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    Clinical Trial Results:
    An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome

    Summary
    EudraCT number
    		 2011-002379-40
    Trial protocol
    		 SE   DE   Outside EU/EEA   GB   ES   DK   IT   FR   NL   BE  
    Global end of trial date
    17 Sep 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Apr 2016
    First version publication date
    13 Apr 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CAFQ056B2278
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01433354
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001003-PIP01-10
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    17 Sep 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Sep 2014
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective was to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:  Incidence and severity of adverse events and serious adverse events  Changes in vital signs, laboratory assessments, and ECGs  Monitoring the hypothalamic-pituitary-adrenal/thyroid axis function and childhood developmental milestones
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    23 Nov 2011
    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
    Israel: 1
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Spain: 12
    Country: Number of subjects enrolled
    Sweden: 3
    Country: Number of subjects enrolled
    United Kingdom: 4
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Germany: 15
    Country: Number of subjects enrolled
    Italy: 9
    Country: Number of subjects enrolled
    United States: 47
    Country: Number of subjects enrolled
    Australia: 6
    Country: Number of subjects enrolled
    Switzerland: 10
    Worldwide total number of subjects
    119
    EEA total number of subjects
    55
    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)
    105
    Adults (18-64 years)
    14
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study was conducted at 28 centres in 13 countries.

    Pre-assignment
    Screening details
    		 A total of 120 subjects were enrolled in the study, of which 119 subjects received study medication.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    As the study was an open-label study, this section was not applicable.

    Arms
    Arm title
    		 AFQ056
    Arm description
    		 Subjects treated with AFQ056 from a feeder study and entered the open-label extension study, were administered with AFQ056 capsule starting dose of 25 milligram (mg) twice daily (bid) and then titrated to 50 mg bid, 75 mg bid and 100 mg bid at weekly intervals.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mavoglurant
    Investigational medicinal product code
    AFQ056
    Other name
    Pharmaceutical forms
    Capsule, hard
    Routes of administration
    Oral use
    Dosage and administration details
    AFQ056 (25 mg bid, 50 mg bid, 75 mg bid and 100 mg bid) was administered at weekly intervals. 25 mg bid = one 25 mg capsule taken twice a day 50 mg bid = two 25 mg capsules taken twice a day 75 mg bid = three 25 mg capsules taken twice a day 100 mg bid = one 100 mg capsule taken twice a day

    Number of subjects in period 1
    		AFQ056
    Started
    119
    Completed
    0
    Not completed
    119
         Consent withdrawn by subject
    3
         Adverse event, non-fatal
    6
         Administrative Problems
    90
         Lost to follow-up
    1
         Protocol deviation
    2
         Lack of efficacy
    17

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    AFQ056
    Reporting group description
    		 Subjects treated with AFQ056 from a feeder study and entered the open-label extension study, were administered with AFQ056 capsule starting dose of 25 milligram (mg) twice daily (bid) and then titrated to 50 mg bid, 75 mg bid and 100 mg bid at weekly intervals.

    Reporting group values
    		 AFQ056 Total
    Number of subjects
    		 119 119
    Age categorical
    Units: Subjects
        12 to 19 years
    		 119 119
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 15.2 ( 1.75 ) -
    Gender categorical
    Units: Subjects
        Female
    		 13 13
        Male
    		 106 106
    Subject analysis sets

    Subject analysis set title
    AFQ056 25 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 25 mg bid during the open-label treatment period.

    Subject analysis set title
    AFQ056 50 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 50 mg bid during the open-label treatment period.

    Subject analysis set title
    Prior to extension study first dose
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 during the core study and entered into the open-label treatment extension study.

    Subject analysis set title
    AFQ056 100 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 100 mg bid during the open-label treatment period

    Subject analysis set title
    AFQ056 75 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 75 mg bid during the open-label treatment period

    Subject analysis set title
    Category 1
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients who entered into this open-label treatment study within one week of completion of Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age were not required to undergo a screening visit. Patients entered the study directly with the baseline (V2) visit as the first visit to be conducted. This baseline visit (V2) was conducted either during the same day with the last study visit of the previous study or at a different date but not more than one week apart.

    Subject analysis set title
    Category 2
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients who entered into this open-label treatment study from Study CAFQ056B2214 or Study CAFQ056B2131, or another study of AFQ056 which included FXS patients below 18 years of age could do so more than one week after completion of the last visit of the respective previous study if any of the following reasons applied:  The patient discontinued the previous study due to lack of tolerability of the maximum dose in their assigned treatment group (entry into the extension trial could occur no sooner than the patient’s originally-scheduled completion of the previous study)  The current open-label treatment study was pending health authority and/or ethics approval or other reasons prevented the patient from entering into this open-label treatment study earlier  unavailability of the current study when the patient completed the previous study

    Subject analysis sets values
    		 AFQ056 25 mg bid AFQ056 50 mg bid Prior to extension study first dose AFQ056 100 mg bid AFQ056 75 mg bid Category 1 Category 2
    Number of subjects
    119
    118
    31
    108
    116
    31
    88
    Age categorical
    Units: Subjects
        12 to 19 years
    119
    118
    31
    108
    116
    31
    88
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    ( )
    ( )
    15.4 ( 1.61 )
    15.1 ( 1.81 )
    Gender categorical
    Units: Subjects
        Female
        Male

    End points

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    End points reporting groups
    Reporting group title
    AFQ056
    Reporting group description
    		 Subjects treated with AFQ056 from a feeder study and entered the open-label extension study, were administered with AFQ056 capsule starting dose of 25 milligram (mg) twice daily (bid) and then titrated to 50 mg bid, 75 mg bid and 100 mg bid at weekly intervals.

    Subject analysis set title
    AFQ056 25 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 25 mg bid during the open-label treatment period.

    Subject analysis set title
    AFQ056 50 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 50 mg bid during the open-label treatment period.

    Subject analysis set title
    Prior to extension study first dose
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 during the core study and entered into the open-label treatment extension study.

    Subject analysis set title
    AFQ056 100 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 100 mg bid during the open-label treatment period

    Subject analysis set title
    AFQ056 75 mg bid
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Subjects were administered with AFQ056 75 mg bid during the open-label treatment period

    Subject analysis set title
    Category 1
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients who entered into this open-label treatment study within one week of completion of Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age were not required to undergo a screening visit. Patients entered the study directly with the baseline (V2) visit as the first visit to be conducted. This baseline visit (V2) was conducted either during the same day with the last study visit of the previous study or at a different date but not more than one week apart.

    Subject analysis set title
    Category 2
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Patients who entered into this open-label treatment study from Study CAFQ056B2214 or Study CAFQ056B2131, or another study of AFQ056 which included FXS patients below 18 years of age could do so more than one week after completion of the last visit of the respective previous study if any of the following reasons applied:  The patient discontinued the previous study due to lack of tolerability of the maximum dose in their assigned treatment group (entry into the extension trial could occur no sooner than the patient’s originally-scheduled completion of the previous study)  The current open-label treatment study was pending health authority and/or ethics approval or other reasons prevented the patient from entering into this open-label treatment study earlier  unavailability of the current study when the patient completed the previous study

    Primary: Number of subjects with adverse events (AEs), treatment related AEs, AEs leading to discontinuation, AEs by severity, serious adverse events (SAEs) and SAEs leading to discontinuation during the study

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    End point title
    		 Number of subjects with adverse events (AEs), treatment related AEs, AEs leading to discontinuation, AEs by severity, serious adverse events (SAEs) and SAEs leading to discontinuation during the study [1]
    End point description
    Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which patients entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study.  AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 patients (‘Prior to Ext. first dose’).  AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated.
    End point type
    Primary
    End point timeframe
    Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
    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: Only descriptive summary statistics was planned for this outcome measure.
    End point values
    		 AFQ056 Prior to extension study first dose AFQ056 25 mg bid AFQ056 50 mg bid AFQ056 75 mg bid AFQ056 100 mg bid
    Number of subjects analysed
    119 [2]
    31 [3]
    119 [4]
    118 [5]
    116 [6]
    108 [7]
    Units: Number of subjects
        At least one AE
    110
    10
    36
    41
    44
    90
        At least one severe AE
    8
    0
    1
    1
    1
    5
        Any serious or significant AE
    4
    0
    0
    1
    0
    3
        SAE
    4
    0
    0
    1
    0
    3
        Discontinued due to AE
    6
    1
    2
    2
    0
    3
        Discontinued due to SAE
    1
    0
    0
    0
    0
    1
        Discontinued due to non serious AE
    5
    1
    2
    2
    0
    2
    Notes
    [2] - Note: A patient can be counted or summarized in more than one dose column.
    [3] - Note: A patient can be counted or summarized in more than one dose column.
    [4] - Note: A patient can be counted or summarized in more than one dose column.
    [5] - Note: A patient can be counted or summarized in more than one dose column.
    [6] - Note: A patient can be counted or summarized in more than one dose column.
    [7] - Note: A patient can be counted or summarized in more than one dose column.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse events are reported in this record from First Patient First Treatment until Last Patient Last Visit.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.0
    Reporting groups
    Reporting group title
    Prior to Ext first dose
    Reporting group description
    		 Prior to Ext first dose

    Reporting group title
    AFQ 25
    Reporting group description
    		 AFQ 25

    Reporting group title
    AFQ 100
    Reporting group description
    		 AFQ 100

    Reporting group title
    AFQ 75
    Reporting group description
    		 AFQ 75

    Reporting group title
    Total
    Reporting group description
    		 Total

    Reporting group title
    AFQ 50
    Reporting group description
    		 AFQ 50

    Serious adverse events
    		 Prior to Ext first dose AFQ 25 AFQ 100 AFQ 75 Total AFQ 50
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    3 / 108 (2.78%)
    0 / 116 (0.00%)
    4 / 119 (3.36%)
    1 / 118 (0.85%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Joint dislocation
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    1 / 108 (0.93%)
    0 / 116 (0.00%)
    1 / 119 (0.84%)
    0 / 118 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Foreign body
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    0 / 108 (0.00%)
    0 / 116 (0.00%)
    1 / 119 (0.84%)
    1 / 118 (0.85%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    1 / 108 (0.93%)
    0 / 116 (0.00%)
    1 / 119 (0.84%)
    0 / 118 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    1 / 108 (0.93%)
    0 / 116 (0.00%)
    1 / 119 (0.84%)
    0 / 118 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    1 / 108 (0.93%)
    0 / 116 (0.00%)
    1 / 119 (0.84%)
    0 / 118 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Prior to Ext first dose AFQ 25 AFQ 100 AFQ 75 Total AFQ 50
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 31 (19.35%)
    23 / 119 (19.33%)
    74 / 108 (68.52%)
    34 / 116 (29.31%)
    103 / 119 (86.55%)
    30 / 118 (25.42%)
    Investigations
    Weight increased
         subjects affected / exposed
    1 / 31 (3.23%)
    1 / 119 (0.84%)
    5 / 108 (4.63%)
    0 / 116 (0.00%)
    7 / 119 (5.88%)
    0 / 118 (0.00%)
         occurrences all number
    1
    1
    5
    0
    7
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 31 (0.00%)
    4 / 119 (3.36%)
    8 / 108 (7.41%)
    4 / 116 (3.45%)
    15 / 119 (12.61%)
    1 / 118 (0.85%)
         occurrences all number
    0
    5
    8
    5
    19
    1
    Psychomotor hyperactivity
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 119 (1.68%)
    3 / 108 (2.78%)
    1 / 116 (0.86%)
    9 / 119 (7.56%)
    3 / 118 (2.54%)
         occurrences all number
    0
    2
    3
    1
    9
    3
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 119 (1.68%)
    5 / 108 (4.63%)
    3 / 116 (2.59%)
    11 / 119 (9.24%)
    2 / 118 (1.69%)
         occurrences all number
    0
    2
    5
    3
    12
    2
    Pyrexia
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    4 / 108 (3.70%)
    2 / 116 (1.72%)
    7 / 119 (5.88%)
    1 / 118 (0.85%)
         occurrences all number
    0
    0
    7
    5
    13
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 119 (1.68%)
    8 / 108 (7.41%)
    0 / 116 (0.00%)
    13 / 119 (10.92%)
    5 / 118 (4.24%)
         occurrences all number
    1
    2
    14
    0
    22
    5
    Diarrhoea
         subjects affected / exposed
    1 / 31 (3.23%)
    2 / 119 (1.68%)
    5 / 108 (4.63%)
    1 / 116 (0.86%)
    12 / 119 (10.08%)
    4 / 118 (3.39%)
         occurrences all number
    1
    2
    6
    1
    14
    4
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 119 (0.84%)
    7 / 108 (6.48%)
    4 / 116 (3.45%)
    12 / 119 (10.08%)
    1 / 118 (0.85%)
         occurrences all number
    0
    1
    9
    4
    15
    1
    Epistaxis
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    3 / 108 (2.78%)
    2 / 116 (1.72%)
    6 / 119 (5.04%)
    2 / 118 (1.69%)
         occurrences all number
    0
    0
    6
    2
    10
    2
    Psychiatric disorders
    Aggression
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 119 (2.52%)
    12 / 108 (11.11%)
    4 / 116 (3.45%)
    19 / 119 (15.97%)
    3 / 118 (2.54%)
         occurrences all number
    0
    4
    22
    8
    36
    3
    Agitation
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 119 (0.84%)
    4 / 108 (3.70%)
    0 / 116 (0.00%)
    6 / 119 (5.04%)
    1 / 118 (0.85%)
         occurrences all number
    0
    1
    5
    0
    8
    2
    Anxiety
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 119 (2.52%)
    11 / 108 (10.19%)
    0 / 116 (0.00%)
    15 / 119 (12.61%)
    1 / 118 (0.85%)
         occurrences all number
    0
    3
    12
    0
    16
    1
    Initial insomnia
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 119 (2.52%)
    9 / 108 (8.33%)
    4 / 116 (3.45%)
    18 / 119 (15.13%)
    2 / 118 (1.69%)
         occurrences all number
    0
    3
    9
    6
    20
    2
    Insomnia
         subjects affected / exposed
    1 / 31 (3.23%)
    4 / 119 (3.36%)
    12 / 108 (11.11%)
    5 / 116 (4.31%)
    25 / 119 (21.01%)
    10 / 118 (8.47%)
         occurrences all number
    1
    4
    12
    5
    31
    10
    Irritability
         subjects affected / exposed
    0 / 31 (0.00%)
    3 / 119 (2.52%)
    9 / 108 (8.33%)
    3 / 116 (2.59%)
    15 / 119 (12.61%)
    1 / 118 (0.85%)
         occurrences all number
    0
    3
    9
    4
    17
    1
    Infections and infestations
    Ear infection
         subjects affected / exposed
    0 / 31 (0.00%)
    2 / 119 (1.68%)
    5 / 108 (4.63%)
    1 / 116 (0.86%)
    8 / 119 (6.72%)
    0 / 118 (0.00%)
         occurrences all number
    0
    2
    6
    1
    9
    0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 31 (0.00%)
    0 / 119 (0.00%)
    5 / 108 (4.63%)
    1 / 116 (0.86%)
    8 / 119 (6.72%)
    2 / 118 (1.69%)
         occurrences all number
    0
    0
    6
    1
    9
    2
    Influenza
         subjects affected / exposed
    0 / 31 (0.00%)
    1 / 119 (0.84%)
    6 / 108 (5.56%)
    2 / 116 (1.72%)
    8 / 119 (6.72%)
    0 / 118 (0.00%)
         occurrences all number
    0
    1
    6
    2
    9
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 31 (3.23%)
    3 / 119 (2.52%)
    9 / 108 (8.33%)
    3 / 116 (2.59%)
    17 / 119 (14.29%)
    5 / 118 (4.24%)
         occurrences all number
    1
    6
    12
    3
    28
    6
    Nasopharyngitis
         subjects affected / exposed
    0 / 31 (0.00%)
    7 / 119 (5.88%)
    25 / 108 (23.15%)
    8 / 116 (6.90%)
    35 / 119 (29.41%)
    3 / 118 (2.54%)
         occurrences all number
    0
    7
    34
    9
    55
    5
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    2 / 31 (6.45%)
    0 / 119 (0.00%)
    0 / 108 (0.00%)
    1 / 116 (0.86%)
    5 / 119 (4.20%)
    2 / 118 (1.69%)
         occurrences all number
    2
    0
    0
    1
    5
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Oct 2011
    Modified the inclusion criteria with respect to enrolment of female subjects of childbearing potential had to undergo additional pregnancy testing.
    13 Aug 2013
    The criteria for liver safety monitoring was included to ensure the safety and to determine the hepatotoxic potential of investigational drug. The description and use of the ABC-CFX scoring algorithm was added throughout the protocol. Changes were made throughout the protocol related to the optional testing to determine the extent of methylation of the FMR1 gene. Changes were made throughout the protocol to allow the possibility for eligible subjects from other studies of AFQ056. An instruction for subjects to avoid drinking grapefruit juice was added to exclusion criterion.

    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 sponsor decided to terminate the study, as the study treatment, AFQ056, failed to demonstrate efficacy in target population in two other clinical studies.
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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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