured using a specialized heart scan called Cardiac Magnetic Resonance Imaging (CMR). Researchers will also assess changes in exercise ability (with a 6-minute walk test), blood markers, and patient symptoms.
Participants will be in the main part of the study for 3 months, with follow-up for a total of 24 months to monitor the long-term effects and safety of the treatment.
Detailed description
Background and Rationale:
Pulmonary Arterial Hypertension (PAH) is a progressive vasculopathy leading to increased pulmonary vascular resistance, right ventricular (RV) failure, and mortality. While guidelines recommend initial combination oral therapy, a substantial number of patients, particularly those at intermediate-risk, do not achieve a low-risk status. Subclinical RV deterioration can occur even in seemingly stable patients, suggesting that more intensive therapeutic strategies may be warranted to improve long-term outcomes.
Parenteral prostacyclin analogues, like Treprostinil, are potent therapies recommended for high-risk PAH. However, there is a lack of prospective data on their efficacy in inducing positive RV remodeling and improving clinical outcomes when initiated in patients with intermediate-risk PAH who are already on background oral therapies. This study aims to address this evidence gap.
Study Design and Objectives:
This is a prospective, single-center, single-arm, open-label, exploratory study designed to evaluate the efficacy and safety of adding subcutaneous Treprostinil to standard-of-care oral therapy for patients with intermediate-risk PAH.
The primary objective is to assess the change from baseline in Right Ventricular Stroke Volume (RVSV) at 3 months, as measured by Cardiac Magnetic Resonance (CMR). The hypothesis is that the addition of Treprostinil will lead to a significant improvement in RVSV, reflecting favorable RV remodeling.
Secondary objectives include evaluating changes from baseline in:
WHO Functional Class (FC) 6-minute walk distance (6MWD) N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels Other CMR-derived parameters of RV structure and function (e.g., RV ejection fraction [RVEF], RV end-systolic volume index [RVESVI]) Risk stratification scores Incidence of clinical worsening events over the follow-up period. Exploratory objectives include the assessment of changes in other echocardiographic parameters, health-related quality of life, and a panel of serum biomarkers associated with inflammation and cardiac remodeling (e.g., TNF-α, IL-6, MMP2, TIMP1).
Methodology:
A total of 32 eligible patients with intermediate-risk PAH will be enrolled. Participants must be on a stable dose of at least one oral PAH-targeted therapy (e.g., an endothelin receptor antagonist and/or a phosphodiesterase-5 inhibitor/soluble guanylate cyclase stimulator) or be treatment-naïve and willing to start oral therapy concurrently.
All participants will initiate subcutaneous Treprostinil, with the dose titrated according to tolerability and clinical response, following the product's prescribing information.
Comprehensive assessments, including CMR, 6MWD, WHO FC, and laboratory tests, will be performed at baseline and at 3 months. Further follow-up assessments will occur at 1 month and continue for a total of 24 months to evaluate long-term safety and outcomes. Safety will be monitored throughout the study by recording all adverse events and laboratory abnormalities.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: TreprostinilTreatment is added to the participant's stable background regimen of standard-of-care oral PAH therapies (e.g., endothelin receptor antagonists, phosphodiesterase-5 inhibitors, and/or soluble guanylate cyclase stimulators). Treprostinil is initiated at a dose of 1.25 ng/kg/min. The dose is then titrated upwards based on individual patient tolerability and clinical response. The planned titration schedule is an increase of 1.25 ng/kg/min weekly for the first four weeks of treatment, followed by weekly increases of 2.5 ng/kg/min thereafter, as tolerated. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Signed the informed consent form.
- Age between 18 and 75 years, inclusive.
- Diagnosed with Pulmonary Arterial Hypertension (PAH, WHO Group 1) of the following etiologies:Idiopathic;Heritable;Drug or toxin-induced (excluding amphetamine-induced PAH);Associated with repaired congenital systemic-to-pulmonary shunts (repair performed ≥1 year prior);Associated with connective tissue disease;Associated with HIV infection;
- Has undergone a right heart catheterization (RHC) within 6 months of the baseline visit.
- Baseline RHC demonstrates the following hemodynamics:Mean pulmonary arterial pressure (mPAP) > 20 mmHg;Pulmonary artery wedge pressure (PAWP) ≤ 15 mmHg;Pulmonary vascular resistance (PVR) > 2 Wood units;
Key exclusion criteria (9 listed)
- Age < 18 years.
- Diagnosis of Pulmonary Hypertension belonging to WHO Groups 2, 3, 4, or 5.
- Patients undergoing dialysis.
- Severe hepatic impairment (Child-Pugh Class C).
- Use of any prostacyclin-pathway drugs (epoprostenol, treprostinil, iloprost, beraprost, or selexipag) within 30 days prior to the baseline visit.
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 |
|---|---|---|
| TreprostinilTreprostinil is initiated at a dose of 1.25 ng/kg/min. The dose is then titrated upwards based on individual patient tolerability and clinical response. The planned titration schedule is an increase of 1.25 ng/kg/min weekly for the first four weeks of treatment, followed by weekly increases of 2.5 ng/kg/min thereafter, as tolerated. | Drug | Treatment Group |
Primary outcome
Change From Baseline in Right Ventricular Stroke Volume (RVSV)
Time frame: 3 months
Change in Right Ventricular Stroke Volume (RVSV) from baseline, as measured by Cardiac Magnetic Resonance (CMR). RVSV is the volume of blood pumped from the right ventricle of the heart during one contraction.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2025.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Treprostinil in people with Pulmonary Arterial Hypertension (PAH), run by Shanghai Zhongshan Hospital.
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 9 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: opening soon, as of the sponsor's last update on 17 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
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?
Change From Baseline in Right Ventricular Stroke Volume (RVSV) (time frame: 3 months). 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 17 Sep 2025.