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    Clinical Trial Results:
    A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Group-sequential, Adaptive, Phase 3 Study With Open-label Extension Period to Assess the Efficacy and Safety of Selexipag as an add-on to Standard of Care Therapy in Subjects With Inoperable or Persistent/Recurrent After Surgical and/or Interventional Treatment Chronic Thromboembolic Pulmonary Hypertension

    Summary
    EudraCT number
    		 2018-002823-41
    Trial protocol
    		 DE   GB   CZ   BE   NL   HU   AT   BG   DK   PT   IT  
    Global end of trial date
    07 Jun 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Jun 2023
    First version publication date
    16 Jun 2023
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    AC-065B302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03689244
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Actelion Pharmaceuticals Ltd
    Sponsor organisation address
    Gewerbestrasse 16, Allschwil, Switzerland, 4123
    Public contact
    Clinical Registry Group, Actelion Pharmaceuticals Ltd, ClinicalTrialsEU@its.jnj.com
    Scientific contact
    Clinical Registry Group, Actelion Pharmaceuticals Ltd, ClinicalTrialsEU@its.jnj.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    07 Jun 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 Jun 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main objective of the study was to evaluate the effect of selexipag on pulmonary vascular resistance (PVR) versus placebo in subjects with inoperable chronic thromboembolic pulmonary hypertension (CTEPH) (that is, technically non-operable) and persistent/recurrent CTEPH after surgical pulmonary endarterectomy (PEA) and/or interventional balloon pulmonary angioplasty [BPA]) treatment at Week 20.
    Protection of trial subjects
    This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    23 Jan 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Argentina: 2
    Country: Number of subjects enrolled
    Australia: 4
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Bulgaria: 4
    Country: Number of subjects enrolled
    Brazil: 11
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    China: 2
    Country: Number of subjects enrolled
    Czechia: 3
    Country: Number of subjects enrolled
    Germany: 15
    Country: Number of subjects enrolled
    Denmark: 7
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    United Kingdom: 11
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Korea, Republic of: 9
    Country: Number of subjects enrolled
    Mexico: 5
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    Portugal: 6
    Country: Number of subjects enrolled
    Russian Federation: 7
    Country: Number of subjects enrolled
    Slovakia: 1
    Country: Number of subjects enrolled
    Sweden: 1
    Country: Number of subjects enrolled
    Thailand: 6
    Country: Number of subjects enrolled
    Turkey: 8
    Country: Number of subjects enrolled
    Taiwan: 2
    Country: Number of subjects enrolled
    Ukraine: 1
    Country: Number of subjects enrolled
    United States: 8
    Worldwide total number of subjects
    128
    EEA total number of subjects
    49
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    60
    From 65 to 84 years
    67
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 321 subjects were screened, of which 128 were randomly assigned and received study intervention.

    Period 1
    Period 1 title
    Double Blind Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Double-blind Period: Placebo
    Arm description
    		 Subjects received a single oral tablet of placebo matching to selexipag 200 micrograms (mcg) in the evening of Day 1 and continued the same dose (200 mcg) twice daily (BID) on Day 2. Upon first dose toleration, placebo dose was up titrated with weekly increments of 200 mcg until reaching the individual maximum tolerated dose (iMTD) in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to end of DB treatment period (EDBT), further up-titration was done but not above 1600 mcg, BID (maximum duration: 24.6 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was to be once daily (QD).
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were administered with single dose of placebo matching to selexipag 200 mcg tablet orally in the evening of day 1 and continued the same dose BID on Day 2. Upon first dose toleration, placebo dose was up titrated with weekly increments of 200 mcg placebo until reaching the individual iMTD in the range of 200 to 1600 mcg placebo BID or QD.

    Arm title
    		 Double-blind Period: Selexipag
    Arm description
    		 Subjects received a single oral tablet of selexipag 200 mcg in the evening of Day 1 and continued the same dose (200 mcg) BID on Day 2. Upon first dose toleration, selexipag dose was up titrated with weekly increments of 200 mcg until reaching iMTD in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 27.7 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was to be QD.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Selexipag
    Investigational medicinal product code
    Other name
    JNJ-67896049
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects were administered with single dose of selexipag 200 mcg tablet orally in the evening of day 1 and continued the same dose BID on Day 2. Upon first dose toleration, selexipag dose was up titrated with weekly increments of 200 mcg until reaching the individual iMTD in the range of 200 to 1600 mcg BID or QD.

    Number of subjects in period 1
    		Double-blind Period: Placebo Double-blind Period: Selexipag
    Started
    64
    64
    Completed
    9
    6
    Not completed
    55
    58
         Consent withdrawn by subject
    7
    8
         Physician decision
    2
    2
         Death
    1
    -
         Unspecified
    1
    1
         Sponsor decision
    44
    47
    Period 2
    Period 2 title
    Open Label Period
    Is this the baseline period?
    		 No
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Open Label Period: Selexipag (Ex-Placebo)
    Arm description
    		 All subjects who completed DB treatment period, entered OL extension period, and received the first dose (selexipag 200 mcg) of OL period in the evening of the EDBT and then 200 mcg BID on the day after. Subjects with moderate hepatic impairment (Child-Pugh B) or who had taken concomitantly moderate CYP2C8 inhibitor(s) started one tablet of OL selexipag 200 mcg in the morning of the day after EDBT visit. Dose up-titration was done as like DB treatment till iMTD was reached. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 20.8 months).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Selexipag
    Investigational medicinal product code
    Other name
    JNJ-67896049
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received single tablet of selexipag 200 mcg orally BID. For subjects with Child-Pugh B or who had taken concomitantly a moderate CYP2C8 inhibitor, the dosing frequency was QD.

    Arm title
    		 Open Label Period: Selexipag (Ex-Selexipag)
    Arm description
    		 All subjects who completed DB treatment period, entered OL extension period, and received the first dose (selexipag 200 mcg) of OL period in the evening of the EDBT and then 200 mcg BID on the day after. Subjects with moderate hepatic impairment (Child-Pugh B) or who had taken concomitantly moderate CYP2C8 inhibitor(s) started one tablet of OL selexipag 200 mcg in the morning of the day after EDBT visit. Dose up-titration was done as like DB treatment till iMTD was reached. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 19.8 months).
    Arm type
    		 Experimental

    Investigational medicinal product name
    Selexipag
    Investigational medicinal product code
    Other name
    JNJ-67896049
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received single tablet of selexipag 200 mcg orally BID. For subjects with Child-Pugh B or who had taken concomitantly a moderate CYP2C8 inhibitor, the dosing frequency was QD.

    Number of subjects in period 2
    		Open Label Period: Selexipag (Ex-Placebo) Open Label Period: Selexipag (Ex-Selexipag)
    Started
    9
    6
    Completed
    0
    0
    Not completed
    9
    6
         Consent withdrawn by subject
    1
    -
         Physician decision
    1
    -
         Sponsor decision
    7
    6

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Double-blind Period: Placebo
    Reporting group description
    		 Subjects received a single oral tablet of placebo matching to selexipag 200 micrograms (mcg) in the evening of Day 1 and continued the same dose (200 mcg) twice daily (BID) on Day 2. Upon first dose toleration, placebo dose was up titrated with weekly increments of 200 mcg until reaching the individual maximum tolerated dose (iMTD) in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to end of DB treatment period (EDBT), further up-titration was done but not above 1600 mcg, BID (maximum duration: 24.6 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was to be once daily (QD).

    Reporting group title
    Double-blind Period: Selexipag
    Reporting group description
    		 Subjects received a single oral tablet of selexipag 200 mcg in the evening of Day 1 and continued the same dose (200 mcg) BID on Day 2. Upon first dose toleration, selexipag dose was up titrated with weekly increments of 200 mcg until reaching iMTD in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 27.7 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was to be QD.

    Reporting group values
    		 Double-blind Period: Placebo Double-blind Period: Selexipag Total
    Number of subjects
    		 64 64 128
    Title for AgeCategorical
    Units: subjects
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 24 36 60
        From 65 to 84 years
    		 39 28 67
        85 years and over
    		 1 0 1
    Title for AgeContinuous
    Units: years
        arithmetic mean (standard deviation)
    		 64 ( 13.22 ) 62 ( 14.38 ) -
    Title for Gender
    Units: subjects
        Female
    		 47 46 93
        Male
    		 17 18 35

    End points

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    End points reporting groups
    Reporting group title
    Double-blind Period: Placebo
    Reporting group description
    		 Subjects received a single oral tablet of placebo matching to selexipag 200 micrograms (mcg) in the evening of Day 1 and continued the same dose (200 mcg) twice daily (BID) on Day 2. Upon first dose toleration, placebo dose was up titrated with weekly increments of 200 mcg until reaching the individual maximum tolerated dose (iMTD) in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to end of DB treatment period (EDBT), further up-titration was done but not above 1600 mcg, BID (maximum duration: 24.6 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was to be once daily (QD).

    Reporting group title
    Double-blind Period: Selexipag
    Reporting group description
    		 Subjects received a single oral tablet of selexipag 200 mcg in the evening of Day 1 and continued the same dose (200 mcg) BID on Day 2. Upon first dose toleration, selexipag dose was up titrated with weekly increments of 200 mcg until reaching iMTD in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 27.7 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was to be QD.
    Reporting group title
    Open Label Period: Selexipag (Ex-Placebo)
    Reporting group description
    		 All subjects who completed DB treatment period, entered OL extension period, and received the first dose (selexipag 200 mcg) of OL period in the evening of the EDBT and then 200 mcg BID on the day after. Subjects with moderate hepatic impairment (Child-Pugh B) or who had taken concomitantly moderate CYP2C8 inhibitor(s) started one tablet of OL selexipag 200 mcg in the morning of the day after EDBT visit. Dose up-titration was done as like DB treatment till iMTD was reached. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 20.8 months).

    Reporting group title
    Open Label Period: Selexipag (Ex-Selexipag)
    Reporting group description
    		 All subjects who completed DB treatment period, entered OL extension period, and received the first dose (selexipag 200 mcg) of OL period in the evening of the EDBT and then 200 mcg BID on the day after. Subjects with moderate hepatic impairment (Child-Pugh B) or who had taken concomitantly moderate CYP2C8 inhibitor(s) started one tablet of OL selexipag 200 mcg in the morning of the day after EDBT visit. Dose up-titration was done as like DB treatment till iMTD was reached. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 19.8 months).

    Primary: Change From Baseline in Pulmonary Vascular Resistance (PVR) at Week 20

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    End point title
    		 Change From Baseline in Pulmonary Vascular Resistance (PVR) at Week 20
    End point description
    Change from baseline in PVR at Week 20 was reported. PVR was assessed by right or right and left heart catheterization. Change from Baseline was measured as ratio of post-dose visit value to baseline value. Hemodynamic set (HES) included all assigned subjects in the hemodynamic cohort. Here, "Measure type (Least Squares Mean)" indicated "Geometric LS mean".
    End point type
    Primary
    End point timeframe
    Baseline (Day 1, pre-dose), within 2 to 5 hours post-dose on Week 20
    End point values
    		 Double-blind Period: Placebo Double-blind Period: Selexipag
    Number of subjects analysed
    44
    47
    Units: Ratio
        least squares mean (confidence interval 95%)
    89.52 (81.78 to 97.99)
    85.15 (77.97 to 92.99)
    Statistical analysis title
    		 Selexipag Versus Placebo
    Statistical analysis description
    Ratio of Geometric mean of Selexipag to Placebo was reported.
    Comparison groups
    Double-blind Period: Placebo v Double-blind Period: Selexipag
    Number of subjects included in analysis
    91
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.412
    Method
    		 ANCOVA
    Parameter type
    		 Ratio of geometric LS mean
    Point estimate
    0.95
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.84
         upper limit
    		 1.07

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    For DB period: from first DB dose up to EDBT (that is, up to 27.7 months) and for OL extension period: from first OL dose up to 30 days after the last OL dose (that is, up to 21.8 months)
    Adverse event reporting additional description
    Safety Analysis Set (SAF) included all subjects who received at least one dose of study treatment.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.0
    Reporting groups
    Reporting group title
    Double-blind Period: Placebo
    Reporting group description
    		 Subjects received a single oral tablet of placebo matching to selexipag 200 micrograms (mcg) in the evening of Day 1 and continued the same dose (200 mcg) BID on Day 2. Upon first dose toleration, placebo dose was up titrated with weekly increments of 200 mcg until reaching the iMTD in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to end of DB treatment period (EDBT), further up-titration was done but not above 1600 mcg, BID (maximum duration: 24.6 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was QD.

    Reporting group title
    Open Label Period: Selexipag (Ex-Selexipag)
    Reporting group description
    		 All subjects who completed DB treatment period, entered OL extension period, and received the first dose (selexipag 200 mcg) of OL period in the evening of the EDBT and then 200 mcg BID on the day after. Subjects with moderate hepatic impairment (Child-Pugh B) or who had taken concomitantly moderate CYP2C8 inhibitor(s) started one tablet of OL selexipag 200 mcg in the morning of the day after EDBT visit. Dose up-titration was done as like DB treatment till iMTD was reached. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 19.8 months).

    Reporting group title
    Open Label Period: Selexipag (Ex-Placebo)
    Reporting group description
    		 All subjects who completed DB treatment period, entered OL extension period, and received the first dose (selexipag 200 mcg) of OL period in the evening of the EDBT and then 200 mcg BID on the day after. Subjects with moderate hepatic impairment (Child-Pugh B) or who had taken concomitantly moderate CYP2C8 inhibitor(s) started one tablet of OL selexipag 200 mcg in the morning of the day after EDBT visit. Dose up-titration was done as like DB treatment till iMTD was reached. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 20.8 months).

    Reporting group title
    Double-blind Period: Selexipag
    Reporting group description
    		 Subjects received a single oral tablet of selexipag 200 mcg in the evening of Day 1 and continued the same dose (200 mcg) BID on Day 2. Upon first dose toleration, selexipag dose was up titrated with weekly increments of 200 mcg until reaching iMTD in the range of 200 to 1600 mcg BID. The up titration up to Week 12 was followed by a stable maintenance treatment period with the iMTD (reached at Week 12) until Week 26. After Week 26 up to EDBT, further up-titration was done but not above 1600 mcg, BID (maximum duration: 27.7 months). For subjects with moderate hepatic impairment (Child-Pugh B) or who were concomitantly taking a moderate CYP2C8 inhibitor, the dosing frequency was QD.

    Serious adverse events
    		 Double-blind Period: Placebo Open Label Period: Selexipag (Ex-Selexipag) Open Label Period: Selexipag (Ex-Placebo) Double-blind Period: Selexipag
    Total subjects affected by serious adverse events
         subjects affected / exposed
    16 / 64 (25.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    9 / 64 (14.06%)
         number of deaths (all causes)
    2
    0
    0
    3
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Uterine Leiomyoma
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Urethral Cancer
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Prostate Cancer
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Foot Fracture
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Lower Limb Fracture
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Subdural Haemorrhage
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Supraventricular Tachycardia
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Right Ventricular Failure
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    5 / 64 (7.81%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
    0 / 0
    0 / 8
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Atrioventricular Block Second Degree
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Atrial Flutter
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Iron Deficiency Anaemia
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Multiple Organ Dysfunction Syndrome
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Catheter Site Haematoma
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Death
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Discoloured Vomit
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hepatitis Acute
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute Respiratory Failure
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    Pulmonary Embolism
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Haemoptysis
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Dyspnoea
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Asthma
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pulmonary Hypertension
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory Failure
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute Kidney Injury
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Covid-19 Pneumonia
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Pneumonia Bacterial
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Septic Shock
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Tonsillitis
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Viral Infection
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypervolaemia
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Double-blind Period: Placebo Open Label Period: Selexipag (Ex-Selexipag) Open Label Period: Selexipag (Ex-Placebo) Double-blind Period: Selexipag
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    45 / 64 (70.31%)
    5 / 6 (83.33%)
    9 / 9 (100.00%)
    60 / 64 (93.75%)
    Investigations
    Blood Glucose Increased
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vascular disorders
    Hypotension
         subjects affected / exposed
    4 / 64 (6.25%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    2 / 64 (3.13%)
         occurrences all number
    7
    0
    0
    6
    Hot Flush
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    2 / 64 (3.13%)
         occurrences all number
    0
    1
    0
    2
    Flushing
         subjects affected / exposed
    2 / 64 (3.13%)
    1 / 6 (16.67%)
    1 / 9 (11.11%)
    4 / 64 (6.25%)
         occurrences all number
    2
    1
    1
    5
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 64 (1.56%)
         occurrences all number
    1
    0
    1
    1
    Palpitations
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    6 / 64 (9.38%)
         occurrences all number
    1
    0
    0
    10
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    7 / 64 (10.94%)
    0 / 6 (0.00%)
    2 / 9 (22.22%)
    8 / 64 (12.50%)
         occurrences all number
    8
    0
    2
    9
    Headache
         subjects affected / exposed
    14 / 64 (21.88%)
    2 / 6 (33.33%)
    6 / 9 (66.67%)
    36 / 64 (56.25%)
         occurrences all number
    24
    2
    11
    65
    Hypoaesthesia
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences all number
    1
    1
    0
    1
    Blood and lymphatic system disorders
    Iron Deficiency Anaemia
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    3 / 64 (4.69%)
         occurrences all number
    0
    1
    0
    3
    Anaemia
         subjects affected / exposed
    4 / 64 (6.25%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 64 (1.56%)
         occurrences all number
    4
    0
    1
    1
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    3 / 64 (4.69%)
         occurrences all number
    0
    0
    1
    4
    Chest Pain
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    2 / 64 (3.13%)
         occurrences all number
    3
    0
    1
    2
    Fatigue
         subjects affected / exposed
    4 / 64 (6.25%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    6 / 64 (9.38%)
         occurrences all number
    4
    0
    0
    11
    Oedema
         subjects affected / exposed
    3 / 64 (4.69%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    0 / 64 (0.00%)
         occurrences all number
    3
    0
    1
    0
    Oedema Peripheral
         subjects affected / exposed
    8 / 64 (12.50%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    5 / 64 (7.81%)
         occurrences all number
    11
    0
    0
    5
    Pain
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    5 / 64 (7.81%)
         occurrences all number
    0
    1
    0
    6
    Gastrointestinal disorders
    Irritable Bowel Syndrome
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    0 / 64 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    4 / 64 (6.25%)
    1 / 6 (16.67%)
    3 / 9 (33.33%)
    17 / 64 (26.56%)
         occurrences all number
    5
    1
    5
    29
    Vomiting
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 6 (16.67%)
    3 / 9 (33.33%)
    11 / 64 (17.19%)
         occurrences all number
    1
    1
    4
    14
    Diarrhoea
         subjects affected / exposed
    9 / 64 (14.06%)
    1 / 6 (16.67%)
    5 / 9 (55.56%)
    38 / 64 (59.38%)
         occurrences all number
    11
    2
    8
    57
    Abdominal Pain Upper
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    5 / 64 (7.81%)
         occurrences all number
    1
    0
    0
    5
    Abdominal Distension
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    4 / 64 (6.25%)
         occurrences all number
    0
    0
    0
    4
    Abdominal Discomfort
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 64 (1.56%)
         occurrences all number
    1
    0
    1
    3
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    9 / 64 (14.06%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    3 / 64 (4.69%)
         occurrences all number
    10
    0
    0
    3
    Pulmonary Hypertension
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    2 / 64 (3.13%)
         occurrences all number
    1
    1
    0
    3
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 64 (6.25%)
    1 / 6 (16.67%)
    3 / 9 (33.33%)
    14 / 64 (21.88%)
         occurrences all number
    6
    1
    4
    16
    Back Pain
         subjects affected / exposed
    5 / 64 (7.81%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    5 / 64 (7.81%)
         occurrences all number
    6
    0
    1
    5
    Flank Pain
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences all number
    1
    1
    0
    0
    Muscle Spasms
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    2 / 64 (3.13%)
         occurrences all number
    0
    1
    0
    3
    Myalgia
         subjects affected / exposed
    5 / 64 (7.81%)
    0 / 6 (0.00%)
    4 / 9 (44.44%)
    7 / 64 (10.94%)
         occurrences all number
    5
    0
    6
    7
    Pain in Extremity
         subjects affected / exposed
    4 / 64 (6.25%)
    1 / 6 (16.67%)
    1 / 9 (11.11%)
    13 / 64 (20.31%)
         occurrences all number
    4
    1
    1
    16
    Pain in Jaw
         subjects affected / exposed
    2 / 64 (3.13%)
    0 / 6 (0.00%)
    2 / 9 (22.22%)
    16 / 64 (25.00%)
         occurrences all number
    2
    0
    2
    20
    Infections and infestations
    Covid-19
         subjects affected / exposed
    4 / 64 (6.25%)
    0 / 6 (0.00%)
    2 / 9 (22.22%)
    5 / 64 (7.81%)
         occurrences all number
    4
    0
    2
    5
    Gastroenteritis
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 64 (1.56%)
         occurrences all number
    1
    0
    1
    1
    Pneumonia
         subjects affected / exposed
    1 / 64 (1.56%)
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    0 / 64 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Metabolism and nutrition disorders
    Hyperlipidaemia
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    1 / 9 (11.11%)
    0 / 64 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Decreased Appetite
         subjects affected / exposed
    0 / 64 (0.00%)
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    6 / 64 (9.38%)
         occurrences all number
    0
    0
    0
    6
    Steroid Diabetes
         subjects affected / exposed
    0 / 64 (0.00%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 64 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vitamin D Deficiency
         subjects affected / exposed
    1 / 64 (1.56%)
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    1 / 64 (1.56%)
         occurrences all number
    1
    1
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Jan 2019
    • Several inclusion criteria were revised with the aim of ensuring the recruitment of subjects who could potentially benefit from medical therapy (only technically non operable participants assessed based on imaging review for all CTEPH subpopulations) and who had substantially impaired pulmonary vascular function (PVR greater than or equal to [>=]400 dynes.second/centimeter^5 or >=5 Wood units). • Statistical changes were made following interactions with health authorities.
    16 Apr 2020
    • The study design was revised to power the study for the additional clinical endpoint, time to clinical worsening (TTCW). As a result, elements of the study design, key secondary endpoints, sample size, testing hierarchy and analysis timepoints were amended to ensure the evaluation of 6-minute walk distance (6MWD) and TTCW endpoints. • The DB period was changed from a fixed duration of 52 weeks to a variable duration (maximum 52 months) based on the timepoint at which the overall target number of TTCW events would be reached (or earlier following the recommendation of the independent data monitoring committee (IDMC) or sponsor’s decision). • The stratification factor of CTEPH population was redefined to inoperable (with or without BPA) versus persistent/recurrent after PEA (including PEA followed by BPA). • For the non-hemodynamic cohort, the PVR threshold for inclusion was changed to >=300 dyn.sec/cm^5 or >=3.75 Wood units. • The sponsor’s standardised guidelines for the assessments heart catheterisation (HC), 6MWT and Borg dyspnea index (BDI) were added to the protocol and the Borg CR10 Scale®was added. • A clinical event committee (CEC) was set up in order to ensure independent adjudication of clinical worsening events.
    29 Sep 2020
    • Statistical changes were made in line with the resolution of food and drug administration (FDA) queries. • The maximum duration of the DB period was increased from 52 months to 59 months. • Safety reporting requirements were updated in line with Janssen processes.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Failure to meet success on the primary endpoint led to termination of the study for futility and thus the pre-planned testing hierarchy was no longer applicable, and all efficacy analyses other than the primary endpoint (PVR) were exploratory.
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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