A Study to Observe the Safety, Tolerability, and Efficacy of Remodulin

A Multi-center, Prospective Study to Observe the Safety, Tolerability, and Efficacy of Remodulin® in Patients With Pulmonary Arterial Hypertension

This is a multi-center, single-arm, prospective study to enroll patients diagnosed as pulmonary arterial hypertension. The objective in this study is to observe the efficacy and safety of treprostinil in subjects with pulmonary arterial hypertension.

Study Overview

• Status: Not yet recruiting
• Conditions: Pulmonary Arterial Hypertension
• Intervention / Treatment: Drug: Treprostinil

Detailed Description

This is a multi-center, single-arm, prospective study to enroll patients diagnosed as pulmonary arterial hypertension. The expected study period will be 2 years. PAH patients, who met NHI criteria, to receive treprostinil subcutaneously/ intravenously, will be invited to join this study for observation. The dosage and administration schedule of study medication will be judged by investigators. If the eligibility criteria have been met and the subject have signed the informed consent, the subjects will be enrolled into this study. There will be 10 visits: Visit 1 (Screening visit, Week -2 ~ -1), Visit 2 (Week 1, initial of use treprostinil), Visit 3 to 9 (Week 13 to 85), Visit 10 (Week 97). The Day 1 in Week 1 should be the start day of treprostinil treatment. Subjects will be returned to the clinics every 3 months (i.e. 12 weeks) after Visit 2. The NT-proBNP level, WHO functional class, 6-minute walk distance, echocardiography, pulse oximetry, modified Borg Dyspnea Scale, SF-36 questionnaire, and dosage regimens of treprostinil (including dose adjustment) will be collected. For time to clinical worsening and death, it will be followed up during study period. All intercurrent events within 24 months will be recorded. Safety information, including vital signs, and physical exams will be checked. Adverse events of special interest (AESIs) will be reviewed every visit by using a predesignated AESI checklist.

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Cliff Lin; Phone Number: 1421 +886226557568; Email: cliff.lin@excelsiorgroup.com.tw

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

The patients who were diagnosed with symptomatic idiopathic or heritable PAH classified as WHO functional class III and IV symptoms and met NHI criteria, will be scheduled to receive subcutaneous/intravenous treprostinil treatment

Description

Inclusion Criteria:

• Diagnosed with symptomatic idiopathic or heritable PAH classified as WHO functional class III and IV symptoms.
• PAH patient, who met NHI criteria, will be scheduled to receive subcutaneous/intravenous treprostinil treatment.
• Ability to adhere to the study visit schedule and understand and comply with all protocol requirements.
• Will provide completed and signed written informed consents

Exclusion Criteria:

• Active treatment with injectable prostanoids different from subcutaneous/intravenous treprostinil

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in NT-proBNP	Calculate the "N terminal pro B type natriuretic peptide" change from baseline to Week 97	Week 97
Change from baseline in WHO functional class	Calculate the World Health Organization functional class change from baseline to Week 97. The World Health Organization (WHO) functional class describes how severe a patient's pulmonary hypertension (PH) symptoms are. There are four different classes - I is the mildest and IV is the most severe form of PH.	Week 97

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to clinical worsening and death	Record the time to clinical worsening and death	96 weeks
Change from baseline in NT-proBNP	Calculate the change from baseline in NT-proBNP at Week 13, 25, 37, 49, 61, 73, and 85	Week 13, 25, 37, 49, 61, 73, and 85
Change from baseline in 6-minute walk distance (6MWD)	Calculate the cange from baseline in 6-minute walk distance (6MWD) at Week 25, 49, 73, and 97	Week 25, 49, 73, and 97
Trend of dose usage from initiation treatment to clinical worsening	Record all the daily doses which the patients administered during the whole study period	96 weeks
Adverse events of special interest (AESIs)	AESIs include infusion site pain, infusion site reaction, headache, diarrhea, nausea, rash, jaw pain, hot flashes, vasodilation, and edema	96 weeks
Change from baseline in WHO functional class	Calculate the change from baseline in WHO functional class at Week 13, 25, 37, 49, 61, 73, and 85 The World Health Organization (WHO) functional class describes how severe a patient's pulmonary hypertension (PH) symptoms are. There are four different classes - I is the mildest and IV is the most severe form of PH.	Week 13, 25, 37, 49, 61, 73, and 85
Change from baseline in right atrium (RA) area	RV structure and function will be monitored by echocardiography, including Right atrium (RA) area.	Week 25, 49, 73, and 97
Change from baseline in TAPSE/sPAP ratio.	RV structure and function will be monitored by echocardiography, including Tricuspid annular plane systolic excursion (TAPSE) / systolic pulmonary artery pressure (sPAP) ratio.	Week 25, 49, 73, and 97
Change from baseline in Pericardial effusion.	RV structure and function will be monitored by echocardiography, including Pericardial effusion.	Week 25, 49, 73, and 97
Change from baseline in modified Borg Dyspnea Scale	Calculate the change from baseline in modified Borg Dyspnea Scale at Week 25, 49, 73, and 97. The modified Borg Dyspnea scales are patient self-reported measures of one's difficulty in breathing upon exertion. The Scale is a categorical scale with a score from 0 to 10, where 0 represents normal breathing and 10 represents maximum dyspnea.	Week 25, 49, 73, and 97
Change from baseline in SF-36 questionnaire	Calculate the change from baseline in SF-36 questionnaire at Week 25, 49, 73, and 97. The 36-Item Short Form Survey (SF-36) is an outcome measure instrument that is often used, well-researched, self-reported measure of health. It stems from a study called the Medical Outcomes Study for the objective measure of the quality of life. It comprises 36 questions that cover eight domains of health. Scores for the different domains are converted and pooled using a scoring key, for a total score indicating a range of low to high QOL.	Week 25, 49, 73, and 97
Intercurrent event	Intercurrent events includes consent withdrawn, use of rescue medication, discontinuation from treprostinil treatment, PAH unrelated hospitalization, switch to other PAH drugs (other than treprostinil), add-on PAH drugs, or new-onset co-morbidity.	96 weeks

Collaborators and Investigators

• Sponsor: Excelsior

Study record dates

Study Major Dates

• Study Start (Estimated): October 16, 2025
• Primary Completion (Estimated): May 31, 2029
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: August 22, 2024
• First Submitted That Met QC Criteria: September 18, 2024
• First Posted (Actual): September 19, 2024

Study Record Updates

• Last Update Posted (Estimated): August 15, 2025
• Last Update Submitted That Met QC Criteria: August 12, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension, Pulmonary; Pulmonary Arterial Hypertension; Hypertension; Familial Primary Pulmonary Hypertension; Antihypertensive Agents; Treprostinil
• Other Study ID Numbers: EB057-PMS-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No