An interventional study of Ambrisentan and Placebo in Pulmonary Arterial Hypertension (PAH), sponsored by Nanjing First Hospital, Nanjing Medical University. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-05-23.
Sponsored by Nanjing First Hospital, Nanjing Medical University · Not applicable, Interventional, and Treatment
This is an investigator-initiated, multicenter, randomized, double-blind, placebo-controlled clinical trial.
Early-stage low-risk PAH is defined as mean pulmonary arterial pressure (mPAP) between 20 and 25 mmHg at rest, measured by right heart catheterization, and classified as low-risk based on the 2022 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension. Patients will be randomized at a 1:1 ratio to either the treatment group (Ambrisentan group) or the control group (Placebo group). Treatment group: Ambrisentan, with an initial dose of 5 mg/day (one tablet per day).Control group: Placebo, which will be provided in the same appearance and taste as Ambrisentan, one tablet per day.
After two weeks of initial treatment, the study drugs' dose will be increased to two tablets per day (10 mg/day). If the patient cannot tolerate the increased dose (e.g., experiencing headache, dizziness, palpitations, hypotension, or other drug-related symptoms or signs), the dose will be reduced to 5 mg/day. If the study drug has reached the maximum allowable dose (two tablets/day) and the patient shows signs of worsening PAH or right heart failure, the clinician may decide to add diuretics (with the type and dosage left at the referring physician's discretion). The number and percentage of patients requiring diuretic combination therapy in both groups will be recorded. Other baseline treatment medications will remain unchanged through follow-up duration.
The study drugs will be administered continuously for 12 months, then unblinding will be performed. Thereafter, patients who have reached the primary endpoint must undertake Ambrisentan. For patients who have not reached the primary endpoint, the subsequent medications treatment will be left at the PAH specialist's discretion. Follow-up will be undertaken at the following timing: Month 1, Month 6, and Month 12, with additional follow-up extending up to 3 years. All clinical drugs involved in this study have completed registration for market approval in China and are currently in clinical use.
761 studies on the registry are indexed under Pulmonary Arterial Hypertension; 142 are open to participants now.
This study's planned enrollment of 410 is above the median of 38 across 509 interventional studies indexed under Pulmonary Arterial Hypertension.
Browse Pulmonary Arterial Hypertension studies →Nanjing First Hospital, Nanjing Medical University is the lead sponsor of 213 studies on the registry; 106 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Monotherapy using ambrisentan will start at a dose of 5 mg (once daily) and will be up-titrated to 10mg (once daily) after 2 weeks apart if patients are tolerable.
Drug: Ambrisentan
Placebo tablet (one to two tablets corresponding to one to two verum tablets).
Drug: Placebo
After two weeks of initial treatment, the study drugs' dose will be increased to two tablets per day (10 mg/day). If the patient cannot tolerate the increased dose (e.g., experiencing headache, dizziness, palpitations, hypotension, or other drug-related symptoms or signs), the dose will be reduced to 5 mg/day. If the study drug has reached the maximum allowable dose (two tablets/day) and the patient shows signs of worsening PAH or right heart failure, the clinician may decide to add diuretics (with the type and dosage left at the referring physician's discretion). The number and percentage of patients requiring diuretic combination therapy in both groups will be recorded. Other baseline treatment medications will remain unchanged through follow-up duration.
Placebo tablet (one to two tablets corresponding to one to two verum tablets). Administration: Placebo will be administrated orally with or without food intake in the morning.
The primary efficacy endpoint is the composite of pulmonary hypertension progression at 12 months
defined as meeting any of the following criteria within 12 months: 1. mPAP ≥ 25 mmHg OR PVR \> 3 WUs as measured by RHC OR 2. worsening of risk stratification compared to baseline, defining as at least one class progression based on the simplified four-tier model of the 2022 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension
Time frame: baseline,12 months
systolic PAP (sPAP) by Hemodynamic measurements
Time frame: baseline,12 months
mPAP by Hemodynamic measurements
Time frame: baseline,12 months
cardiac output (CO) by Hemodynamic measurements
Time frame: baseline,12 months
cardiac index (CI) by Hemodynamic measurements
Time frame: baseline,12 months
PVR by Hemodynamic measurements
Time frame: baseline,12 months
PVRi by Hemodynamic measurements
Time frame: baseline,12 months
PAWP by Hemodynamic measurements
Time frame: baseline,12 months
right atrial pressure by Hemodynamic measurements
Time frame: baseline,12 months
pulmonary arterial compliance by Hemodynamic measurements
Time frame: baseline,12 months
diameter of chambers by Echocardiographic measurements
Time frame: baseline,12 months
ejection fraction by Echocardiographic measurements
Time frame: baseline,12 months
right ventricular (RV) fraction of area change (FAC) by Echocardiographic measurements
Time frame: baseline,12 months
TAPSE by Echocardiographic measurements
Time frame: baseline,12 months
pulmonary artery accelation time (PAAT) by Echocardiographic measurements
Time frame: baseline,12 months
regurgitation of tricuspid or pulmonary valve by Echocardiographic measurements
Time frame: baseline,12 months
sPAP by Echocardiographic measurements
Time frame: baseline,12 months
RAP by Echocardiographic measurements
Time frame: baseline,12 months
WHO functional class through follow - up
Time frame: baseline,12 months
Borg index through follow - up
Time frame: baseline,12 months
N-terminal-pro BNP
Time frame: baseline,12 months
6-minute walk distance (6MWD)
Time frame: baseline,12 months
Time to the first occurrence of the following events, analyzed using log-rank and LWYY model
* All-cause mortality * Hospitalization due to PAH worsening * Clinical deterioration of PAH, defined as the need for diuretics or digoxin (oral or intravenous), or the need of combination therapy with PAH specific medications * 6MWD decreases by 10% or 30m
Time frame: baseline,12 months
No study locations are listed for this record.
Plan to share: No
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Pulmonary Arterial Hypertension→
Nanjing First Hospital, Nanjing Medical University