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

  • STATUS
    Recruiting
  • End date
    Dec 23, 2022
  • participants needed
    700
  • sponsor
    United Therapeutics
Updated on 22 September 2021
hypertension
calcium
beta blockers
diuretics
beta-blocker
angiotensin
arginine
angiotensin ii
calcium channel blockers
digoxin
pulmonary arterial hypertension
right heart catheterization
endothelin
ralinepag
endothelin receptor antagonist

Summary

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.

Description

Study ROR-PH-301 is a multicenter, randomized, double-blind, placebo-controlled study. Subjects who meet entry criteria will be randomly allocated 1:1 to receive ralinepag or placebo, in addition to their standard of care or PAH-specific background therapy, as applicable. The primary endpoint is the time (in days) from randomization to the first adjudicated protocol-defined clinical worsening event. All primary endpoint events will be adjudicated by an independent Clinical Event Committee (CEC) in a blinded fashion. Subjects who have a confirmed primary endpoint event adjudicated by the CEC at any time during the study and all subjects on treatment at the conclusion of the study (after the target number of events is achieved) will have the option to enroll in an open-label extension (OLE) study. Subjects who do not choose to participate in the OLE study will discontinue study drug, and should remain in the study for long-term follow-up of survival status and will receive standard of care PAH treatment, at the discretion of the treating physician.

Details
Condition Pulmonary Disease, Vascular Diseases, CONNECTIVE TISSUE DISEASE, Respiratory Tract Diseases, Hypertension, Pulmonary Hypertension, Cardiovascular Disease, polycyclic aromatic hydrocarbons, Cancer Prevention, Surviving Abuse, Joint Injuries, Abdominal Surgery, Mental Disability, Pelvic Adhesions, Low Testosterone, Dental Filling, Habit Reversal, Complicated Grief, Diabetes and Hypertension, Chronic Pelvic Pain, Gambling Problems, Myopic Macular Degeneration, Nerve Injury, Severe Premenstrual Symptom, High Blood Pressure (Hypertension), High Blood Pressure (Hypertension - Pediatric), Dermatomyositis (Connective Tissue Disease), Stasis Dermatitis, Pulmonary Arterial Hypertension, Open Heart Surgery, Recurrent Pregnancy Loss, Effects of Chemotherapy, Renal Anemia, Functional Dyspepsia, Catheter Complications, Serial Evaluation of Ductal Epithelium, Chronic Renal Anemia, Cancer Treatment, Anemic Cancer, Spinocerebellar Disorders, Pseudobulbar Affect, Spine Athroplasty, Indikation: Diabetes - Typ II, Partial Medial Meniscectomy, Primary Insulin Hypersecretion, Testotoxikose, Infantile Fibrosarcoma, Late Infantile Neuronal Ceroid Lipfuscinsosis, Elevated Blood Pressure, Familial Primary Pulmonary Hypertension, Connective Tissue Diseases, Lung Disease, Memory Problems, vascular disorder, connective tissue disorder, p-aminohippuric acid, hpah, high blood pressure, arterial hypertension, pulmonary diseases, lung diseases, pulmonary disorders, cardiovascular diseases, cardiovascular disease (cvd), cardiovascular system diseases, cardiovascular disorders, vascular disease, vasculopathy, vasc, respiratory tract disease
Treatment Placebo, Placebo tablet, Ralinepag Tablet, Extended Release, Ralinepag
Clinical Study IdentifierNCT03626688
SponsorUnited Therapeutics
Last Modified on22 September 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

At least 18 years of age
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
Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures
Primary diagnosis of symptomatic PAH
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
Has WHO/ NYHA functional class II to IV symptoms
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. Subjects may be nave to PAH-specific treatment
Has a 6MWD of 150 meters
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
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

Exclusion Criteria

For subjects with known HIV-associated PAH, a cluster designation 4 (CD4+) T-cell count <200/mm3 within 90 days of Baseline
Must not have 3 or more left ventricular dysfunction risk factors as defined in the study protocol
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
Has evidence of more than mild lung disease on pulmonary function tests performed within 180 days prior to, or during Screening
Current diagnosis of ongoing and clinically significant sleep apnea as defined by the Investigator
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
Confirmed active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV)
Severe chronic liver disease (ie, Child-Pugh Class C), portal hypertension, cirrhosis or complications of cirrhosis/portal hypertension (eg, history of variceal hemorrhage, encephalopathy)
Chronic renal insufficiency as defined by serum creatinine >2.5 mg/dL or requiring dialysis at Screening
Hemoglobin concentration <9 g/dL at Screening
Subjects with alanine aminotransferase or aspartate aminotransferase 3 times the upper limit of normal (ULN) or total bilirubin 2 ULN at Screening
Subject has pulmonary veno-occlusive disease
Subjects treated with an IV or SC prostacyclin pathway agent (eg, epoprostenol, treprostinil, or iloprost) for PAH at any time prior to Baseline (use in vasoreactive testing is permitted)
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
Subjects currently on or who were treated with an inhaled or oral prostacyclin pathway agent (iloprost, treprostinil, beraprost, or selexipag) within 90 days prior to Baseline
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
Initiation or discontinuation of a cardio-pulmonary rehabilitation program based upon exercise within 90 days prior to Screening and/or planned during study participation
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
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
Known hypersensitivity to ralinepag or any of the excipients
Life expectancy <12 months based on the Investigator's opinion
Women who are pregnant, lactating or breast-feeding
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