New drug aims to help lungs and heart work better during exercise

NCT ID NCT04084678

Disease control Sponsor: United Therapeutics Source: ClinicalTrials.gov ↗

First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time

Summary

This phase 3 trial tests whether ralinepag, an oral medication, can improve exercise capacity in people with pulmonary arterial hypertension (PAH) who have recently started PAH therapy. Participants take ralinepag or a placebo for 28 weeks, and their peak oxygen consumption during exercise is measured. The study focuses on safety and how well the drug helps the heart and lungs work during physical activity.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Ralinepag (oral drug)
What this could lead to: If successful, ralinepag could become a new treatment option to improve exercise ability and daily function in people with pulmonary arterial hypertension.
What could go wrong: This is a small, early-phase study, and results may not confirm benefit. Side effects or lack of improvement in exercise capacity are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

10 people

The number who actually took part.

Started

Jan 2021

Finished

Apr 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Signed informed consent form. 2. At least 18 years of age. 3. Primary diagnosis of PAH. 4. Has had a diagnostic RHC performed at or within 3 years before Screening (or at Screening if one is not available) that is consistent with the diagnosis of PAH. 5. Has World Health Organization (WHO)/New York Heart Association (NYHA) Functional Class (FC) II to III symptoms 6. Must be on a stable dose of PAH-specific oral therapy, defined as no change in dose or regimen for at least 90 days prior to randomization. Allowable PAH-specific therapy is an endothelin receptor antagonist and/or a phosphodiesterase type 5 inhibitor (PDE5-I) or a soluble guanylate cyclase (sGC) stimulator. Subjects may be on a stable dose of either a PDE5-I or a sGC stimulator, not both. 7. Has a 6-minute walk distance (6MWD) of ≥150 meters at Screening. 8. Has a peak VO2 of ≥9 to \<18 mL/min/kg during the Screening CPET, as assessed by the CPET core laboratory. 9. If the subject is taking concomitant medications that may affect the clinical manifestations of PAH, the subject must be on a stable dose for at least 30 days prior to randomization. The exception is that the dose of diuretics must be stable for at least the 10 days prior to randomization. 10. Both male and female subjects agree to use a highly effective method of birth control throughout the entire study period from informed consent through to the Week 28 Visit/28-day Follow-up Visit, if the possibility of conception exists. Eligible male and female subjects must also agree not to participate in a conception process during the study and for 30 days after the final dose of study drug. Eligible male subjects must agree not to participate in sperm donation for 90 days after the final dose of study drug. Women who are surgically sterile or postmenopausal are not considered to be of childbearing potential. If of childbearing potential, female partners of male study participants should agree to utilize medically acceptable methods of contraception for the duration of study participation. Exclusion Criteria: 1. For subjects with known human immunodeficiency virus-associated PAH, a cluster of differentiation 4 T-cell count \<200/mm3 at Screening. 2. Has 3 or more left ventricular disease/dysfunction risk factors. 3. Symptomatic coronary artery disease and/or myocardial infarction within past 6 months. 4. Current symptomatic aortic or mitral valve disease. 5. Has evidence of more than mild lung disease on pulmonary function tests performed within 1 year prior to, or during, Screening. 6. Has evidence of thromboembolic disease as determined by ventilation-perfusion lung scan or local standard of care diagnostic evaluation at or after diagnosis of PAH. 7. Current diagnosis of ongoing and clinically significant sleep apnea as defined by the Investigator. 8. Requires use of supplemental oxygen during CPET. 9. Respiratory exchange ratio \<1.0 at Screening CPET as determined by the CPET core laboratory. 10. Acute non-cardiac disorder that may affect exercise performance or be aggravated by exercise (eg, infection, renal failure, thyrotoxicosis). 11. Male subjects with a QTcF \>450 msec and female subjects with a QTcF \>470 msec on electrocardiogram (ECG) recorded at Screening and analyzed by the central ECG laboratory. Subjects with evidence of intraventricular conduction delay, defined as a QRS interval \>110 msec, will be excluded if QTcF is \>500 msec for both males and females. 12. Severe chronic liver disease (ie, Child-Pugh Class C), portal hypertension, cirrhosis, or complications of cirrhosis/portal hypertension (eg, history of variceal hemorrhage, encephalopathy). 13. Confirmed active infection with hepatitis B virus or hepatitis C virus. 14. Subjects with alanine aminotransferase or aspartate aminotransferase ≥3 times the upper limit of normal or total bilirubin ≥2 times the upper limit of normal at Screening. 15. Chronic renal insufficiency as defined by an estimated glomerular filtration rate using the Modification of Diet in Renal Disease Study equation of \<30 mL/min/1.73 m2 or requiring dialysis at Screening. 16. Hemoglobin concentration \<9 g/dL at Screening. 17. Subjects treated with an intravenous, subcutaneous, inhaled, or oral prostacyclin pathway agent (eg, epoprostenol, treprostinil, iloprost, beraprost, or selexipag) for PAH within 90 days of randomization (use in vasoreactive testing is permitted). Subject is not eligible if previous prostacyclin therapy was stopped for a safety or tolerability issue related to systemic prostacyclin adverse effects. 18. Subject has pulmonary veno-occlusive disease. 19. Malignancy diagnosed and/or treated within 3 years of Screening, with the exception of localized non-metastatic basal cell or squamous cell carcinoma of the skin or in-situ carcinoma of the cervix excised with curative intent. 20. Subject tests positive for amphetamine, cocaine, methamphetamine, methylenedioxymethamphetamine, or phencyclidine in urine drug screen performed at Screening, or has a recent history (6 months) of alcohol or drug abuse. Subjects will not be excluded due to a positive drug screen caused by prescribed medications. 21. Initiation or discontinuation of a cardio-pulmonary rehabilitation program based upon exercise within 90 days prior to Screening and/or planned during study participation. 22. Prior participation in any study of ralinepag or another interventional clinical study with medicinal products within 30 days prior to Screening. Concurrent participation in registry or observational studies is allowed, if the subject can fulfill all other entry criteria and comply with all study procedures. 23. Any reason that, in the opinion of the Investigator, precludes the subject from participating in the study (eg, any previous or intercurrent medical condition) that may increase the risk associated with study participation or that would confound study analysis (eg, right-to-left shunt detected during CPET) or impair study participation or cooperation. 24. Known hypersensitivity to ralinepag or any of the excipients. 25. Life expectancy \<12 months based on the Investigator's opinion. 26. Women who are pregnant, lactating, or breast-feeding.

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Conditions

The condition(s) this trial relates to.

pulmonary arterial hypertension pulmonary hypertension

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Pulmonary arterial hypertension Pulmonary hypertension

Contacts and locations

Locations

AKH Wien, Innere Med. II, Kardiologie

Vienna, 1090, Austria
AOU Policlinico Umberto I

Rome, 00161, Italy
Banner University Medical Center (University of Arizona)

Tucson, Arizona, 85724, United States
Centro Cardiologico Monzino, IRCCS

Milan, 20138, Italy
Centro de Hipertensão Pulmonar

Porto Alegre, 90035074, Brazil
Erasme University Hospital - Department of Cardiology

Brussels, 1070, Belgium
Europejskie Centrum Zdrowia Otwock Szpital im. Fryderyka Chopina, Oddział Kardiologiczny

Otwock, 05-400, Poland
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy
Gasthuisberg University Hospital - Department of Pulmonology

Leuven, 3000, Belgium
Hospital Britanico de Buenos Aires

Ciudad Autónoma de Bs. As., 1280, Argentina
Hospital Clínic I Provincial de Barcelona

Barcelona, 08036, Spain
Hospital Madre Teresa

Belo Horizonte, Minas Gerais, 30441-070, Brazil
Hospital Sao Paulo

São Paulo, São Paulo, 04037-002, Brazil
Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain
Hospital Universitario 12 de Octubre

Madrid, 28041, Spain
Imperial college Healthcare NHS Trust

London, W12 0HS, United Kingdom
Instituto de Cardiología de Corrientes

Corrientes, W3400AMZ, Argentina
Instituto do Coração do Hospital das Clínicas da Faculdade de Medicina de São Paulo - InCor-HCFMUSP

São Paulo, São Paulo, 05403-000, Brazil
London Health Science Centre- Victoria Hospital

London, Ontario, N6A 5W9, Canada
Macquarie University

North Ryde, New South Wales, 2109, Australia
Medical College of Wisconsin/Froedtert Hospital

Milwaukee, Wisconsin, 53226, United States
National Jewish Health

Denver, Colorado, 80206, United States
Ordensklinikum Linz GmbH, Elisabethinen

Linz, 4020, Austria
Peter Lougheed Center

Calgary, Alberta, T1Y 6J4, Canada
Princess Alexandra Hospital

Woolloongabba, Queensland, 4102, Australia
Tampa General Hospital/University of South Florida Center for Advanced Lung Disease and Lung Transplant

Tampa, Florida, 33606, United States
The Prince Charles Hospital

Chermside, Queensland, 4032, Australia
Thoraxklinik-Heidelberg, Zentrum für Pulmonale Hypertonie

Heidelberg, Baden-Wurttemberg, 69126, Germany
University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada
University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States
Universitätsklinikum Carl Gustav Carus TU Dresden, Medizinische Klinik I, Abteilung für Pneumologie

Dresden, Saxony, 01307, Germany
Uniwersytecki Szpital Kliniczny w Białymstoku, Klinika Kardiologii z Oddziałem Intensywnego Nadzoru Kardiologicznego

Bialystok, 15-276, Poland
Westmead Hospital, Dept Respiratory and Sleep Medicine

Westmead, New South Wales, 2145, Australia

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