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    Summary
    EudraCT Number:2018-000145-39
    Sponsor's Protocol Code Number:AC-065A203
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-07-26
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2018-000145-39
    A.3Full title of the trial
    A prospective, multicenter, open-label, single-arm, Phase 2 study to investigate the safety, tolerability and pharmacokinetics of selexipag in children with pulmonary arterial hypertension
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to confirm the doses of selexipag to be used in children with pulmonary arterial hypertension
    A.4.1Sponsor's protocol code numberAC-065A203
    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 SponsorActelion Pharmaceuticals Ltd
    B.1.3.4CountrySwitzerland
    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 supportActelion Pharmaceuticals Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationActelion Pharmaceuticals Ltd
    B.5.2Functional name of contact pointClinical Trial Disclosure Desk
    B.5.3 Address:
    B.5.3.1Street AddressGewerbestrasse 16
    B.5.3.2Town/ cityAllschwil
    B.5.3.3Post code4123
    B.5.3.4CountrySwitzerland
    B.5.6E-mailclinical-trials-disclosure@its.jnj.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 Yes
    D.2.1.1.1Trade name Uptravi
    D.2.1.1.2Name of the Marketing Authorisation holderJanssen-Cilag International NV
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSelexipag
    D.3.2Product code ACT-293987
    D.3.4Pharmaceutical form Film-coated 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 INNSELEXIPAG
    D.3.9.2Current sponsor codeACT-293987
    D.3.9.4EV Substance CodeSUB130805
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSelexipag
    D.3.2Product code ACT-293987
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation Yes
    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 INNSELEXIPAG
    D.3.9.2Current sponsor codeACT-293987
    D.3.9.4EV Substance CodeSUB130805
    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: 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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSelexipag
    D.3.2Product code ACT-293987
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation Yes
    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 INNSELEXIPAG
    D.3.9.2Current sponsor codeACT-293987
    D.3.9.4EV Substance CodeSUB130805
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pulmonary arterial hypertension
    E.1.1.1Medical condition in easily understood language
    Pulmonary arterial hypertension is a condition in which the pressure in the blood vessels going to the lungs (the pulmonary arteries) is higher than normal
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10064911
    E.1.2Term Pulmonary arterial hypertension
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposures as adult doses in children from ≥ 2 to < 18 years of age, with pulmonary arterial hypertension (PAH), by investigating the PK of selexipag and its active metabolite ACT-333679 in this population.
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of selexipag in children from ≥ 2 to < 18 years of age with PAH.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed and dated informed consent by the parent(s) or Legally authorized representative(s) AND assent from developmentally capable children.
    2. Males or females between ≥ 2 and < 18 years of age at Baseline / Enrollment / Visit 2 weighing ≥ 9 kg.
    3. PAH diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before the subject’s enrollment
    4. PAH with one of the following etiologies:
    • idiopathic (iPAH),
    • heritable (hPAH),
    • associated with congenital heart disease (CHD):
    – PAH with co-incidental CHD
    – Post-operative PAH (persisting/ recurring/ developing ≥ 6 months after repair of CHD)
    • Drug or toxin-induced PAH
    • PAH associated with HIV
    • PAH associated with connective tissue disease
    5. Word Health Organizaition functional class (WHO FC) II to III.
    6. Subjects treated with an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor, provided that the treatment dose(s) has been stable for at least 3 months prior to enrollment, or patients who are not candidates for these therapies.
    7. Females of childbearing potential must have a negative pregnancy test at Screening and at Enrollment, and must agree to undertake monthly pregnancy tests, and to use a reliable method of contraception (if sexually active) from screening up to study drug discontinuation + 30 days (EOS).
    E.4Principal exclusion criteria
    Etiology
    1. Subjects with PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease (PVOD) and/or pulmonary capillary hemangiomatosis.
    2. Subjects with PAH associated with Eisenmenger syndrome.
    3. Subjects with moderate to large left-to-right shunts.
    4. Subjects with cyanotic congenital cardiac lesions such as transposition of the great arteries, truncus arteriosus, univentricular heart, or pulmonary atresia with ventricular septal defect, as well as subjects with Fontan palliation.
    5. Subjects with PH due to lung disease and/or hypoxia. For subjects with Down syndrome, exclusion of lung disease and hypoxia causing PH should be documented (e.g., computed tomography scan, polysomnography, lung function tests).

    Treatment and intervention
    6. Previous treatment with Uptravi® (selexipag) within 2 weeks prior to enrollment.
    7. Subjects having received prostacyclin (epoprostenol) or prostacyclin analogs2 (i.e., treprostinil, iloprost, beraprost) within 2 months prior to enrollment or who are scheduled to receive any of these compounds during the trial.
    8. Treatment with another investigational drug within 4 weeks prior to enrollment.
    9. Treatment with strong and moderate inhibitors of CYP2C8 (e.g., gemfibrozil, clopidogrel, deferasirox, teriflunomide) within 2 weeks prior to enrollment until the last dose of selexipag + 3 days.
    10. Treatment with inhibitors of UGT1A3 and UGT2B7 (valproic acid, probenecid, and fluconazole) are prohibited from 2 weeks prior to enrollment and until the last dose of selexipag + 3 days.
    11. Any PAH-related surgical intervention planned, or subjects listed for organ transplantation related to PAH.
    12. History or current suspicion of intussusception or ileus or gastrointestinal obstruction, per investigator’s judgment.

    Baseline abnormalities
    13. Known concomitant life-threatening disease with a life expectancy < 12 months.
    14. Uncontrolled thyroid disease, per investigator’s judgment.
    15. Hemoglobin or hematocrit < 75% of the lower limit of normal range.
    16. Known severe or moderate hepatic impairment, i.e., Child-Pugh Class B or C [see Appendix 4].
    17. Clinical signs of hypotension that, in the investigator’s judgment, would preclude the initiation of a PAH-specific therapy.
    18. Subjects with severe renal insufficiency (estimated creatinine clearance < 30 mL/min or serum creatinine > 221 μmol/L).
    19. Severe coronary heart disease or unstable angina as assessed by the investigator.
    20. Myocardial infarction within the last 6 months prior to enrollment.
    21. Decompensated cardiac failure if not under close supervision.
    22. Severe arrhythmias as assessed by the investigator.
    23. Cerebrovascular events (e.g., transient ischemic attack, stroke) within the last 3 months prior to enrollment.
    24. Congenital or acquired valvular defects with clinically relevant myocardial function disorders not related to PH.

    Pregnancy and breastfeeding
    25. Pregnancy (including family planning) or breastfeeding.

    Other categories
    26. Known hypersensitivity to the investigational treatment or to any of the excipients of the drug formulations.
    27. Drug or substance abuse, or any condition that, in the opinion of the investigator, may prevent compliance with the protocol or adherence to study treatment.
    28. Loss of 250 mL or more of blood within 3 months prior to screening.
    29. History or clinical evidence of any disease and/or existence of any surgical or medical condition that might interfere with the absorption, distribution, metabolism, or excretion of the study treatment(s) (e.g., cholecystectomy).
    30. Inability to swallow tablets whole.
    E.5 End points
    E.5.1Primary end point(s)
    Primary pharmacokinetic (PK) endpoint:
    - Model-based exposure (AUCτ,ss, combined) of selexipag and ACT-333679 corrected for their potency, determined during the 12 weeks up-titration period.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - PK sampling at Weeks 1, 2, 4, 6 and 12
    E.5.2Secondary end point(s)
    Secondary PK endpoints:
    - Area under the plasma concentration-time curve over one dosing interval at steady state (AUCτ,ss), maximum plasma concentration at steady state (Cmax,ss), and the time at which Cmax,ss is observed (tmax,ss) for selexipag and ACT-333679, based on non-compartmental analysis (NCA).
    -Ttrough concentration at steady state (Ctrough, ss) for selexipag and ACT-333679.

    Safety endpoints
    • Treatment-emergent AEs (TEAEs), treatment-emergent serious adverse events (TESAEs) and treatment-emergent deaths (all causes), AEs leading to permanent discontinuation of study drug.
    • Treatment-emergent marked laboratory abnormalities and ECG abnormalities up to EOT+ 3 days.
    • Change from baseline up to EOT + 3 days in:
    - selected hematology and blood chemistry laboratory variables.
    - TSH
    - Vital signs (blood pressure, heart rate)
    - Height and body mass index
    - Sexual maturation (Tanner stage)
    E.5.2.1Timepoint(s) of evaluation of this end point
    - AUCτ,ss, cmax,ss, tmax,ss: PK sampling at Weeks 1, 2, 4, 6 and 12
    - Ctrough: PK sampling at Weeks 2, 4,and 6
    - TEAEs, TESAEs,treatment emergent deaths and AEs leading to permanent discontinuation: continuously from Day 1 up to 3 days after the end of treatment
    - Tanner stage: at screening, Week 16, Month 22 and every 6 months afterwards
    - Other safety endpoints: on Day 1 (baseline), Week 12 and Week 16, then every 3 months during the first 12 months and every 6 months thereafter up to the end of treatment visit, with additional timepoints for vital signs (Week 4), ECG (Weeks 1 & 4), laboratory tests (Week 4), TSH (Weeks 4 & 8)
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Belarus
    Canada
    China
    France
    Germany
    Hungary
    Israel
    Italy
    Malaysia
    Netherlands
    Poland
    Romania
    Russian Federation
    Serbia
    Singapore
    Taiwan
    Ukraine
    United Kingdom
    United States
    Vietnam
    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.8.9.2In all countries concerned by the trial years7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 55
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 35
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under age incapable of giving consent personally. However assent from developmentally capable children will be required.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 55
    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)
    After the child has ended his/her participation in the study, the investigator/delegate will explain what treatment(s) / medical care is necessary and available according to local regulations
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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