EMPAGLIFLOZIN for Pulmonary arterial hypertension

Inclusion criteria

• {"criterion_text":"- Male and female ≥18 years\n- Body Mass Index (BMI) >18kg/m2\n- Symptomatic PAH belonging to one of the following 2018 WHO Clinical Group 1 subtypes: a. Idiopathic PAH b. Heritable PAH c. Drug- or toxin-induced d. PAH associated with: 1.\tConnective tissue disease 2.\tCongenital systemic to pulmonary shunt (atrial septal defect, ventricular septal defect, patent ductus arteriosus) repaired at least one year prior to Screening 3.\tHuman immunodeficiency virus (HIV) infection - if diagnosed with HIV, must have stable disease status defined as follows: a.\tstable treatment with HIV medications for at least 8 weeks prior to screening b.\tno active opportunistic infection during the screening period c.\tno hospitalizations due to HIV for at least 4 weeks prior to screening\n- WHO FC II or III\n- Confirmed diagnosis of PAH and meeting all the following hemodynamic criteria by means of a screening RHC completed prior to randomization: a.\tmPAP of >20 mmHg b.\tPVR ≥ 240 dyne•sec/cm5 c.\tPulmonary capillary wedge pressure (PCWP) ≤ 15 mmHg\n- Currently on a stable treatment regimen with one or more treatments approved for PAH. Stable therapy is defined as receiving the same medication(s) for ≥ 12 weeks prior to the screening RHC and at a stable dose level for each for ≥ 8 weeks prior to the screening RHC.\n- For women of childbearing potential: a negative result in a pregnancy test AND agreement to practice a highly effective method of contraception* during the entire period from informed consent up to 30 days after the last administration of the IMP.\n- Written informed consent"}

Exclusion criteria

• {"criterion_text":"- Major change in diuretic management during 48 hours prior to screening visit or 48 hours prior to randomization visit (major change defined by doubling of diuretic dose or addition of another diuretic medications).\n- Type 1 Diabetes\n- Pregnant or breastfeeding women\n- Following WHO PH Groups: · WHO PH Group 1 PAH associated with portal hypertension or schistosomiasis; · PH due to left heart disease (WHO PH Group 2); · lung diseases and/or hypoxia (WHO PH Group 3); ·\tchronic thromboembolic PH (WHO PH Group 4); or ·\tPH with unclear multifactorial mechanisms (WHO PH Group 5)\n- PH associated with: ·\tsignificant venous or capillary involvement (PCWP > 15 mmHg); ·\tpulmonary capillary hemangiomatosis; ·\tportal hypertension; or ·\tunrepaired congenital heart defects\n- Hypersensitivity to the active substance or other ingredients like: hydroxypropyl methylcellulose (HPMC), mannitol, colloidal silicon dioxide (highly dispersed)\n- Lithium therapy"}

Primary endpoints

• {"endpoint_text":"- Difference in the S´/RAAi ratio (measured by echocardiography) from baseline to week 4.","definition_or_measurement_approach":"Measured by echocardiography; change in S´/RAAi ratio from baseline to Week 4 (end of treatment)."}

Secondary endpoints

• {"endpoint_text":"- \tEchocardiography (RA Area, TAPSE/PASP, pericardial effusion, S´/RAAi) •\tcMRI (RV EF, SVI, RVESVI, PA flow for impedance calculation, RV and LV EDV, RV and LV ESV, RV and LS SV, RV and LV EF, RV and LV CO, RV and LV CO, RV and LV Myocardial Mass, LVEDVI, LVESVI, LVMI, RV and LV GLS, RV and LV T1, RV and LV T2, RV and LV GLE, PA Flow and Aortic Flow) •\tpulmonary hemodynamics via Swan-Ganz catheterization (mPAP, dPAP, sPAP, PAWP, cardiac output by Fick (direct or indirect), PVR, SvO2, CVP/RAP, Ca","definition_or_measurement_approach":"Echocardiography and cMRI measurements as listed; invasive pulmonary hemodynamics via Swan-Ganz catheterization measuring mPAP, dPAP, sPAP, PAWP, cardiac output (Fick), PVR, SvO2, CVP/RAP, etc."}
• {"endpoint_text":"- \tbody plethysmography •\tFEV1, FEV1%pred, FVC, FVCpred, TLC%pred, DLCO%pred, FVC obs.v., FEV1 obs.v., TLC obs. V., TLC%pred, RV obs. V., VC obs. V., DLCO, sRaw effektiv, •\tBlood gas analysis: Capillary pO2, Capillary pCO2","definition_or_measurement_approach":"Pulmonary function testing by body plethysmography (FEV1, FVC, TLC, DLCO and derived %pred values) and capillary blood gas analysis (pO2, pCO2)."}
• {"endpoint_text":"- \tSix minutes walking distance (6MWD) •\tBNP •\tCPET (VO2 and VE/VCO2 slope, as well as WHO FC)","definition_or_measurement_approach":"Functional exercise capacity measured by 6MWD; biomarker BNP; cardiopulmonary exercise testing (CPET) measures VO2, VE/VCO2 slope and assessment of WHO Functional Class."}
• {"endpoint_text":"- \tPatient-reported outcome measures (PAH-Sympact, MLC questionnaire, EQ-5D-5L Mobility Score)","definition_or_measurement_approach":"Patient-reported outcome instruments including PAH-Sympact, MLC questionnaire and EQ-5D-5L Mobility Score as specified."}
• {"endpoint_text":"- \tAdverse event (AE), Serious (S) AE assessment","definition_or_measurement_approach":"Safety monitoring including recording and assessment of adverse events (AEs) and serious adverse events (SAEs) according to standard definitions."}

Germany

Earliest CTIS Part Ii Submission Date

08-08-2025

Latest Decision Or Authorization Date

05-05-2026

Processing Time Days

270

Number Of Sites

2

Number Of Participants

34

Primary sponsor

Full Name

Philipps-Universitaet Marburg

Organisation Type

Educational Institution

Country Of Registered Address

Germany

Third parties

• {"country":"Germany","full_name":"Universitaetsklinikum Erlangen AöR","duties_or_roles":"Sponsor duty code: 14 (as listed in sponsorDuties)","organisation_type":"Hospital/Clinic/Other health care facility"}