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    Summary
    EudraCT Number:2018-001187-33
    Sponsor's Protocol Code Number:ROR-PH-301(APD811-301)
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-10-29
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSweden - MPA
    A.2EudraCT number2018-001187-33
    A.3Full title of the trial
    A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study evaluating the efficacy and safety of ralinepag in treatment of patients with pulmonary hypertension.
    A.3.2Name or abbreviated title of the trial where available
    ADVANCE-Outcomes
    A.4.1Sponsor's protocol code numberROR-PH-301(APD811-301)
    A.5.4Other Identifiers
    Name:IND NumberNumber:109021
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUnited Therapeutics Corporation
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportUnited Therapeutics Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUnited Therapeutics Corporation
    B.5.2Functional name of contact pointRegulatory Department
    B.5.3 Address:
    B.5.3.1Street Address55 T.W. Alexander Drive, PO Box 14186
    B.5.3.2Town/ cityResearch Triangle Park
    B.5.3.3Post codeNC 27709
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1(919) 485-8350
    B.5.5Fax number+1(919) 485-8352
    B.5.6E-mailinfo1@unither.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2130
    D.3 Description of the IMP
    D.3.1Product nameRalinepag
    D.3.2Product code APD811
    D.3.4Pharmaceutical form Prolonged-release tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNralinepag
    D.3.9.1CAS number 1187856-49-0
    D.3.9.2Current sponsor codeAPD811
    D.3.9.3Other descriptive nameAR392830
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2130
    D.3 Description of the IMP
    D.3.1Product nameRalinepag
    D.3.2Product code APD811
    D.3.4Pharmaceutical form Prolonged-release tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNralinepag
    D.3.9.1CAS number 1187856-49-0
    D.3.9.2Current sponsor codeAPD811
    D.3.9.3Other descriptive nameAR392830
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2130
    D.3 Description of the IMP
    D.3.1Product nameRalinepag
    D.3.2Product code APD811
    D.3.4Pharmaceutical form Prolonged-release tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNralinepag
    D.3.9.1CAS number 1187856-49-0
    D.3.9.2Current sponsor codeAPD811
    D.3.9.3Other descriptive nameAR392830
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    pulmonary arterial hypertension (PAH)
    E.1.1.1Medical condition in easily understood language
    high lung blood pressure
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10077731
    E.1.2Term Pulmonary hypertension WHO functional class I
    E.1.2System Organ Class 100000004855
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is:
    To demonstrate the effect of ralinepag on the time to first adjudicated protocol-defined clinical worsening event in subjects with PAH.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    1) To evaluate the effects of ralinepag from Baseline to Week 28 on:
    • N-terminal pro b-type natriuretic peptide (NT-proBNP)
    • 6 minute walk distance (6MWD)
    • WHO/ New York Heart Association (NYHA) functional class
    • Shift and proportion of subjects who attain all three of the following:
    o NT-proBNP <300 pg/mL
    o 6MWD >440 meters
    o WHO/NYHA functional class I or II
    • Health-related quality of life (HRQoL) measures
    • Heart rate recovery (HRR) following completion of the 6-minute walk test (6MWT)
    2) To evaluate the time to all-cause hospitalization
    3) To evaluate the time to all-cause mortality
    4) To evaluate the safety and tolerability of ralinepag in subjects with PAH
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional evaluation of proteomics, metabolomics, transcriptomics, and genetics:
    For subjects who agree to participate in the optional proteomics, metabolomics, and transcriptomics analyses, blood samples will be collected at Baseline, the Week 28 Visit, and the Study Drug Termination Visit; for subjects who agree to participate in the optional genetic analysis, a blood sample will be collected at Baseline. Comparing treatment responses and treatment-related change to these parameters may reveal important information with regard to: 1) the pathophysiology and natural history of PAH, 2) treatment response and effect of treatment on proteomics, and 3) ralinepag mechanism of action. Subjects can consent to participate in all, part, or none of these analyses, and subjects who do not wish to participate may still participate in the main clinical study.
    E.3Principal inclusion criteria
    Each subject must meet ALL of the following inclusion criteria to be eligible for enrollment into the study:
    1. At least 18 years of age
    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. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
    4. Primary diagnosis of symptomatic PAH classified by one of the following subgroups:
    a. Idiopathic pulmonary arterial hypertension (IPAH);
    b. Heritable pulmonary arterial hypertension (HPAH);
    c. Drugs or toxins induced based on prior exposure to drugs, chemicals, or toxins, such as fenfluramine derivatives, other anorexigens, toxic rapeseed oil, or L-tryptophan.
    d. PAH associated with: Connective tissue disease (CTD), HIV infection; Congenital systemic-pulmonary shunt (must have undergone surgical correction at least 1 year prior to Screening and have no, or a clinically
    insignificant, shunt fraction [1.0 ≤pulmonary-systemic flow ratio (Qp/Qs) ≤1.5]) in the opinion of the Investigator.
    5. Has had a right heart catheterization (RHC) performed at or within 3 years of Screening (RHC will be performed during Screening if not available) that is consistent with the diagnosis of PAH, meeting all of the following criteria:
    a. Mean pulmonary arterial pressure (mPAP) ≥20 mmHg (at rest)
    b. PAWP ≤15 mmHg (if PAWP cannot be reliably attained, then left ventricular end diastolic pressure [LVEDP] ≤15 mmHg)
    c. PVR >3.00 Wood units (≥240 dynes/sec/cm5).
    6. Has WHO/NYHA functional class II to IV symptoms.
    7. If on PAH-specific background oral therapy, subject is on stable therapy with either an endothelin receptor antagonist (ERA) and/or a PDE5-I or a soluble guanylate cyclase (sGC) stimulator. Subjects may be naïve to PAH-specific treatments; however, subjects must have access to locally available standard of care treatment in accordance with national guidelines
    a. Stable is defined as no change in dose or regimen within 30 days prior to Baseline and for the duration of the study.
    b. Subjects may be on either a PDE5 inhibitor or an sGC at stable dose (but not both).
    c. If the subject’s disease-specific PAH therapy does not include a PDE-5 inhibitor, the use of PDE5-I as needed for erectile dysfunction, up to 3 doses per week, is permitted. The subject should not have taken a dose within 48-hours of any Baseline or study related efficacy assessment.
    8. Has a 6MWD of ≥150 meters.
    9. If the subject is taking concomitant medications that may affect PAH (e.g., calcium channel blockers, digoxin, or L-arginine supplementation), the subject must be on a stable dose for at least 30 days prior to the Baseline Visit and the dosage maintained throughout the study.
    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 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 (i.e., actively attempt to become pregnant or to impregnate, sperm donation, in vitro fertilization) during the study and for 30 days after the last dose of IMP.
    E.4Principal exclusion criteria
    Subjects must not meet ANY of the following exclusion criteria to be eligible for enrollment into the study, unless otherwise indicated:
    1. For subjects with known HIV-associated PAH, a cluster designation 4 (CD4+) T-cell count <200/mm3 within 90 days of Baseline.
    2. Subjects must not have 3 or more of the following left ventricular dysfunction risk factors:
    a. Body mass index (BMI) ≥30 kg/m2
    b. History of systemic hypertension
    c. Diabetes mellitus – any type
    d. Historical evidence of significant coronary artery disease established by any 1 of the following: History of myocardial infarction or percutaneous coronary intervention or angiographic evidence of coronary artery disease (>50% stenosis in at least 1 coronary artery); Positive stress test with imaging; Previous coronary artery bypass graft; Stable angina
    e. Any chronic atrial fibrillation.
    3. Has evidence of more than mild lung disease on PFTs performed within 180 days prior to, or during Screening. Subjects with any of the following criteria will be excluded:
    a. Forced expiratory volume in 1 second (FEV1) <60% (predicted); or
    b. Total lung capacity (TLC) <60% predicted.
    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. Current diagnosis of uncontrolled sleep apnea as defined by the Investigator.
    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 measured at Screening or Baseline in subjects without evidence of intraventricular conduction delay (IVCD). In the presence of IVCD, subjects will be excluded if the QTcF >500 msec for both males and females.
    7. Severe chronic liver disease (i.e., Child-Pugh C), portal hypertension, cirrhosis or complications of cirrhosis/portal hypertension (e.g., history of variceal hemorrhage, encephalopathy).
    8. Confirmed active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV).
    9. Subjects with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3 times the upper limit of normal (ULN) or total bilirubin ≥2 × ULN at Screening.
    10. Chronic renal insufficiency as defined by serum creatinine >2.5 mg/dL or requiring dialysis at Screening.
    11. Hemoglobin concentration <9 g/dL at Screening.
    12. Subjects treated with an IV or SC prostacyclin pathway agent (e.g., epoprostenol, treprostinil, or iloprost) at any time prior to Baseline (use in vasoreactive testing is permitted).
    13. Subjects treated with an inhaled or oral prostacyclin pathway agent (iloprost, treprostinil, beraprost, or selexipag) that was stopped for a safety or tolerability issue.
    If a subject discontinued for other reasons, the subject is eligible if the subject has been off therapy and stable (i.e., no change in WHO/NYHA FC or change in PAHspecific background oral therapy) for 90 days prior to Baseline.
    14. Subject has pulmonary veno-occlusive disease.
    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. 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.
    17. Initiation of a cardio-pulmonary rehabilitation program based upon exercise within 90 days prior to Screening and/or planned during study participation.
    18. Prior participation in any study of ralinepag or participation in another interventional clinical study 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. Any reason that, in the opinion of the Investigator or Medical Monitor, precludes the subject from participating in the study (e.g., 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. Known hypersensitivity to ralinepag or any of the excipients.
    21. Life expectancy <12 months based on the Investigator’s opinion.
    22. Women who are pregnant, lactating, or breast-feeding
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the time (in days) from randomization to the first adjudicated protocol-defined worsening event. Subjects without a protocol-defined clinical worsening event will be censored at date of last contact, 7 days after last study dose, or end of study date, whichever is the earliest.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study
    E.5.2Secondary end point(s)
    • NT-proBNP with log transformation will be analyzed using MMRM analysis with treatment, the stratification factors, week, and treatment-by-week interaction as factors and baseline NT-proBNP as a covariate. Least squares means, standard errors (SE), and 95% CIs for the treatments and their difference will be presented together with the p-value.
    • 6MWD will be analyzed using MMRM analysis with treatment measured >12 hours post dose, the stratification factors (less 6MWD stratum), week, and treatment-by-week interaction as factors and baseline 6MWD as a covariate. Least squares means, SEs, and 95% CIs for the treatments and their difference will be presented together with the p-value.
    • WHO/NYHA functional class will be analyzed using using CMH method adjusted for baseline WHO/NYHA functional class and using modified ridit scores to compute the test statistic and p-value for the between treatment comparison.
    • Time to first all-cause hospitalization will be analyzed using Cox regression with a model that includes treatment and the stratification factors. The hazard ratio for treatment together with its 95% CI and p-value will be presented.
    • Time to all-cause mortality will be analyzed using Cox regression with a model that includes treatment and the stratification factors. The hazard ratio for treatment together with its 95% CI and p-value will be presented.
    • HRR following completion of 6MWT will be analyzed using MMRM analysis with treatment, the stratification factors, week, and treatment-by-week interaction as factors and baseline HRR as a covariate. Least squares means, SEs, and 95% CIs for the treatments and their difference will be presented together with the p-value.
    • The proportion of subjects who achieve all three of the following: NT-proBNP <300 pg/mL, 6MWD >440 meters, WHO/NYHA functional class I or II status or better will be analyzed using CMH method. The odds ratio will be presented together with 95% CIs and the p-value.
    • HRQoL measures (PAH-SYMPACT Questionnaire and SF-36) will be analyzed using MMRM analysis with treatment, the stratification factors, week, and treatment-by-week interaction as factors and baseline domain score as a covariate. Least squares means, SEs, and 95% CIs for the treatments and their difference will be presented together with the p-value.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study; secondary endpoints will be assessed as the change from baseline to Week 28, except for time to all-cause hospitalization and time to all-cause mortality.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Optional evaluation of proteomics, metabolomics, transcriptomics, and genetics
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA67
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Brazil
    Canada
    Chile
    China
    European Union
    Israel
    Korea, Republic of
    Mexico
    Serbia
    Singapore
    Taiwan
    Turkey
    Ukraine
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days15
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 560
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 140
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 235
    F.4.2.2In the whole clinical trial 700
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who experience a primary endpoint event at any time during the study and all subjects on IMP 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 and receive treatment with ralinepag. Subjects who do not choose to participate in the OLE will discontinue IMP and may receive standard-of-care PAH treatment off-study, at the discretion of the treating physician.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-11-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-05
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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