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    Clinical Trial Results:
    Ribociclib-endocrine combination therapy versus chemotherapy as 1st line treatment in patients with visceral metastatic breast cancer. A multicenter, randomized phase III trial.

    Summary
    EudraCT number
    		 2018-003648-22
    Trial protocol
    		 BE   AT  
    Global end of trial date
    15 Apr 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Jun 2022
    First version publication date
    26 Jun 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SAKK_2118
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03905343
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Swiss Group for Clinical Cancer Research (SAKK)
    Sponsor organisation address
    Effingerstrasse 33, Bern, Switzerland, 3008
    Public contact
    Head Regulatory Affairs, Swiss Group for Clinical Cancer, +41 31389 91 91, sakkcc@sakk.ch
    Scientific contact
    Head Regulatory Affairs, Swiss Group for Clinical Cancer, +41 31389 91 91, sakkcc@sakk.ch
    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 Dec 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    15 Apr 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    15 Apr 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The aim of this trial is to assess if patients treated with the combination of ribociclib and endocrine therapy respond to treatment as fast as patients treated with chemotherapy only, without decreasing their quality of life (QoL).
    Protection of trial subjects
    Protection of trial subjects was ensured by Safety Monitoring, i.e. assessment of adverse events, serious adverse events, adverse drug reactions, and the continous assessment of laboratory values and vital signs.
    Background therapy
    		 None
    Evidence for comparator
    		 Mono-chemotherapy according to local guidelines for at least 12 weeks or until progression was used as reference therapy. The choice of mono-chemotherapy was up to the investigator. It had to be registered and reimbursed by health insurances. Metronomic chemotherapy, combination chemotherapy and antiangiogenic therapy was not allowed.
    Actual start date of recruitment
    26 Aug 2019
    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
    Belgium: 1
    Country: Number of subjects enrolled
    Switzerland: 24
    Worldwide total number of subjects
    25
    EEA total number of subjects
    1
    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)
    10
    From 65 to 84 years
    15
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The recruitment phase started on 26-Aug-2019 and was prematurely stopped due to financial reasons on 25-Nov-2021 after the recruitment of 25 out of 400 planned patients. Last patient was enrolled on 12-Nov-2020. Twenty-four patients at ten centres were enrolled in Switzerland and one patient in Belgium.

    Pre-assignment
    Screening details
    		 Eligibility criteria of a patient were checked by the investigator. Once a patient fullfils all inclusion criteria and not any of the exclusion criteria, he/she was randomized.

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Arm A
    Arm description
    		 Ribociclib and endocrine therapy. The selection of endocrine treatment was according to local guidelines and depended on registration and reimbursement by the health insurance. Endocrine therapy consisted of a registered aromatase inhibitor or fulvestrant, only if reimbursed by the health insurance.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ribociclib
    Investigational medicinal product code
    Other name
    Kisqali®
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    600 mg p.o. on day 1 to day 21, q4w

    Investigational medicinal product name
    Endocrine therapy (according to local quidelines)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Not assigned
    Routes of administration
    Not mentioned
    Dosage and administration details
    According to local guidelines.

    Arm title
    		 Arm B
    Arm description
    		 Mono-chemotherapy for at least 12 weeks or until progression. Chemotherapy had to be given according to local guidelines. The choice of mono-chemotherapy was up to the investigator. It had to be registered and reimbursed by health insurances. Metronomic chemotherapy, combination chemotherapy and antiangiogenic therapy was not allowed. After at least 12 weeks and no PD, the investigator could perform a riskbenefit assessment and decide whether to continue the chemotherapy or to start a maintenance endocrine therapy with or without ribociclib.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Mono-chemotherapy
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Not assigned
    Routes of administration
    Not mentioned
    Dosage and administration details
    Chemotherapy had to be given according to local guidelines.

    Number of subjects in period 1
    		Arm A Arm B
    Started
    13
    12
    Completed
    13
    12
    Period 2
    Period 2 title
    Treatment Phase
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Arm A
    Arm description
    		 Ribociclib and endocrine therapy. The selection of endocrine treatment was according to local guidelines and depended on registration and reimbursement by the health insurance. Endocrine therapy consisted of a registered aromatase inhibitor or fulvestrant, only if reimbursed by the health insurance.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ribociclib
    Investigational medicinal product code
    Other name
    Kisqali®
    Pharmaceutical forms
    Coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    600 mg p.o. on day 1 to day 21, q4w

    Investigational medicinal product name
    Endocrine therapy (according to local quidelines)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Not assigned
    Routes of administration
    Not mentioned
    Dosage and administration details
    According to local guidelines.

    Arm title
    		 Arm B
    Arm description
    		 Mono-chemotherapy for at least 12 weeks or until progression. Chemotherapy had to be given according to local guidelines. The choice of mono-chemotherapy was up to the investigator. It had to be registered and reimbursed by health insurances. Metronomic chemotherapy, combination chemotherapy and antiangiogenic therapy was not allowed. After at least 12 weeks and no PD, the investigator could perform a riskbenefit assessment and decide whether to continue the chemotherapy or to start a maintenance endocrine therapy with or without ribociclib.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Mono-chemotherapy
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Not assigned
    Routes of administration
    Not mentioned
    Dosage and administration details
    Chemotherapy had to be given according to local guidelines.

    Number of subjects in period 2
    		Arm A Arm B
    Started
    13
    12
    Completed
    0
    0
    Not completed
    13
    12
         Physician decision
    1
    1
         Study termination by sponsor
    7
    4
         Progressive disease
    5
    6
         Other - Second malignancy
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Arm A
    Reporting group description
    		 Ribociclib and endocrine therapy. The selection of endocrine treatment was according to local guidelines and depended on registration and reimbursement by the health insurance. Endocrine therapy consisted of a registered aromatase inhibitor or fulvestrant, only if reimbursed by the health insurance.

    Reporting group title
    Arm B
    Reporting group description
    		 Mono-chemotherapy for at least 12 weeks or until progression. Chemotherapy had to be given according to local guidelines. The choice of mono-chemotherapy was up to the investigator. It had to be registered and reimbursed by health insurances. Metronomic chemotherapy, combination chemotherapy and antiangiogenic therapy was not allowed. After at least 12 weeks and no PD, the investigator could perform a riskbenefit assessment and decide whether to continue the chemotherapy or to start a maintenance endocrine therapy with or without ribociclib.

    Reporting group values
    		 Arm A Arm B Total
    Number of subjects
    		 13 12 25
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 6 4 10
        From 65-84 years
    		 7 8 15
    Gender categorical
    Units: Subjects
        Female
    		 13 12 25
        Male
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Arm A
    Reporting group description
    		 Ribociclib and endocrine therapy. The selection of endocrine treatment was according to local guidelines and depended on registration and reimbursement by the health insurance. Endocrine therapy consisted of a registered aromatase inhibitor or fulvestrant, only if reimbursed by the health insurance.

    Reporting group title
    Arm B
    Reporting group description
    		 Mono-chemotherapy for at least 12 weeks or until progression. Chemotherapy had to be given according to local guidelines. The choice of mono-chemotherapy was up to the investigator. It had to be registered and reimbursed by health insurances. Metronomic chemotherapy, combination chemotherapy and antiangiogenic therapy was not allowed. After at least 12 weeks and no PD, the investigator could perform a riskbenefit assessment and decide whether to continue the chemotherapy or to start a maintenance endocrine therapy with or without ribociclib.
    Reporting group title
    Arm A
    Reporting group description
    		 Ribociclib and endocrine therapy. The selection of endocrine treatment was according to local guidelines and depended on registration and reimbursement by the health insurance. Endocrine therapy consisted of a registered aromatase inhibitor or fulvestrant, only if reimbursed by the health insurance.

    Reporting group title
    Arm B
    Reporting group description
    		 Mono-chemotherapy for at least 12 weeks or until progression. Chemotherapy had to be given according to local guidelines. The choice of mono-chemotherapy was up to the investigator. It had to be registered and reimbursed by health insurances. Metronomic chemotherapy, combination chemotherapy and antiangiogenic therapy was not allowed. After at least 12 weeks and no PD, the investigator could perform a riskbenefit assessment and decide whether to continue the chemotherapy or to start a maintenance endocrine therapy with or without ribociclib.

    Subject analysis set title
    Arm A
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    FAS = PPS The FAS was defined as all randomized patients who received at least one dose of trial treatment excluding patients with major eligibility violations. Following the intention-to-treat principle, patients in this set were analyzed according to the treatment they were randomized to.

    Subject analysis set title
    Arm B
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    FAS = PPS The FAS was defined as all randomized patients who received at least one dose of trial treatment excluding patients with major eligibility violations. Following the intention-to-treat principle, patients in this set were analyzed according to the treatment they were randomized to. One patient had a second tumor present at baseline, which was detected when the patient was already on trial treatment. The patient violated inclusion criterion 6.1.6 and was excluded from FAS (and PPS).

    Primary: PE - Quality of life adjusted early disease control (composite EP - 1a/2 Disease Control - Response at week 12)

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    End point title
    		 PE - Quality of life adjusted early disease control (composite EP - 1a/2 Disease Control - Response at week 12) [1]
    End point description
    The primary enpoint, quality of life-adjusted early disease control at 12 weeks is a composite endpoint. The first component is disease control (CR, PR or SD according to RECIST v1.1) at 12 weeks (+1 week for delayed assessments). If a patient had no assessment at 12 weeks but CR, PR or SD thereafter it was counted as SD at 12 weeks. The second component was defined as follows: FACTB TOI score does not worsen by 5 points or more during the first 12 weeks after randomization (+1 week for delayed assessments).
    End point type
    Primary
    End point timeframe
    Responses at week 12
    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: Due to the low sample size, statistical analysis was not performed and data were analyzed descriptively.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
    number (not applicable)
        Partial response (PR)
    30.8
    27.3
        Stable disease (SD)
    69.2
    54.5
        Progressive disease (PD)
    0
    9.1
        Not evaluable (NA)
    0
    9.1
    No statistical analyses for this end point

    Primary: PE - Quality of life adjusted early disease control (composite EP - 1b/2 Disease Control - Disease control at week 12)

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    End point title
    		 PE - Quality of life adjusted early disease control (composite EP - 1b/2 Disease Control - Disease control at week 12) [2]
    End point description
    The primary enpoint, quality of life-adjusted early disease control at 12 weeks is a composite endpoint. The first component is disease control (CR, PR or SD according to RECIST v1.1) at 12 weeks (+1 week for delayed assessments). If a patient had no assessment at 12 weeks but CR, PR or SD thereafter it was counted as SD at 12 weeks. The second component was defined as follows: FACTB TOI score does not worsen by 5 points or more during the first 12 weeks after randomization (+1 week for delayed assessments).
    End point type
    Primary
    End point timeframe
    Disease control at week 12.
    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: Due to the low sample size, statistical analysis was not performed and data were analyzed descriptively.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
    number (not applicable)
        Yes
    100
    81.8
        No
    0
    18.2
    No statistical analyses for this end point

    Primary: PE - Quality of life adjusted early disease control (composite EP - 2/2 FACT-B TOI worsening during first 12 weeks)

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    End point title
    		 PE - Quality of life adjusted early disease control (composite EP - 2/2 FACT-B TOI worsening during first 12 weeks) [3]
    End point description
    The primary enpoint, quality of life-adjusted early disease control at 12 weeks is a composite endpoint. The first component is disease control (CR, PR or SD according to RECIST v1.1) at 12 weeks (+1 week for delayed assessments). If a patient had no assessment at 12 weeks but CR, PR or SD thereafter it was counted as SD at 12 weeks. The second component was defined as follows: FACTB TOI score does not worsen by 5 points or more during the first 12 weeks after randomization (+1 week for delayed assessments).
    End point type
    Primary
    End point timeframe
    TOI worsening of at least 5 points within first 12 weeks
    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: Due to the low sample size, statistical analysis was not performed and data were analyzed descriptively.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
    number (not applicable)
        No
    53.8
    54.5
        Yes
    38.5
    45.5
        NA
    7.7
    0
    No statistical analyses for this end point

    Primary: PE - Quality of life adjusted early disease control (composite EP - Composite)

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    End point title
    		 PE - Quality of life adjusted early disease control (composite EP - Composite) [4]
    End point description
    The primary enpoint, quality of life-adjusted early disease control at 12 weeks is a composite endpoint. The first component is disease control (CR, PR or SD according to RECIST v1.1) at 12 weeks (+1 week for delayed assessments). If a patient had no assessment at 12 weeks but CR, PR or SD thereafter it was counted as SD at 12 weeks. The second component was defined as follows: FACTB TOI score does not worsen by 5 points or more during the first 12 weeks after randomization (+1 week for delayed assessments).
    End point type
    Primary
    End point timeframe
    Composite of quality of life-adjusted disease control at week 12
    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: Due to the low sample size, statistical analysis was not performed and data were analyzed descriptively.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
    number (not applicable)
        Yes
    53.8
    45.5
        No
    46.2
    54.5
    No statistical analyses for this end point

    Primary: PE - Quality of life adjusted early disease control (composite EP - Composite rate)

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    End point title
    		 PE - Quality of life adjusted early disease control (composite EP - Composite rate) [5]
    End point description
    The primary enpoint, quality of life-adjusted early disease control at 12 weeks is a composite endpoint. The first component is disease control (CR, PR or SD according to RECIST v1.1) at 12 weeks (+1 week for delayed assessments). If a patient had no assessment at 12 weeks but CR, PR or SD thereafter it was counted as SD at 12 weeks. The second component was defined as follows: FACTB TOI score does not worsen by 5 points or more during the first 12 weeks after randomization (+1 week for delayed assessments).
    End point type
    Primary
    End point timeframe
    Composit quality of life-adjusted disease control rate at week 12
    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: Due to the low sample size, statistical analysis was not performed and data were analyzed descriptively.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
        number (confidence interval 95%)
    53.8 (25.1 to 80.8)
    45.5 (16.7 to 76.6)
    No statistical analyses for this end point

    Secondary: SE - Disease control at week 12

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    End point title
    		 SE - Disease control at week 12
    End point description
    Disease control at week 12 (see also primary endpoint)
    End point type
    Secondary
    End point timeframe
    12 weeks
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
        number (confidence interval 95%)
    100.0 (75.3 to 100.0)
    81.8 (48.2 to 97.7)
    No statistical analyses for this end point

    Secondary: SE - Objective response rate (ORR) 1/2 Best overall response

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    End point title
    		 SE - Objective response rate (ORR) 1/2 Best overall response
    End point description
    Best overall response according to RECIST v1.1, i.e. CR/PR rates
    End point type
    Secondary
    End point timeframe
    From Baseline until EoS
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
    number (not applicable)
        Complete remission (CR)
    0
    0
        Partial response (PR)
    61.5
    36.4
        Stable disease (SD)
    38.5
    63.6
    No statistical analyses for this end point

    Secondary: SE - Objective response rate (ORR) 2/2 Objective response rate

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    End point title
    		 SE - Objective response rate (ORR) 2/2 Objective response rate
    End point description
    Objective response rate
    End point type
    Secondary
    End point timeframe
    From baseline until EoS.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Patients (%)
        number (confidence interval 95%)
    61.5 (31.6 to 86.1)
    36.4 (10.9 to 69.2)
    No statistical analyses for this end point

    Secondary: SE - Objective response rate (ORR) - Time to objective response

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    End point title
    		 SE - Objective response rate (ORR) - Time to objective response
    End point description
    Time to event analysis (Kaplan-Meier) for patient with ORR partial response; 8 patients in arm A and 4 in arm B reached PR.
    End point type
    Secondary
    End point timeframe
    Time to event from enrollment.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13 [6]
    11 [7]
    Units: Months
        median (full range (min-max))
    4.1 (1.1 to 8.1)
    1.9 (0.9 to 8.2)
    Notes
    [6] - Analysis only for patients with ORR = PR.
    [7] - Analysis only for patients with ORR = PR.
    No statistical analyses for this end point

    Secondary: SE - Progression-free survival (PFS)

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    End point title
    		 SE - Progression-free survival (PFS)
    End point description
    Time to event analysis (Kaplan-Meier) for progression-free survival (PFS); 6 patients in arm A and 6 in arm B had experienced disease progression. NOTE: UPPER LIMITS FOR 95% CI FOR MEDIAN PFS WERE NOT REACHED. HOWEVER, DUMMY VALUES FOR UPPER 95% CI LIMITS ENTERED DUE TO DATABASE RESTRICTIONS.
    End point type
    Secondary
    End point timeframe
    Time to event from enrollment.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Months
        median (confidence interval 95%)
    11.0 (5.5 to 100000)
    10.6 (2.6 to 100000)
    No statistical analyses for this end point

    Secondary: SE - Time to treatment failure (TTF)

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    End point title
    		 SE - Time to treatment failure (TTF)
    End point description
    Time to event analysis (Kaplan-Meier) for time to treatment failure (TTF); all patients stopped trial treatment before the planned 3 years of treatment. One patient in arm A and one in arm B were treated beyond progression (see Section 6.5). For these patients TTF was longer than PFS.
    End point type
    Secondary
    End point timeframe
    Time to event from enrollment.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Months
        median (confidence interval 95%)
    10.2 (6.4 to 11.4)
    5.7 (2.6 to 12.9)
    No statistical analyses for this end point

    Secondary: SE - Overall survival (OS)

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    End point title
    		 SE - Overall survival (OS)
    End point description
    Time to event analysis (Kaplan-Meier) for overall survival (OS); two patients in arm A and one patient in arm B had died. NOTE: MEDIAN OS TIMES WERE NOT REACHED. DUMMY DATA ENTERED DUE TO DATABASE RESTRICTIONS.
    End point type
    Secondary
    End point timeframe
    Time to event from enrollment.
    End point values
    		 Arm A Arm B
    Number of subjects analysed
    13
    11
    Units: Months
        median (confidence interval 95%)
    0 (0 to 0)
    0 (0 to 0)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From enrollment until EoS.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    Arm A (SAF)
    Reporting group description
    		 -

    Reporting group title
    Arm B (SAF)
    Reporting group description
    		 -

    Serious adverse events
    		 Arm A (SAF) Arm B (SAF)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 13 (46.15%)
    2 / 12 (16.67%)
         number of deaths (all causes)
    2
    1
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Rectal carcinoma
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Embolism
         subjects affected / exposed
    3 / 13 (23.08%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastritis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Uterine hemorrhage
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pleural effusion
         subjects affected / exposed
    2 / 13 (15.38%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Lung infection
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin infection
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Arm A (SAF) Arm B (SAF)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 13 (53.85%)
    8 / 12 (66.67%)
    Investigations
    Neutrophil count decreased
         subjects affected / exposed
    6 / 13 (46.15%)
    1 / 12 (8.33%)
         occurrences all number
    13
    4
    Platelet count decreased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    White blood cell count decreased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Fracture
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    Vascular disorders
    Embolism arterial
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Hypertension
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Embolism
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Nervous system disorders
    Aphonia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    2
    Skin and subcutaneous tissue disorders
    Rash maculo-papular
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    2
    Urticaria
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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.
    The study was terminated prematurely by the sponsor. All patients stopped trial treatment before the planned three years of treatment. The sample size was low (planned 400 patients; actual: 25 patients). Thus data have to be interpreted with caution.
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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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