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A Study of a Mean Pulmonary Artery Pressure-Targeted Approach With Early and Rapid Treprostinil Therapy to Reverse Right Ventricular Remodeling in Participants With Pulmonary Arterial Hypertension

Phase 4
Recruiting
Conditions
Pulmonary Arterial Hypertension
Interventions
Drug: Parenteral Treprostinil
Drug: Oral Treprostinil
Registration Number
NCT05203510
Lead Sponsor
United Therapeutics
Brief Summary

The primary objective of this study is to assess the effect of early and rapid treprostinil therapy for mean pulmonary artery pressure (mPAP) reduction to improve right ventricular (RV) function and reverse RV remodeling in participants with pulmonary arterial hypertension (PAH).

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
50
Inclusion Criteria
  • Confirmed PAH (WHO Group 1) classified by one of the following subgroups:

    • Idiopathic, heritable or drug/toxin induced (with the exception of amphetamine-induced PAH)
    • Associated with repaired congenital systemic-to-pulmonary shunts (repaired ≥1 year)
    • Associated with connective tissue disease
    • Associated with human immunodeficiency virus infection
  • Baseline visit right heart catheterization (RHC) must also meet the following criteria:

    • mPAP >35 mmHg
    • Pulmonary vascular resistance (PVR) >2 Wood units
    • Pulmonary artery wedge pressure (PAWP) ≤15 mmHg
  • On a stable dose of an endothelin receptor antagonist (ERA) and/or phosphodiesterase type 5 inhibitor (PDE-5i) or soluble guanylate cyclase stimulator (sGC) therapy or if treatment naïve, willing to take one of these medications in addition to study drug

  • REVEAL Lite 2 risk score ≤9

  • WHO FC II or III

  • 6MWD >165 meters

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Exclusion Criteria

PAH-related Exclusion Criteria:

  • Prior or current use of epoprostenol, treprostinil, iloprost, beraprost, or selexipag
  • Positive vasoreactivity test in idiopathic, heritable, or drug/toxin induced PAH
  • Amphetamine use within the past 12 months
  • WHO Groups 2, 3, 4, and 5
  • Use of any other investigational drug, device, or therapy within 30 days of the Baseline visit
  • Moderate or severe hepatic impairment (Child-Pugh Class B and C)
  • Any other clinically significant illness or abnormal laboratory value(s) measured during screening that, in the opinion of the Investigator, might adversely affect interpretation of the study data or participant safety (for example, active infection, chronic thromboembolic pulmonary hypertension, or acute/recent deep vein thrombosis or pulmonary embolism)
  • Chronic atrial fibrillation, multiple premature ventricular or atrial contractions of clinical significance, or any other condition that would interfere with proper cardiac gating during cMRI
  • Permanent cardiac pacemaker or automatic internal cardioverter that would interfere with conduct of cMRI
  • Metallic implant (for example, defibrillator, neurostimulator, hearing aid, permanent infusion device, implantable pump, or body plates/screws/bolts) that would interfere with conduct of cMRI

CardioMEMS-related Exclusion Criteria, if applicable:

  • Previously implanted with CardioMEMS pulmonary artery Sensor or unwilling/unable to permit collection and perform upload (transmission) of pulmonary artery pressure (PAP) readings
  • Unable to take dual antiplatelet or anticoagulation therapy for 30 days after CardioMEMS PA Sensor implantation unless the participant has an indication for warfarin or direct oral anticoagulant

NOTE: Other inclusion and exclusion criteria may apply.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
TreprostinilParenteral TreprostinilParticipants will receive parenteral treprostinil at initial dose of 1.25 nanograms/kilogram/minute (at minimum) either intravenously or subcutaneously. Based on mPAP assessments and after a minimum dose of parenteral treprostinil is reached, at Investigator's (PI's) discretion, participants may transition to oral treprostinil and continue dose uptitration for further reduction of mPAP. Based on Month 12 mPAP assessment, participants may transition from parenteral to oral treprostinil at PI's discretion after completion of Month 12 assessment and continue uptitration for further reduction of mPAP. If minimum dose of parenteral treprostinil is not reached at Month 6/12 at PI's discretion, uptitration of parenteral treprostinil or oral treprostinil transition may occur to maintain normal mPAP. Treprostinil therapy (parenteral or oral) may continue as tolerated toward goal of further reduction of mPAP until Month 36.
TreprostinilOral TreprostinilParticipants will receive parenteral treprostinil at initial dose of 1.25 nanograms/kilogram/minute (at minimum) either intravenously or subcutaneously. Based on mPAP assessments and after a minimum dose of parenteral treprostinil is reached, at Investigator's (PI's) discretion, participants may transition to oral treprostinil and continue dose uptitration for further reduction of mPAP. Based on Month 12 mPAP assessment, participants may transition from parenteral to oral treprostinil at PI's discretion after completion of Month 12 assessment and continue uptitration for further reduction of mPAP. If minimum dose of parenteral treprostinil is not reached at Month 6/12 at PI's discretion, uptitration of parenteral treprostinil or oral treprostinil transition may occur to maintain normal mPAP. Treprostinil therapy (parenteral or oral) may continue as tolerated toward goal of further reduction of mPAP until Month 36.
Primary Outcome Measures
NameTimeMethod
Change From Baseline in Right Ventricular Ejection Fraction (RVEF), as Measured by Cardiac Magnetic Resonance Imaging (cMRI) at Month 12Baseline, Month 12
Secondary Outcome Measures
NameTimeMethod
Change From Baseline in NT-proBNP at Months 12, 24, and 36Baseline, Months 12, 24, and 36
Change From Baseline in Borg Dyspnea Score at Months 12, 24, and 36Baseline, Months 12, 24, and 36
Change From Baseline in mPAP at Month 12Baseline, Month 12
Number of Participants With Clinical Improvement From Baseline to Month 12, 24, and 36Baseline to Months 12, 24, and 36

Clinical improvement is defined as meeting all 3 criteria: - improvement in six-minute walk distance (6MWD) increase ≥10% or ≥30 meters; - improvement in World Health Organization (WHO) functional class (FC) or maintenance of WHO FC I/II; - improvement in N-terminal pro-brain natriuretic peptide (NT-proBNP) decrease ≥30% or \<300 ng/liter (L).

Change From Baseline in RV-Pulmonary Artery (PA) Coupling Estimated by the Ratio of Stroke Volume by End Systolic Volume at Month 12Baseline, Month 12
Change From Baseline in RV End-Diastolic Volume Index at Month 12Baseline, Month 12
Change From Baseline in RV Stroke Volume Index at Month 12Baseline, Month 12
Change From Baseline in 6MWD at Month 12, 24, and 36Baseline, Months 12, 24, and 36
Change From Baseline in Registry to EValuate EArly and Long-Term PAH Disease Management (REVEAL) Lite 2 Risk Score at Months 12, 24, and 36Baseline, Months 12, 24, and 36
Change From Baseline in WHO FC at Months 12, 24, and 36Baseline, Months 12, 24, and 36
Change From Baseline in RV-PA Coupling Estimated by the Ratio of Tricuspid Annular Plane Systolic Excursion by Pulmonary Artery Systolic Pressure (TAPSE/PASP) at Months 12, 24, and 36Baseline, Months 12, 24, and 36
Survival Rate: Number of Participants who Survived at Months 12, 24, and 36Baseline to Months 12, 24, and 36
Change From Baseline in mPAP at Months 24 and 36Baseline, Months 24 and 36

Trial Locations

Locations (28)

Banner University Medical Center (University of Arizona)

🇺🇸

Phoenix, Arizona, United States

HonorHealth John C. Lincoln Medical Center

🇺🇸

Phoenix, Arizona, United States

University of California San Francisco - Fresno

🇺🇸

Fresno, California, United States

Cedars-Sinai Medical Center

🇺🇸

Los Angeles, California, United States

University of California Davis Medical Center

🇺🇸

Sacramento, California, United States

University of California San Francisco Pulmonary, Critical Care, Allergy and Sleep Medicine

🇺🇸

San Francisco, California, United States

Hartford Hospital

🇺🇸

Hartford, Connecticut, United States

USF

🇺🇸

Tampa, Florida, United States

Georgia Clinical Research

🇺🇸

Austell, Georgia, United States

Northwestern Memorial Hospital

🇺🇸

Chicago, Illinois, United States

Indiana University Health North Hospital

🇺🇸

Indianapolis, Indiana, United States

Community Health Network

🇺🇸

Indianapolis, Indiana, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Henry Ford Health System

🇺🇸

Detroit, Michigan, United States

University of Nebraska Medical Center

🇺🇸

Omaha, Nebraska, United States

Morristown Medical Center

🇺🇸

Morristown, New Jersey, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

University of North Carolina at Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

University of Cincinnati

🇺🇸

Cincinnati, Ohio, United States

The Ohio State University Wexner Medical Center

🇺🇸

Columbus, Ohio, United States

Integris Baptist Medical Center

🇺🇸

Oklahoma City, Oklahoma, United States

Oklahoma Heart Institute

🇺🇸

Tulsa, Oklahoma, United States

Temple Hospital

🇺🇸

Philadelphia, Pennsylvania, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Prisma Health

🇺🇸

Greenville, South Carolina, United States

UT Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Carilion Clinic

🇺🇸

Roanoke, Virginia, United States

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