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    Clinical Trial Results:
    A Multicenter, Single-arm, Open-label, Long-term Follow-up Safety Study of Selexipag in Participants who Participated in a Previous Selexipag Study

    Summary
    EudraCT number
    		 2020-000475-21
    Trial protocol
    		 RO  
    Global end of trial date
    10 Nov 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    24 Oct 2024
    First version publication date
    24 Oct 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    67896049PUH3001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04565990
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Actelion Pharmaceuticals Ltd
    Sponsor organisation address
    16 Gewerbestrasse, 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
    10 Nov 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    10 Nov 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this trial was to assess the long-term safety of selexipag while providing continued selexipag treatment for subjects who were previously enrolled in an Actelion-sponsored study with selexipag and who derived benefit from selexipag in indications for which a positive benefit-risk has been established.
    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
    03 May 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belarus: 21
    Country: Number of subjects enrolled
    India: 4
    Country: Number of subjects enrolled
    Korea, Republic of: 4
    Country: Number of subjects enrolled
    Romania: 2
    Country: Number of subjects enrolled
    Taiwan: 3
    Country: Number of subjects enrolled
    Ukraine: 9
    Worldwide total number of subjects
    43
    EEA total number of subjects
    2
    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)
    37
    From 65 to 84 years
    6
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 43 subjects were rolled over from GRIPHON OL (originating study) and entered in this open label (OL) (SOMBRERO) study of which 36 subjects completed the study.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 Selexipag
    Arm description
    		 Subjects with pulmonary arterial hypertension (PAH) who completed the parent study AC-065A302 continued treatment with selexipag in this study (200 to 1600 micrograms [mcg] selexipag tablet orally twice daily [bid]) from Day 1 up to 28 months with the same individual maximum tolerated dose (iMTD) that they were taking at the end of their parent study. Subjects were then followed for safety up to 30 days after the last dose of selexipag. Eligible subjects were then followed up for safety for up to 30 days after the last dose of selexipag.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Selexipag
    Investigational medicinal product code
    JNJ-67896049
    Other name
    ACT-293987
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects with pulmonary arterial hypertension (PAH) who completed the parent study AC-065A302 continued treatment with selexipag in this study (200 to 1600 micrograms [mcg] selexipag tablet orally twice daily [bid]) from Day 1 up to 28 months with the same individual maximum tolerated dose (iMTD) that they were taking at the end of their parent study.

    Number of subjects in period 1
    		Selexipag
    Started
    43
    Completed
    36
    Not completed
    7
         Consent withdrawn by subject
    1
         Death
    5
         Lost to follow-up
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Selexipag
    Reporting group description
    		 Subjects with pulmonary arterial hypertension (PAH) who completed the parent study AC-065A302 continued treatment with selexipag in this study (200 to 1600 micrograms [mcg] selexipag tablet orally twice daily [bid]) from Day 1 up to 28 months with the same individual maximum tolerated dose (iMTD) that they were taking at the end of their parent study. Subjects were then followed for safety up to 30 days after the last dose of selexipag. Eligible subjects were then followed up for safety for up to 30 days after the last dose of selexipag.

    Reporting group values
    		 Selexipag Total
    Number of subjects
    		 43 43
    Title for AgeCategorical
    Units: subjects
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 37 37
        From 65 to 84 years
    		 6 6
        85 years and over
    		 0 0
    Title for AgeContinuous
    Units: years
        arithmetic mean (standard deviation)
    		 50.6 ( 13.25 ) -
    Title for Gender
    Units: subjects
        Female
    		 36 36
        Male
    		 7 7

    End points

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    End points reporting groups
    Reporting group title
    Selexipag
    Reporting group description
    		 Subjects with pulmonary arterial hypertension (PAH) who completed the parent study AC-065A302 continued treatment with selexipag in this study (200 to 1600 micrograms [mcg] selexipag tablet orally twice daily [bid]) from Day 1 up to 28 months with the same individual maximum tolerated dose (iMTD) that they were taking at the end of their parent study. Subjects were then followed for safety up to 30 days after the last dose of selexipag. Eligible subjects were then followed up for safety for up to 30 days after the last dose of selexipag.

    Primary: Number of Subjects With Treatment-emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Subjects With Treatment-emergent Adverse Events (TEAEs) [1]
    End point description
    Number of Subjects with TEAEs were reported. Adverse event (AE) was defined as any untoward medical occurrence in a clinical study subject administered a pharmaceutical (investigational or non investigational) product. An AE did not necessarily have a causal relationship with the pharmaceutical/biological agent under study. TEAEs were defined as AEs occurring at or after the initial administration of study intervention through the day of last dose plus 3 days. Data includes all TEAEs irrespective of whether they were serious or non-serious. Safety analysis set included all subjects who received at least 1 dose of study intervention in this study.
    End point type
    Primary
    End point timeframe
    From Day 1 up to 3 days after last dose of drug (up to 28 months 3 days)
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics was done, no inferential statistical analysis was performed.
    End point values
    		 Selexipag
    Number of subjects analysed
    43
    Units: Subjects
    22
    No statistical analyses for this end point

    Primary: Number of Subjects With TEAEs Leading to Premature Discontinuation of Selexipag

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    End point title
    		 Number of Subjects With TEAEs Leading to Premature Discontinuation of Selexipag [2]
    End point description
    Number of subjects with TEAEs leading to premature discontinuation of selexipag were reported. AE was defined as any untoward medical occurrence in a clinical study subject administered a pharmaceutical (investigational or non investigational) product. An AE did not necessarily have a causal relationship with the pharmaceutical/biological agent under study. TEAEs were defined as AEs occurring at or after the initial administration of study intervention through the day of last dose plus 3 days. Safety analysis set included all subjects who received at least 1 dose of study intervention in this study.
    End point type
    Primary
    End point timeframe
    From Day 1 up to 3 days after last dose of drug (up to 28 months 3 days)
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics was done, no inferential statistical analysis was performed.
    End point values
    		 Selexipag
    Number of subjects analysed
    43
    Units: Subjects
    0
    No statistical analyses for this end point

    Primary: Number of Pregnant Females With Maternal Exposure to Selexipag

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    End point title
    		 Number of Pregnant Females With Maternal Exposure to Selexipag [3]
    End point description
    Number of pregnant females with maternal exposure to selexipag were reported. Safety analysis set included all subjects who received at least 1 dose of study intervention in this study. Here, 'N' (number of subjects analysed) signifies subjects evaluable for this endpoint.
    End point type
    Primary
    End point timeframe
    From Day 1 up to 30 days after last dose of drug (up to 29 months)
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics was done, no inferential statistical analysis was performed.
    End point values
    		 Selexipag
    Number of subjects analysed
    36
    Units: Subjects
    0
    No statistical analyses for this end point

    Primary: Number of Subjects With TEAE Deaths

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    End point title
    		 Number of Subjects With TEAE Deaths [4]
    End point description
    Number of subjects with TEAE deaths during the study were reported. Safety analysis set included all subjects who received at least 1 dose of study intervention in this study.
    End point type
    Primary
    End point timeframe
    From Day 1 up to 3 days after last dose of drug (up to 28 months 3 days)
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics was done, no inferential statistical analysis was performed.
    End point values
    		 Selexipag
    Number of subjects analysed
    43
    Units: Subjects
    5
    No statistical analyses for this end point

    Primary: Number of Subjects With Treatment-emergent Serious Adverse Events (TESAEs)

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    End point title
    		 Number of Subjects With Treatment-emergent Serious Adverse Events (TESAEs) [5]
    End point description
    Number of subjects with TESAEs were reported. AE was defined as any untoward medical occurrence in a clinical study subject administered a pharmaceutical (investigational or non investigational) product. An AE did not necessarily have a causal relationship with the pharmaceutical/biological agent under study. A SAE was any untoward medical occurrence at any dose that: resulted in death, was life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or resulted in congenital anomaly/birth defect. TESAEs were defined as TSAEs occurring at or after the initial administration of study intervention through the day of last dose plus 3 days. Safety analysis set included all subjects who received at least 1 dose of study intervention in this study.
    End point type
    Primary
    End point timeframe
    From Day 1 up to 3 days after last dose of drug (up to 28 months 3 days)
    Notes
    [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Descriptive statistics was done, no inferential statistical analysis was performed.
    End point values
    		 Selexipag
    Number of subjects analysed
    43
    Units: Subjects
    7
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All cause mortality: From Day 1 up to 30 days after last dose of drug (up to 29 months), Other AEs and SAEs: From Day 1 up to 3 days after last dose of drug (up to 28 months 3 days)
    Adverse event reporting additional description
    Safety analysis set included all enrolled subjects who received at least 1 dose of study intervention in this study.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Selexipag
    Reporting group description
    		 Subjects with pulmonary arterial hypertension (PAH) who completed the parent study AC-065A303 (NCT01112306) continued treatment with selexipag in this study (200 to 1600 micrograms [mcg] selexipag tablet orally twice daily [bid]) from Day 1 up to 28 months with the same individual maximum tolerated dose (iMTD) that they were taking at the end of their parent study. Eligible subjects were then followed for safety up to 30 days after the last dose of selexipag.

    Serious adverse events
    		 Selexipag
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 43 (16.28%)
         number of deaths (all causes)
    5
         number of deaths resulting from adverse events
    5
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cardiac disorders
    Cardiac Failure Acute
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Respiratory, thoracic and mediastinal disorders
    Pulmonary Arterial Hypertension
         subjects affected / exposed
    3 / 43 (6.98%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 2
    Musculoskeletal and connective tissue disorders
    Systemic Scleroderma
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Covid-19 Pneumonia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Subacute Endocarditis
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Syphilis
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Selexipag
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    20 / 43 (46.51%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Haemangioma of liver
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Benign biliary neoplasm
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 43 (4.65%)
         occurrences all number
    2
    Pain
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Heavy menstrual bleeding
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Respiratory disorder
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Pulmonary arterial hypertension
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Dyspnoea
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Investigations
    Double stranded DNA antibody positive
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Complement factor C4 decreased
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Complement factor C3 decreased
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Vaccination complication
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Rib fracture
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Cardiac disorders
    Pericardial effusion
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Sinus tachycardia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Atrial fibrillation
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Thrombocytopenia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Anaemia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Gastric mucosa erythema
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Chronic gastritis
         subjects affected / exposed
    2 / 43 (4.65%)
         occurrences all number
    2
    Hepatobiliary disorders
    Autoimmune hepatitis
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Steatohepatitis
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Non-alcoholic fatty liver
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Cholecystitis chronic
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Skin ulcer
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Alopecia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Endocrine disorders
    Goitre
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Infections and infestations
    Covid-19
         subjects affected / exposed
    4 / 43 (9.30%)
         occurrences all number
    4
    Influenza
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Otitis media acute
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Respiratory tract infection
         subjects affected / exposed
    2 / 43 (4.65%)
         occurrences all number
    3
    Urinary tract infection
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Hyperuricaemia
         subjects affected / exposed
    1 / 43 (2.33%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    08 Dec 2020
    The purpose of this amendment was to adapt safety reporting processes as part of the full transition of Actelion into Janssen, to align with transCelerate protocol template, coronavirus disease-2019 (COVID-19).
    26 Oct 2021
    The purpose of this amendment was to clarify the definition of end-of-study (EOS) and safety reporting requirements for subjects who completed treatment in the study and who continued with selexipag treatment by rolling over into a post-trial access (PTA) program or Study NOPRODPAPUH3001 (PLATYPUS).

    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.
    None reported
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    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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