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    Clinical Trial Results:
    A phase II, multicenter, open-label, randomized two-year study to evaluate the efficacy and safety of deferasirox film-coated tablet versus phlebotomy in patients with Hereditary Hemochromatosis.

    Summary
    EudraCT number
    		 2016-002529-12
    Trial protocol
    		 ES   SK   DE   BE   RO  
    Global end of trial date
    17 Apr 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Apr 2024
    First version publication date
    27 Apr 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CICL670F2203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03203850
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharmaceuticals  
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, novartis.email@novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, novartis.email@novartis.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
    17 Apr 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Apr 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the trial was to assess the response rate in the deferasirox FCT and phlebotomy treatment arms where response is defined by achieving target serum ferritin (SF) ≤ 100 μg/L on or before 24 months.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    11 Jan 2018
    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
    Romania: 1
    Country: Number of subjects enrolled
    Belgium: 5
    Country: Number of subjects enrolled
    Spain: 22
    Country: Number of subjects enrolled
    Switzerland: 2
    Country: Number of subjects enrolled
    Russian Federation: 8
    Country: Number of subjects enrolled
    Slovakia: 2
    Country: Number of subjects enrolled
    France: 5
    Worldwide total number of subjects
    45
    EEA total number of subjects
    35
    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)
    43
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in 11 investigative sites in 7 countries.

    Pre-assignment
    Screening details
    		 There was a screening period of 4 weeks to assess participants eligibility.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Deferasirox FCT 7mg/kg
    Arm description
    		 Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks)
    Arm type
    		 Experimental

    Investigational medicinal product name
    Deferasirox film-coated tablet
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks)

    Arm title
    		 Phlebotomy
    Arm description
    		 Phlebotomy - standard of care
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Deferasirox FCT 7mg/kg Phlebotomy
    Started
    30
    15
    Completed
    22
    12
    Not completed
    8
    3
         Adverse event, serious fatal
    1
    -
         Adverse events
    3
    -
         Subject/guardian decision
    4
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Deferasirox FCT 7mg/kg
    Reporting group description
    		 Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks)

    Reporting group title
    Phlebotomy
    Reporting group description
    		 Phlebotomy - standard of care

    Reporting group values
    		 Deferasirox FCT 7mg/kg Phlebotomy Total
    Number of subjects
    		 30 15 45
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 29 14 43
        From 65-84 years
    		 1 1 2
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 51.6 ( 8.20 ) 52.1 ( 8.13 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 4 3 7
        Male
    		 26 12 38
    Race/Ethnicity, Customized
    Units: Subjects
        Caucasian
    		 30 15 45

    End points

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    End points reporting groups
    Reporting group title
    Deferasirox FCT 7mg/kg
    Reporting group description
    		 Deferasirox film-coated tablet 7mg/kg, oral dose daily (starting dose for the first 12 weeks)

    Reporting group title
    Phlebotomy
    Reporting group description
    		 Phlebotomy - standard of care

    Primary: Proportion of patients achieving target SF ≤ 100 μg/L for the first time

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    End point title
    		 Proportion of patients achieving target SF ≤ 100 μg/L for the first time [1]
    End point description
    Proportion of participants achieving target serum ferritin (SF) ≤ 100 μg/L on or before Month 24. Participants were considered responders if they met response criteria (target SF ≤100 µg/L) on or before Month 24 (Week 104) during the treatment phase. Any participant who discontinued treatment prematurely before meeting such criterion and participants with unknown or missing SF by Month 24 were counted as non-responder.
    End point type
    Primary
    End point timeframe
    Up to Month 24
    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 analyzed descriptively.
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: percentage of participants
    number (confidence interval 95%)
        Responder
    40 (22.7 to 59.4)
    80 (51.9 to 95.7)
        Non-Responder
    60 (40.6 to 77.3)
    20 (4.3 to 48.1)
    No statistical analyses for this end point

    Secondary: Number of participants with ocular treatment emergent adverse events (AEs)

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    End point title
    		 Number of participants with ocular treatment emergent adverse events (AEs)
    End point description
    Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline).
    End point type
    Secondary
    End point timeframe
    Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks.
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        At least one ocular AE
    9
    0
        Treatment-related ocular AEs
    2
    0
    No statistical analyses for this end point

    Secondary: Number of participants with ocular treatment emergent adverse events (AEs) by preferred term

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    End point title
    		 Number of participants with ocular treatment emergent adverse events (AEs) by preferred term
    End point description
    Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). Preferred terms are based on Medical Dictionary of Regulatory Activities (MedDRA) version 26.0.
    End point type
    Secondary
    End point timeframe
    Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks.
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        Cataract nuclear
    2
    0
        Glaucoma
    2
    0
        Blepharitis
    1
    0
        Cellulitis orbital
    1
    0
        Dry eye
    1
    0
        Eye pain
    1
    0
        Eye ulcer
    1
    0
        Macular oedema
    1
    0
        Open angle glaucoma
    1
    0
        Optic nerve disorder
    1
    0
        Panophthalmitis
    1
    0
        Retinal degeneration
    1
    0
        Retinal haemorrhage
    1
    0
        Visual acuity reduced
    1
    0
        Vitreous haemorrhage
    1
    0
    No statistical analyses for this end point

    Secondary: Number of participants with treatment emergent adverse events (AEs) and serious adverse events (SAEs)

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    End point title
    		 Number of participants with treatment emergent adverse events (AEs) and serious adverse events (SAEs)
    End point description
    Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs leading to study treatment discontinuation, SAEs and SAEs leading to study treatment discontinuation.
    End point type
    Secondary
    End point timeframe
    Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks.
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        At least one AE
    28
    12
        At least one SAE
    7
    0
        AEs leading to discontinuation
    3
    0
        SAEs leading to discontinuation
    0
    0
    No statistical analyses for this end point

    Secondary: Number of adverse events in participants who had study treatment interrupted due to SF ≤ 100 μg/L and re-initiated study treatment when ≥ 300 μg/L

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    End point title
    		 Number of adverse events in participants who had study treatment interrupted due to SF ≤ 100 μg/L and re-initiated study treatment when ≥ 300 μg/L
    End point description
    Number of participants who interrupt deferasirox FCT at least once due to SF level ≤ 100 μg/L and re-initiate therapy at SF level ≥ 300 μg/L. There were no participants that re-initiated therapy when reached 300 ug/L.
    End point type
    Secondary
    End point timeframe
    Up to 24 months
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: participants
    Notes
    [2] - There were no participants that re-initiated therapy when reached 300 ug/L.
    [3] - There were no participants that re-initiated therapy when reached 300 ug/L.
    No statistical analyses for this end point

    Secondary: Categorical analysis of logMAR score changes from baseline to best/worst post-baseline changes in one eye with more extreme change

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    End point title
    		 Categorical analysis of logMAR score changes from baseline to best/worst post-baseline changes in one eye with more extreme change
    End point description
    Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Increase in logMAR score from baseline indicates worsening in visual acuity. Decrease in logMAR score category from baseline indicates improvement in visual acuity.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 104
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        Best change: Decrease <0.1
    9
    4
        Best change: Decrease >=0.1 - <0.2
    9
    5
        Best change: Decrease >=0.2 - <0.3
    1
    0
        Best change: Decrease >=0.3 - <0.6
    0
    0
        Best change: Decrease >=0.6
    1
    0
        Best change: Decrease Missing baseline assessment
    2
    0
        Best change: Decrease No decrease from baseline
    8
    6
        Worst change: Increase <0.1
    11
    11
        Worst change: Increase >=0.1 - <0.2
    10
    2
        Worst change: Increase >=0.2 - <0.3
    1
    2
        Worst change: Increase >=0.3 - <0.6
    3
    0
        Worst change: Increase >=0.6
    0
    0
        Worst change: Increase Missing baseline assessment
    2
    0
        Worst change: Increase No increase from baseline
    3
    0
    No statistical analyses for this end point

    Secondary: Categorical analysis of worst post-baseline values of intraocular pressure in one eye with more extreme change

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    End point title
    		 Categorical analysis of worst post-baseline values of intraocular pressure in one eye with more extreme change
    End point description
    Intraocular pressure was measured by tonometry. Intraocular pressure values >5 to ≤21 mmHg were considered normal.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 104
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        Worst post-baseline value- <=5 mmHg
    0
    0
        Worst post-baseline value- >5 to <=21 mmHg
    27
    12
        Worst post-baseline value- >21 to <=30 mmHg
    1
    3
        Worst post-baseline value- >30 mmHg
    0
    0
        missing post-baseline assessment
    2
    0
    No statistical analyses for this end point

    Secondary: Categorical analysis of changes in intraocular pressure from baseline to best/worst post-baseline changes in one eye with more extreme change

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    End point title
    		 Categorical analysis of changes in intraocular pressure from baseline to best/worst post-baseline changes in one eye with more extreme change
    End point description
    Intraocular pressure was measured by tonometry. A decrease in intraocular pressure from baseline indicated improvement.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 104
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        Increase from baseline >=5 mmHg and <10 mmHg
    4
    2
        Increase from baseline >=10 mmHg
    0
    1
        Decrease from baseline >=5 mmHg and <10 mmHg
    6
    3
        Decrease from baseline >=10 mmHg
    1
    0
        No change from baseline or min. change (>5 mmHg)
    17
    9
        Missing post-baseline values
    2
    0
    No statistical analyses for this end point

    Secondary: Number of participants with slit lamp results for any evaluation and worst eye

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    End point title
    		 Number of participants with slit lamp results for any evaluation and worst eye
    End point description
    Slit lamp examination was used to evaluate lids, cornea, conjunctiva, iris, anterior chamber, aqueous flare, aqueous inflammatory cells and lens. Any post-baseline abnormalities (not present at baseline) in slit lamp examination were assesses by the investigator and classified as insignificant or clinically significant. Number of participants with slit lamp results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 104
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        Baseline|Normal
    12
    6
        Any post-baseline|Normal
    7
    3
        Baseline|Insignificant
    14
    9
        Any post-baseline|Insignificant
    18
    12
        Baseline|Significant
    2
    0
        Any post-baseline|Significant
    3
    0
        Baseline|Missing
    2
    0
        Any post-baseline|Missing
    2
    0
    No statistical analyses for this end point

    Secondary: Number of participants with fundus oculi results for any evaluation and worst eye

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    End point title
    		 Number of participants with fundus oculi results for any evaluation and worst eye
    End point description
    Fundus oculi examination was used to evaluate peripheral retina, macula, optic nerve, and vitreous hemorrhage. Any post-baseline abnormalities (not present at baseline) in fundus oculi examination were assessed by the investigator and classified as insignificant or clinically significant. Number of participants with fundus oculi results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 104
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        Baseline|Normal
    15
    10
        Any post-baseline|Normal
    12
    10
        Baseline|Insignificant
    12
    5
        Any post-baseline|Insignificant
    14
    5
        Baseline|Significant
    1
    0
        Any post-baseline|Significant
    2
    0
        Baseline|Missing
    2
    0
        Any post-baseline|Missing
    2
    0
    No statistical analyses for this end point

    Secondary: Number of participants with an increase from baseline of ≥1 and ≥2 in LOCS III grades

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    End point title
    		 Number of participants with an increase from baseline of ≥1 and ≥2 in LOCS III grades
    End point description
    Lens Opacities Classification System III (LOCS III) grading scales include lens opacities defined as nuclear opalescence (NO), nuclear color (NC), cortical (C) cataract and posterior subcapsular (P) cataract with several degrees of extend, i.e. severity. The LOCS III scale for nuclear opalescence and for nuclear color ranges from 0 to 6. The LOCS III scale for cortical cataract and posterior subcapsular cataract opacity ranges from 0 to 5. For all scales, higher values indicate higher opacity, opalescence, or color (range: NO0/NC0/C0/P0 to NO6/NC6/C5/P5). Number of participants with an increase from baseline of ≥1 and increase of ≥2 in LOCS III grades is reported.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 104
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: participants
        ≥1 grade increase from baseline
    10
    5
        ≥2 grade increase from baseline
    2
    0
    No statistical analyses for this end point

    Secondary: Time to response (TTR)

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    End point title
    		 Time to response (TTR)
    End point description
    Time to response (TTR) is defined as the time from the date of randomization to the date of the first time the SF achieved a value ≤ 100 μg/L during the treatment phase. Participants who did not achieve SF ≤ 100 μg/L were censored as follows: at the last serum ferritin assessment date on or before month 24 (week 104), at the day of randomization if a subject does not have any post-baseline serum ferritin value or at the death date. TTR was analyzed using the Kaplan-Meier method. Due to EudraCT system limitations, data fields in the table cannot contain letters (eg. NA indicating ‘not applicable’). Therefore, not applicable values are indicated as ‘999’.
    End point type
    Secondary
    End point timeframe
    Up to Month 24
    End point values
    		 Deferasirox FCT 7mg/kg Phlebotomy
    Number of subjects analysed
    30
    15
    Units: months
        median (confidence interval 95%)
    999 (19.4 to 999)
    13.6 (4.0 to 22.1)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    DFX FCT
    Reporting group description
    		 DFX FCT

    Reporting group title
    Phlebotomy
    Reporting group description
    		 Phlebotomy

    Serious adverse events
    		 DFX FCT Phlebotomy
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 30 (23.33%)
    0 / 15 (0.00%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Transaminases increased
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Bladder neoplasm
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Lower limb fracture
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Thoracic outlet syndrome
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Sudden death
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal impairment
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cellulitis
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Erysipelas
         subjects affected / exposed
    1 / 30 (3.33%)
    0 / 15 (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
    		 DFX FCT Phlebotomy
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    23 / 30 (76.67%)
    12 / 15 (80.00%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Hypertension
         subjects affected / exposed
    3 / 30 (10.00%)
    3 / 15 (20.00%)
         occurrences all number
    4
    3
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Fatigue
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Asthenia
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Vaccination site pain
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Rhinorrhoea
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Nasal congestion
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Cough
         subjects affected / exposed
    1 / 30 (3.33%)
    3 / 15 (20.00%)
         occurrences all number
    1
    3
    Catarrh
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Asthma
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Investigations
    Blood uric acid increased
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Blood urea increased
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Blood creatinine increased
         subjects affected / exposed
    10 / 30 (33.33%)
    1 / 15 (6.67%)
         occurrences all number
    13
    1
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Blood cholesterol increased
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Glycosylated haemoglobin increased
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Vitamin D decreased
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Road traffic accident
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Procedural pain
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Ligament sprain
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Cardiac disorders
    Sinus bradycardia
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Nervous system disorders
    Taste disorder
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Headache
         subjects affected / exposed
    4 / 30 (13.33%)
    2 / 15 (13.33%)
         occurrences all number
    8
    3
    Dizziness
         subjects affected / exposed
    0 / 30 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    4
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 30 (3.33%)
    2 / 15 (13.33%)
         occurrences all number
    1
    2
    Ear and labyrinth disorders
    Deafness unilateral
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Ear pain
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Phobic postural vertigo
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Middle ear inflammation
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Eye disorders
    Glaucoma
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Cataract nuclear
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Abdominal distension
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Gastrointestinal disorder
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Diarrhoea
         subjects affected / exposed
    6 / 30 (20.00%)
    2 / 15 (13.33%)
         occurrences all number
    8
    3
    Constipation
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Nausea
         subjects affected / exposed
    5 / 30 (16.67%)
    0 / 15 (0.00%)
         occurrences all number
    8
    0
    Haemorrhoids
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Gastrointestinal pain
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Vomiting
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 30 (3.33%)
    2 / 15 (13.33%)
         occurrences all number
    1
    3
    Arthritis
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Back pain
         subjects affected / exposed
    4 / 30 (13.33%)
    2 / 15 (13.33%)
         occurrences all number
    4
    2
    Neck pain
         subjects affected / exposed
    0 / 30 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    2
    Infections and infestations
    Wound infection
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Urinary tract infection
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Suspected COVID-19
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Sinusitis
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Nasopharyngitis
         subjects affected / exposed
    2 / 30 (6.67%)
    3 / 15 (20.00%)
         occurrences all number
    2
    5
    Escherichia urinary tract infection
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Ear infection
         subjects affected / exposed
    1 / 30 (3.33%)
    1 / 15 (6.67%)
         occurrences all number
    1
    1
    Cystitis
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    COVID-19
         subjects affected / exposed
    2 / 30 (6.67%)
    0 / 15 (0.00%)
         occurrences all number
    2
    0
    Bone abscess
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Folate deficiency
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Hyperglycaemia
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    Vitamin B12 deficiency
         subjects affected / exposed
    0 / 30 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    30 Aug 2018
    The purpose of this amendment is to modify the inclusion and exclusion criteria, to correct inconsistencies, typos, add some clarifications and to update withdrawal of consent language. Additionally, the local French amendment text is formally integrated in this global amendment. However, the French specific requirements remain valid for France only.

    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
    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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