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Trial to Evaluate Parenteral Treprostinil and Riociguat on Right Ventriculo-vascular Coupling and Morphology in Those With Advanced PAH

Phase 3
Recruiting
Conditions
Pulmonary Arterial Hypertension
Interventions
Registration Number
NCT04062565
Lead Sponsor
University of Arizona
Brief Summary

The purpose of this study is to determine if there is a greater effect to patients with advanced pulmonary arterial hypertension (PAH) by using a combination of two drugs, Treprostinil and Riociquat versus Treprostinil alone

Detailed Description

The purpose of this study is to evaluate the combined effect of parenteral treprostinil (TRE) and riociguat (RIO) versus parenteral TRE alone on right ventricular (RV)-pulmonary artery (PA) interaction (RVPA coupling) and global RV function in patients with advanced pulmonary arterial hypertension (PAH).

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
20
Inclusion Criteria
  • WHO Category I PAH
  • Resting mPAP ≥ 25 mmHg with a wedge pressure of ≤ 15mmHg during right heart catheterization.
  • Need for parenteral TRE as determined by the PH specialist caring for the patient
Exclusion Criteria
  • Patients with a mean arterial pressure <60, and/or requiring vasopressor support

  • Patients whom expected device (i.e. ECMO, RVAD) assistance or early pulmonary transplantation (within 3 months) seems inevitable

  • Patients with a left ventricular ejection fraction <50% or clinical, echocardiographic, and/or catheterization data consistent with heart failure with preserved ejection fraction (HFpEF) and/or moderate-severe aortic or mitral valve abnormality

  • Patients with severe restrictive lung disease (FVC<70% predicted) and/or obstructive lung disease (FEV1 <70% predicted and FEV1/FVC <70%).

  • Patients with a history of pulmonary embolism within the last three months or evidence of chronic pulmonary embolism.

  • Patients with a known contraindication to right heart catheterization.

  • Patients whom have received active or previous pulmonary vasoactive medication within the previous 12 weeks.

  • PAH associated with significant venous or capillary involvement (PCWP > 15 mmHg), known pulmonary veno-occlusive disease, and pulmonary capillary hemangiomatosis.

  • Pulmonary Hypertension belonging to groups 2 to 5 of the WHO classification.

  • Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.

  • Estimated creatinine clearance < 30 mL/min

  • Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 1.5 times the upper limit of normal.

  • Hemoglobin < 75% of the lower limit of the normal range.

  • Acute or chronic physical impairment (other than dyspnea), limiting the ability to comply with study requirements.

  • Pregnant or breast-feeding.

    • Females must either abstain from intercourse (when it is in line with their preferred and usual lifestyle), or
    • Use 2 medically acceptable, highly effective forms of contraception for the duration of study, and at least 30 after discontinuing study drug.
  • Known concomitant life-threatening disease with a life expectancy < 12 months.

  • Body weight < 40 kg and/or >150 kg.

  • Any condition that prevents compliance with the protocol or adherence to therapy.

  • Concurrent therapy with strong CYP3A4 inhibitors/inducers (i.e. protease inhibitors, azole antibiotics, macrolides), theophylline, and any medication in the PI's opinion may substantially potentiate the hypotensive effect of RIO.

  • Treatment with nitrates of any kind within the 4 weeks prior to enrollment.

  • Known hypersensitivity to drugs of the same class as TRE and/or RIO, or any of their excipients.

  • Planned treatment, or treatment, with another investigational drug within 1 month prior to randomization.

  • Recent (<6 months) hemoptysis and/or history of severe hemoptysis requiring intervention (bronchial artery embolization).

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
ExperimentalRiociguat PillTreprostinil and Riociguat
ExperimentalTreprostinil Injectable ProductTreprostinil and Riociguat
Primary Outcome Measures
NameTimeMethod
Change in stroke volume/end systolic volume (SV/ESV)Baseline to 3 months

Change in stroke volume/end systolic volume (SV/ESV)

Secondary Outcome Measures
NameTimeMethod
Change in pulmonary and cardiac pressuresBaseline to 3 months

Increase or decrease in pressures

Change in pulmonary blood flowBaseline to 3 months

Increase or decrease in pulmonary blood flow

Change in derived VO2 max3 months

Change in how much oxygen the body consumes at peak exercise

Change in derived Ve/VCO23 months

Change in how much oxygen the body consumes at peak exercise

Change in adverse event profileBaseline to 3 months

Change in side effects or other adverse events between combination therapy and historical control

Change in end-systolic elastance/arterial elastance (Ees/Ea)Baseline to 3 months

Change in the interaction of the right heart and lung blood vessels

Change in Right Ventricle (RV) diastolic stiffness (Beta)Baseline to 3 months

Change in how stiff the wall of the right heart is at the end of relaxation

Change in 6 minute walk distanceBaseline to 3 months

Change in how far a participant can walk during a self paced 6 minute walk test

Change in brain natriuretic peptide (BNP)Baseline to 3 months

Change in biomarker BNP that examines stretch on the right heart

Change in magnetic resonance imaging (MRI) right ventricle volumesBaseline to 3 months

Change in the volume ejected per beat and the end systolic and diastolic values of the right heart

Change in composite time to clinical worseningBaseline to 3 months

Difference relative to historical control in the time from diagnosis to followup, hospitalization, death or transplant

Change in Cardio pulmonary Exercise Testing (CPET)3 months

Change in how much oxygen the body consumes at peak exercise

Trial Locations

Locations (1)

University of Arizona

🇺🇸

Tucson, Arizona, United States

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