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    Summary
    EudraCT Number:2018-004887-74
    Sponsor's Protocol Code Number:AC-065D301
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-06-03
    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 ConcernedUK - MHRA
    A.2EudraCT number2018-004887-74
    A.3Full title of the trial
    A Multicenter, Randomized, Double-blind, Placebo-controlled Study in
    Participants with Sarcoidosis-associated Pulmonary Hypertension (SAPH)
    to Assess the Efficacy and Safety of Oral Selexipag.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of the effectiveness and safety of selexipag in patients with sarcoidosis-associated pulmonary hypertension.
    A.4.1Sponsor's protocol code numberAC-065D301
    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+31715242166
    B.5.5Fax number+31715242110
    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 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 / JNJ-67896049
    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 / JNJ-67896049
    D.3.9.3Other descriptive nameSELEXIPAG
    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.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
    sarcoidosis-associated pulmonary hypertension (SAPH)
    E.1.1.1Medical condition in easily understood language
    Elevated pressure in lung vessels due to sarcoidosis (i.e. sarcoidosis-associated pulmonary hypertension [SAPH])
    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 assess the effect of selexipag versus placebo on pulmonary vascular resistance (PVR) in participants with sarcoidosis-associated pulmonary hypertension (SAPH) up to Week 26.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Must sign an ICF indicating that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.
    2. Male or female.
    3. 18 (or the legal age of consent in the jurisdiction in which the study is taking place) to 75 years of age, inclusive, at Screening.
    4. Confirmed diagnosis of sarcoidosis as per ATS criteria.
    5. Sarcoidosis-associated precapillary PH, confirmed by RHC (at rest) within 90 days prior to randomization:
    a. PVR ≥320 dyn*sec/cm5 (≥4.0 Wood units)
    b. Mean pulmonary artery pressure (mPAP) ≥25 mmHg
    c. Pulmonary artery wedge pressure (PAWP) ≤15 mm Hg, or if not available or unreliable, a left ventricular end diastolic pressure (LVEDP) ≤15 mmHg.
    6. PH severity according to modified WHO FC II–IV at Screening and randomization; participants of WHO FC IV must be in a stable condition and able to perform a 6MWT.
    7. Criterion modified per Amendment 2.
    7.1 Either not receiving PH-specific treatment, or receiving PH-specific oral monotherapy (ie, riociguat or PDE5i or ERA); if on oral PH-specific monotherapy then treatment had to be stable (i.e. no introduction of new therapies or changes in dose) for at least 90 days prior to both the RHC qualifying for enrollment and randomization.
    8. Criterion modified per Amendment 2.
    8.1 Stable sarcoidosis treatment regimen, ie, no new specific anti-inflammatory treatment for sarcoidosis for at least 90 days, and stable dose(s) for at least 30 days prior to both the RHC qualifying for enrollment and randomization.
    9. 6MWD between 50 and 450m both at Screening and at the time of randomization.
    10. Forced vital capacity (FVC) >50% of predicted at Screening.
    11. FEV1/FVC ≥60%, or if FEV1/FVC <60% then FEV1 must be ≥60% of predicted at Screening.
    12. For patients enrolled or planned to be enrolled in a cardio-pulmonary rehabilitation program based on exercise training, one of the following must apply:
    • In the maintenance phase of the program at the time of randomization with no plans to stop the program until Week 39, or
    • Start of the cardio-pulmonary rehabilitation program based on exercise training is planned after Week 39.
    13. A woman must be
    a. Not of childbearing potential
    b. Of childbearing potential and
    o Have a negative highly sensitive serum (β-human chorionic gonadotropin [β-hCG]) at Screening and a negative urine pregnancy test at randomization.
    o Agree to undertake monthly urine pregnancy tests during the study and up to at least 30 days after study intervention discontinuation.
    o Practicing an acceptable method of contraception and agree to remain on an acceptable method while receiving study intervention and until 30 days after last dose of study intervention. Examples of acceptable methods of contraception are located in Appendix 5.
    14. A woman using oral contraceptives must have been using this method for at least 1 month prior to randomization.
    15. Willing and able to adhere to the lifestyle restrictions specified in Section 5.3.
    E.4Principal exclusion criteria
    1. PH due to left heart disease (PAWP >15 mmHg).
    2. PH due to compression of pulmonary arteries and/or pulmonary veins.
    3. History of left heart failure (LHF) as assessed by the investigator including cardiomyopathies and cardiac sarcoidosis, with a left ventricular ejection fraction (LVEF) <40%.
    4. Severe coronary heart disease (CHD) or unstable angina as assessed by the investigator.
    5. Myocardial infarction within the last 6 months prior to or during Screening.
    6. Criterion modified per Amendment 2.
    6.1 Decompensated cardiac failure not receiving optimal medical treatment according to local guidelines.
    7. Arrhythmias assessed as severe by the investigator.
    8. Criterion modified per Amendment 2.
    8.1 Implantable cardioverter defibrillator (ICD) for secondary prevention (current or planned).
    9. Cerebrovascular events (eg, transient ischemic attack, stroke) within the last 90 days prior to or during Screening.
    10. Congenital or acquired valvular defects with clinically relevant myocardial function disorders not related to PH.
    11. Overt features of pulmonary veno-occlusive disease (PVOD).
    12. Significant emphysema as assessed by the investigator.
    13. Criterion modified per Amendment 2.
    13.1 Known and documented severe hepatic impairment (eg. Child-Pugh Class C). Note: the assessment of hepatic impairment (Child-Pugh Score) must be fully documented for patients who have clinical signs and evidence (from central and/or local lab) of hepatic impairment.
    14. Severe renal failure (estimated glomerular filtration rate [eGFR] <30 mL/min/1.73 m2 or serum creatinine >2.5 mg/dL) based on central laboratory results from the Screening blood sample.
    15. Criterion modified per Amendment 2.
    15.1 Treatment with prostacyclin, prostacyclin analogues or IP receptor agonists (ie, selexipag) within 90 days prior to randomization and/or prior to the RHC qualifying for enrollment, except those given at vasodilator testing during RHC.
    16. Included on a lung transplant list or planned to be included until Visit 6 / Week 39.
    17. Known or suspected uncontrolled thyroid disease as per investigator judgment.
    18. Treatment with moderate inducers of CYP2C8, eg rifampicin or strong inhibitors of CYP2C8, eg, gemfibrozil at or within 14 days prior to randomization.
    19. Change in dose or initiation of new diuretics and/or calcium channel blockers within 1 week prior to RHC qualifying for enrollment.
    20. SBP <90 mmHg at Screening or at randomization.
    21. Known allergies, hypersensitivity, or intolerance to selexipag or its excipients (refer to Investigator’s Brochure).
    22. Planned or current treatment with another investigational treatment up to 90 days prior to randomization.
    23. Received an investigational intervention or used an invasive investigational medical device within 90 days prior to randomization.
    24. Any condition for which, in the opinion of the investigator, participation would not be in the best interests of the participant (eg, compromise well-being), or that could prevent, limit, or confound the protocol-specified assessments.
    25. Any acute or chronic impairment that may influence the ability to comply with study requirements such as to perform RHC, a reliable and reproducible 6MWT (eg, use of walking aids (cane, walker, etc.), or lung function tests.
    26. Employee of the investigator or study site, with direct involvement in the proposed study or other studies under the direction of that investigator or study site, as well as family members of the employees or the investigator.
    27. Pregnant or breast-feeding, or planning to become pregnant while enrolled in this study of within 30 days after the last dose of study intervention.
    E.5 End points
    E.5.1Primary end point(s)
    • PVR on study intervention up to Week 26 expressed as percent of the baseline value.
    E.5.1.1Timepoint(s) of evaluation of this end point
    2 to 5 hours post-dose at week 26
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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 EEA29
    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
    Belgium
    Brazil
    Canada
    Czechia
    France
    Germany
    Italy
    Netherlands
    Spain
    United Kingdom
    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 days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 60
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 14
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 74
    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 participant has completed or discontinued the study, the investigator/delegate will explain what treatment(s) / medical care is necessary and available according to local regulations. If indicated, the sponsor will provide participants who completed the study and did not prematurely discontinue study intervention with selexipag until access to commercial selexipag is possible in this indication in the participant’s country of residence, according to local regulatory requirements.
    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 Decision2019-07-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-14
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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