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    Summary
    EudraCT Number:2021-002297-11
    Sponsor's Protocol Code Number:NOPRODPAPUH3001
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-12-16
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2021-002297-11
    A.3Full title of the trial
    A Prospective, Open-label, Platform Study for Long-term Follow-up of Participants Using Study Intervention in Pulmonary Hypertension Parent Studies
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Providing long-term treatment access in patients with pulmonary hypertension completing a parent study and having no other treatment access alternative
    A.3.2Name or abbreviated title of the trial where available
    PLATYPUS
    A.4.1Sponsor's protocol code numberNOPRODPAPUH3001
    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 organisationJanssen-Cilag International NV
    B.5.2Functional name of contact pointClinical Registry Group
    B.5.3 Address:
    B.5.3.1Street AddressArchimedesweg 29
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 CM
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+3171524 2166
    B.5.5Fax number+3171524 2110
    B.5.6E-mailClinicalTrialsEU@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 Opsumit
    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 nameMacitentan
    D.3.2Product code JNJ-67896062
    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 INNMacitentan
    D.3.9.1CAS number 441798-33-0
    D.3.9.2Current sponsor codeJNJ-67896062
    D.3.9.4EV Substance CodeSUB89247
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 nameFixed dose combination of macitentan and tadalafil (JNJ-68150420)
    D.3.2Product code CJNJ-68150420-ZZZ-G001 (ACT064992D)
    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 INNMacitentan
    D.3.9.1CAS number 441798-33-0
    D.3.9.2Current sponsor codeJNJ-67896062
    D.3.9.4EV Substance CodeSUB89247
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTadalafil
    D.3.9.1CAS number 171596-29-5
    D.3.9.2Current sponsor codeJNJ-10291697
    D.3.9.4EV Substance CodeSUB12602MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Hypertension
    E.1.1.1Medical condition in easily understood language
    Pulmonary hypertension (PH) is a disease associated with abnormally high pressure in the blood vessels of the lungs
    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 10037400
    E.1.2Term Pulmonary hypertension
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the long-term safety of the respective study interventions in treated participants
    E.2.2Secondary objectives of the trial
    Secondary objectives of the trial are not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participant must sign an informed consent form (ICF) (or their legally acceptable representative must sign) indicating that participant understands the purpose of, and procedures required for, the study and is willing to participate in the study.
    2.Participant treated with macitentan 10 mg or FDC of macitentan 10 mg and tadalafil 40 mg at the end of a sponsor parent study and:
    a. The indication of the parent study is included in the protocol
    b. Participant has completed the parent study
    c. No alternative means of access to study intervention have been
    identified
    d. Participant may continue to benefit from treatment with the study
    intervention
    3. A woman of childbearing potential must:
    a. have a negative urine or serum pregnancy test prior to first intake of study intervention,
    b. agree to perform monthly urine pregnancy test up to the end of the safety follow-up period,
    c. Agree to follow contraceptive methods as defined in the protocol until 30 days after the last intake of the study intervention
    E.4Principal exclusion criteria
    General exclusion criteria
    1. Participants prematurely discontinued from their parent study.
    2. Female participant being pregnant, or breastfeeding, or planning to become pregnant while enrolled in this study.
    3. Planned or current treatment with another investigational treatment.
    Macitentan exclusion criteria
    1. Known allergies, hypersensitivity, or intolerance to macitentan or its excipients (refer to the macitentan IB).
    2. Hemoglobin <80 g/L. Participants with hemoglobin <80 g/L at Screening are allowed to enter the study at the
    discretion of the investigator provided they meet all inclusion criteria. Study treatment
    is to be initiated as soon as their hemoglobin is ≥80 g/L, assuming it is within the
    allowed treatment interruption period.
    3. Serum aspartate (AST) and/or alanine aminotransferases (ALT) >3 ×
    the upper limit of normal (ULN) range. Participants with AST and/ or ALT >3 × ULN at Screening are allowed to enter the
    study at the discretion of the investigator, provided they meet all inclusion criteria as
    long as the liver chemistry abnormality does not meet one of the permanent
    discontinuation criteria listed for this ISA. Study treatment is to be initiated as soon as
    their serum AST and/or ALT is ≤3 × ULN, assuming it is within the allowed treatment
    interruption period.
    4. Known and documented severe hepatic impairment ie, Child-Pugh
    Class C. For participants with hepatic impairment, Child-Pugh Class
    should be fully assessed and documented in the source documents at
    Screening.
    5. Treatment with a strong cytochrome P450 (CYP)3A4 inhibitor (eg,
    ketoconazole, itraconazole, voriconazole, clarithromycin, telithromycin, nefazodone, ritonavir, and saquinavir).
    6. Treatment with a moderate dual CYP3A4/CYP2C9 inhibitor (eg,
    fluconazole, amiodarone) or uses a co-administration of a combination of moderate CYP3A4 (eg ciprofloxacin, cyclosporine, diltiazem, erythromycin, verapamil) and moderate CYP2C9 inhibitors (eg, miconazole, piperine). If a participant coming from a parent
    study is currently under such a concomitant treatment, the participant may be enrolled as per the investigator's discretion based on his/her clinical judgement and risk-benefit assessment.
    7. Treatment with a strong CYP3A4 inducer (eg, rifabutin, rifampin,
    rifampicin, rifapentin, carbamazepine, phenobarbital, phenytoin, St.
    John's Wort) within 1 month prior to baseline.
    8. Interruption of study intervention for more than 4 weeks since the
    last dose of study intervention taken in the parent study.
    Macitentan and Tadalafil exclusion criteria:
    1. Known allergies, hypersensitivity, or intolerance to macitentan or
    tadalafil or their excipients (refer to the macitentan/tadalafil FDC IB).
    2. Hemoglobin <80 g/L. Participants with hemoglobin <80 g/L at Enrollment are allowed to enter the study at
    the discretion of the investigator provided they meet all inclusion criteria. Study
    treatment is to be initiated as soon as their hemoglobin is ≥80 g/L, assuming it is within
    the allowed treatment interruption period.
    3. Serum aspartate (AST) and/or alanine aminotransferases (ALT) >3 ×
    the upper limit of normal (ULN) range. Participants with serum AST and/ or ALT >3 × ULN at Enrollment are allowed to enter
    the study at the discretion of the investigator, provided they meet all inclusion criteria,
    as long as the liver chemistry abnormality does not meet one of the permanent
    discontinuation criteria listed for this ISA. Study treatment is to be initiated as soon as
    their serum AST and/or ALT is ≤3 × ULN, assuming it is within the allowed treatment
    interruption period.
    4. Known and documented severe hepatic impairment ie, Child-Pugh
    Class C.
    5. Severe renal impairment (estimated glomerular filtration rate
    (eGFR)/creatinine clearance <30 mL/min).
    6. Loss of vision in one or both eyes because of non-arteritic anterior
    ischemic optic neuropathy, regardless of whether or not this episode
    was in connection with phosphodiesterase type 5 inhibitor treatment.
    7. Treatment with a strong CYP3A4 inhibitor (eg, ketoconazole, itraconazole,
    voriconazole, clarithromycin, telithromycin, nefazodone, ritonavir, and saquinavir).
    8. Treatment with a moderate dual CYP3A4/CYP2C9 inhibitor or uses a co-administration of a combination of moderate CYP3A4 and moderate CYP2C9 inhibitors.
    9. Treatment with a strong CYP3A4 inducer within 1 month prior to baseline.
    10. Treatment with doxazosin.
    11. Treatment with any form of organic nitrate, either regularly or
    intermittently.
    12. Treatment with an ERA, phosphodiesterase type 5 inhibitor, or
    soluble guanylate cyclase stimulator.
    13. Interruption of study intervention for more than 4 weeks since the last dose of study intervention taken in the parent study
    E.5 End points
    E.5.1Primary end point(s)
    Frequency of treatment-emergent adverse events (AEs), treatment-emergent AEs leading to discontinuation, serious adverse events
    (SAEs), and/or deaths
    E.5.1.1Timepoint(s) of evaluation of this end point
    from baseline until End-of-Study (EOS)
    E.5.2Secondary end point(s)
    Secondary endpoints not applicable.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoints not applicable.
    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 No
    E.6.6Pharmacokinetic No
    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 No
    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 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 Yes
    E.8.1.7.1Other trial design description
    Platform study
    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.8.3 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA37
    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
    South Africa
    Turkey
    Belarus
    Belgium
    Bulgaria
    China
    India
    Korea, Republic of
    Poland
    Romania
    Russian Federation
    Taiwan
    Ukraine
    France
    E.8.7Trial has a data monitoring committee No
    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
    The End of Study is considered as the last scheduled study End of Trial or safety follow-up (EOS) visit, whichever comes last for the last participant in the study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days17
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days0
    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: 299
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 91
    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 state28
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 123
    F.4.2.2In the whole clinical trial 390
    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)
    None
    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 Decision2022-03-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-07
    P. End of Trial
    P.End of Trial StatusOngoing
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