Clinical trial NCT06603285: A Study to Observe the Safety, Tolerability, and Efficacy of Remodulin
Official title: A Multi-center, Prospective Study to Observe the Safety, Tolerability, and Efficacy of Remodulin® in Patients with Pulmonary Arterial Hypertension
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Pulmonary Arterial Hypertension
Lead sponsor: Excelsior
Sponsor class: INDUSTRY
First posted: 2024-09-19
Enrollment: 20
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline in NT-proBNP
timeFrame: Week 97
measure: Change from baseline in WHO functional class
timeFrame: Week 97
Secondary outcomes:
measure: Time to clinical worsening and death
timeFrame: 96 weeks
measure: Change from baseline in NT-proBNP
timeFrame: Week 13, 25, 37, 49, 61, 73, and 85
measure: Change from baseline in 6-minute walk distance (6MWD)
timeFrame: Week 25, 49, 73, and 97
measure: Trend of dose usage from initiation treatment to clinical worsening
timeFrame: 96 weeks
measure: Adverse events of special interest (AESIs)
timeFrame: 96 weeks
measure: Change from baseline in WHO functional class
timeFrame: Week 13, 25, 37, 49, 61, 73, and 85
measure: Change from baseline in right atrium (RA) area
timeFrame: Week 25, 49, 73, and 97
measure: Change from baseline in TAPSE/sPAP ratio.
timeFrame: Week 25, 49, 73, and 97
measure: Change from baseline in Pericardial effusion.
timeFrame: Week 25, 49, 73, and 97
measure: Change from baseline in modified Borg Dyspnea Scale
timeFrame: Week 25, 49, 73, and 97
measure: Change from baseline in SF-36 questionnaire
timeFrame: Week 25, 49, 73, and 97
measure: Intercurrent event
timeFrame: 96 weeks
Other outcomes:
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