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A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

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Conditions

PAHPulmonary HypertensionPulmonary Arterial HypertensionHypertensionConnective Tissue DiseasesFamilial Primary Pulmonary HypertensionVascular DiseasesCardiovascular DiseasesHypertension, PulmonaryLung DiseasesRespiratory Tract DiseaseProstacyclinConnective Tissue Disease-Associated6 Minute Walk Test6 Minute Walk DistancePulmonary Vascular ResistanceRight Ventricular Function

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1 PAH.

Official Title

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Ralinepag When Added to PAH Standard of Care or PAH Specific Background Therapy in Subjects With WHO Group 1 PAH

Quick Facts

Study Start:2018-08-30
Study Completion:2025-12-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:COMPLETED

Study ID

NCT03626688

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. At least 18 years of age.
  2. 2. Evidence of a personally signed and dated informed consent form indicating that the subject has been informed of all pertinent aspects of the study prior to initiation of any study-related procedures.
  3. 3. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures
  4. 4. Primary diagnosis of symptomatic PAH.
  5. 5. Has had a right heart catheterization (RHC) performed at or within 3 years prior to Screening (RHC will be performed during Screening if not available) that is consistent with the diagnosis of PAH.
  6. 6. Has WHO/ NYHA functional class II to IV symptoms.
  7. 7. If on PAH-specific background oral therapy, subject is on stable therapy with either an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 inhibitor (PDE5-I) or a soluble guanylate cyclase (sGC) stimulator.
  8. 8. Has a 6MWD of ≥150 meters.
  9. 9. If taking concomitant medications that may affect the clinical manifestations of PAH (eg, calcium channel blockers, diuretics, digoxin, or L arginine supplementation, beta blockers, angiotensin-converting enzyme inhibitors, or angiotensin II receptor blockers), must be on a stable dose for at least 30 days prior to the Baseline Visit and the dosage maintained throughout the study. The exception is that the dose of diuretics must be stable for at least the 10 days prior to Baseline.
  10. 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 30-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 last dose of IMP. Eligible male subjects must agree not to participate in sperm donation for 90 days after the last dose of IMP.
  1. 1. For subjects with known HIV-associated PAH, a cluster designation 4 (CD4+) T-cell count \<200/mm3 within 90 days of Baseline.
  2. 2. Must not have 3 or more left ventricular dysfunction risk factors as defined in the study protocol.
  3. 3. Has evidence of more than mild lung disease on pulmonary function tests performed within 180 days prior to, or during Screening.
  4. 4. Has evidence of thromboembolic disease as determined by a V/Q lung scan or local standard of care diagnostic evaluation at or after diagnosis of PAH.
  5. 5. Current diagnosis of ongoing and clinically significant sleep apnea as defined by the Investigator.
  6. 6. Male subjects with a corrected QT interval using Fridericia's formula (QTcF) \>450 msec and female subjects with a QTcF \>470 msec on ECG recorded at Screening and analyzed by the central ECG laboratory. Subjects with evidence of intraventricular conduction delay, defined as a QRS interval greater than 110 msec, will be excluded if the QTcF is \>500 msec for both males and females.
  7. 7. Severe chronic liver disease (ie, Child-Pugh Class C), portal hypertension, cirrhosis or complications of cirrhosis/portal hypertension (eg, history of variceal hemorrhage, encephalopathy).
  8. 8. Confirmed active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV).
  9. 9. Subjects with alanine aminotransferase or aspartate aminotransferase ≥3 times the upper limit of normal (ULN) or total bilirubin ≥2 × ULN at Screening.
  10. 10. Chronic renal insufficiency as defined by serum creatinine \>2.5 mg/dL or requiring dialysis at Screening.
  11. 11. Hemoglobin concentration \<9 g/dL at Screening.
  12. 12. Subjects treated with an IV or SC prostacyclin pathway agent (eg, epoprostenol, treprostinil, or iloprost) or activin signaling inhibitor for PAH at any time prior to Baseline (use in vasoreactive testing is permitted).
  13. 13. Subjects currently on or who were treated with an inhaled or oral prostacyclin pathway agent (iloprost, treprostinil, beraprost, or selexipag) for \>6 months or within 90 days prior to Baseline.
  14. 14. Subject has pulmonary veno-occlusive disease.
  15. 15. Malignancy diagnosed and/or treated within 5 years prior to 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.
  16. 16. 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. A subject will not be excluded due to a positive drug screen caused by prescribed medications.
  17. 17. Initiation or discontinuation of a cardio-pulmonary rehabilitation program based upon exercise within 90 days prior to Screening and/or planned during study participation.
  18. 18. Prior participation in any study of ralinepag or participation in another interventional clinical study with medicinal products within 30 days prior to Screening. Concurrent participation in registry or observational studies is allowed, as long as the subject can fulfill all other entry criteria and comply with all study procedures.
  19. 19. Any reason that, in the opinion of the Investigator or Medical Monitor, 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 or impair study participation or cooperation.
  20. 20. Known hypersensitivity to ralinepag or any of the excipients.
  21. 21. Life expectancy \<12 months based on the Investigator's opinion.
  22. 22. Women who are pregnant, lactating or breast-feeding.

Contacts and Locations

Study Locations (Sites)

University of Alabama at Birmingham
Birmingham, Alabama, 35294
United States
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, 85013
United States
University of Arizona
Tucson, Arizona, 85724
United States
UCSD Health Sciences
La Jolla, California, 92037
United States
Loma Linda University Medical Center
Loma Linda, California, 92354
United States
Ronald Reagan UCLA Medical Center
Los Angeles, California, 90024
United States
Kaiser Permanente Los Angeles Medical Center
Los Angeles, California, 90027
United States
Keck Hospital of USC
Los Angeles, California, 90033
United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048
United States
VA Greater Los Angeles Healthcare System
Los Angeles, California, 90073
United States
University of California, Irvine
Orange, California, 19103
United States
University of California Davis Medical Center
Sacramento, California, 95817
United States
SBPA Research LLC
Santa Barbara, California, 93105
United States
Stanford Healthcare
Stanford, California, 94305
United States
LA Biomedical Research Institute Harbor-UCLA Medical Center
Torrance, California, 90502
United States
University of Colorado Hospital
Aurora, Colorado, 80045
United States
National Jewish Health
Denver, Colorado, 80206
United States
University of Florida
Gainesville, Florida, 32610
United States
University of Miami
Miami, Florida, 33136
United States
Central Florida Pulmonary Group
Orlando, Florida, 32803
United States
Cleveland Clinic Florida
Weston, Florida, 33331
United States
Emory University Hospital
Atlanta, Georgia, 30322
United States
Piedmont Healthcare Pulmonary and Critical Care Research
Austell, Georgia, 30106
United States
University of Chicago Medical Center
Chicago, Illinois, 60637
United States
Ascension Alexian Brothers
Elk Grove Village, Illinois, 60007
United States
Indiana University School of Medicine
Indianapolis, Indiana, 46202
United States
Community Health Network Cancer Center North
Indianapolis, Indiana, 46250
United States
St. Vincent Medical Group, Inc.
Indianapolis, Indiana, 46260
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
United States
Kentuckiana Pulmonary Research Center
Louisville, Kentucky, 40202
United States
Ochsner Medical Center
New Orleans, Louisiana, 70121
United States
Chest Medicine Associates
South Portland, Maine, 04106
United States
University of Maryland Medical Center
Baltimore, Maryland, 21201
United States
Tufts Medical Center
Boston, Massachusetts, 02111
United States
Massachusetts General Hospital
Boston, Massachusetts, 02114
United States
Boston University Medical Center
Boston, Massachusetts, 02118
United States
Spectrum Health Medical Group
Grand Rapids, Michigan, 49546
United States
Washington University School of Medicine
St Louis, Missouri, 63110
United States
Nebraska Medical Center
Omaha, Nebraska, 68198
United States
University of New Mexico
Albuquerque, New Mexico, 87131
United States
Winthrop Hospital
Mineola, New York, 11501
United States
NYU Langone Medical Center
New York, New York, 10016
United States
Weill-Cornell-New York Presbyterian Hospital
New York, New York, 10021
United States
Mount Sinai School of Medicine
New York, New York, 10029
United States
University of Rochester
Rochester, New York, 14642
United States
Duke University Medical Center
Durham, North Carolina, 27710
United States
East Carolina University
Greenville, North Carolina, 27834
United States
University of Cincinnati-Medical Science Building
Cincinnati, Ohio, 45267
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
United States
Integris Baptist Medical Center
Oklahoma City, Oklahoma, 73112
United States
Oregon Clinic-Pulmonary West
Portland, Oregon, 97225
United States
Oregon Health & Science University
Portland, Oregon, 97239
United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
United States
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
United States
Temple University
Philadelphia, Pennsylvania, 19140
United States
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
United States
Statcare Pulmonary Consultants
Knoxville, Tennessee, 37919
United States
Ascension Texas Cardiovascular
Austin, Texas, 78705
United States
Baylor University Medical Center
Dallas, Texas, 75246
United States
UT Southwestern Medical Center
Dallas, Texas, 75390
United States
Memorial Hermann Hospital
Houston, Texas, 77030
United States
The Methodist Hospital Research Institute
Houston, Texas, 77030
United States
Vermont Lung Center
Colchester, Vermont, 05446
United States
University of Virginia Medical Center
Charlottesville, Virginia, 22908
United States
Sentara Cardiovascular Research Institute
Norfolk, Virginia, 23507
United States
Carilion Clinic Pulmonary and Sleep Medicine
Roanoke, Virginia, 24014
United States
Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin, 53226
United States

Collaborators and Investigators

Sponsor: United Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-08-30
Study Completion Date2025-12-31

Study Record Updates

Study Start Date2018-08-30
Study Completion Date2025-12-31

Terms related to this study

Keywords Provided by Researchers

  • Prostacyclin
  • Connective Tissue Disease-Associated
  • 6 Minute Walk Test
  • 6 Minute Walk Distance
  • Pulmonary Vascular Resistance
  • Right Ventricular Function

Additional Relevant MeSH Terms

  • PAH
  • Pulmonary Hypertension
  • Pulmonary Arterial Hypertension
  • Hypertension
  • Connective Tissue Diseases
  • Familial Primary Pulmonary Hypertension
  • Vascular Diseases
  • Cardiovascular Diseases
  • Hypertension, Pulmonary
  • Lung Diseases
  • Respiratory Tract Disease