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    Clinical Trial Results:
    Evaluation of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia-Teleangectasia Patients

    Summary
    EudraCT number
    		 2010-022315-19
    Trial protocol
    		 IT  
    Global end of trial date
    31 Aug 2011

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

    Trial information

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    Trial identification
    Sponsor protocol code
    IEDAT-ERY01-2010
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01255358
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Erydel S.p.A.
    Sponsor organisation address
    Via Meucci, 3, Bresso (MI), Italy, 20091
    Public contact
    Clinical Trial Transparency Manager, Erydel S.p.A., +39 02 36504470, info@erydel.com
    Scientific contact
    Clinical Trial Transparency Manager, Erydel S.p.A., +39 02 36504470, info@erydel.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
    31 Aug 2011
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Aug 2011
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Aug 2011
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the improvement in central nervous system (CNS) symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with Ataxia-Teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).
    Protection of trial subjects
    This study was performed in accordance with the ethical principles that have their origin in the Declaration of Helsinki (52nd WMA General Assembly, Edinburgh, Scotland, October 2000 - Notes of Clarification on Paragraph 29 added by the World Medical Association (WMA) General Assembly, Washington 2002 and on Paragraph 30 added by the WMA General Assembly, Tokyo 2004) and that are consistent with International Conference on Harmonization (ICH)/GCP and applicable regulatory requirements.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    28 Feb 2011
    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
    Italy: 22
    Worldwide total number of subjects
    22
    EEA total number of subjects
    22
    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)
    15
    Adolescents (12-17 years)
    5
    Adults (18-64 years)
    2
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Twenty-two patients were enrolled: 18 patients completed the study while there were four dropouts/withdrawals during the study period.

    Pre-assignment
    Screening details
    		 A total of 26 patients were screened at the two centers involved in the study. Only 22 patients were enrolled in the study. Of the 26 patients screened, four were screening failures (three for low levels of CD4+ lymphocytes and one for concomitant disease disallowed by the protocol).

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    The study was non-blinded since IEDAT-ERY01-2010 was an open-label study.

    Arms
    Arm title
    		 EryDex
    Arm description
    		 Six-month monthly treatment with Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes). Patients received intravenous infusion of 50 ml of autologous erythrocytes engineered to contain dexamethasone sodium phosphate encapsulated into erythrocytes that was previously taken from the same patient.
    Arm type
    		 Experimental

    Investigational medicinal product name
    EryDex
    Investigational medicinal product code
    Other name
    dexamethasone sodium phosphate
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Dexamethasone sodium phosphate encapsulated in human erythrocytes (Er-Dex): two vials of 250 mg/10 ml each. Patients received intravenous infusion of dexamethasone sodium phosphate (final amount about 10-15 mg) ex vivo encapsulated into autologous erythrocytes (2 vials of 250 mg each of dexamethasone sodium phosphate were used in the ex vivo process together with 50 ml of blood). The treatment was repeated at intervals of 30 days (±10 days), for a total of 6 infusions.

    Number of subjects in period 1
    		EryDex
    Started
    22
    Completed
    18
    Not completed
    4
         Consent withdrawn by subject
    1
         Adverse event, non-fatal
    2
         Protocol deviation
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    EryDex
    Reporting group description
    		 Six-month monthly treatment with Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes). Patients received intravenous infusion of 50 ml of autologous erythrocytes engineered to contain dexamethasone sodium phosphate encapsulated into erythrocytes that was previously taken from the same patient.

    Reporting group values
    		 EryDex Total
    Number of subjects
    		 22 22
    Age categorical
    Units: Subjects
        Children (2-11 years)
    		 15 15
        Adolescents (12-17 years)
    		 5 5
        Adults (18-64 years)
    		 2 2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 11.2 ( 3.5 ) -
    Gender categorical
    Units: Subjects
        Female
    		 11 11
        Male
    		 11 11
    Subject analysis sets

    Subject analysis set title
    EryDex - ITT population
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex.

    Subject analysis set title
    EryDex - PP population
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    The PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation].

    Subject analysis set title
    Visit 1 (Baseline) population - ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex

    Subject analysis set title
    Visit 1 (baseline) population - PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation].

    Subject analysis set title
    Visit 4 population - ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex

    Subject analysis set title
    Visit 7 population - ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex

    Subject analysis set title
    Visit 4 population - PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation]

    Subject analysis set title
    Visit 7 population - PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation].

    Subject analysis set title
    Screening population
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population includes all 22 patients enrolled into the study.

    Subject analysis sets values
    		 EryDex - ITT population EryDex - PP population Visit 1 (Baseline) population - ITT Visit 1 (baseline) population - PP Visit 4 population - ITT Visit 7 population - ITT Visit 4 population - PP Visit 7 population - PP Screening population
    Number of subjects
    22
    18
    22
    18
    19
    22
    18
    18
    22
    Age categorical
    Units: Subjects
        Children (2-11 years)
    15
    12
    15
    12
        Adolescents (12-17 years)
    5
    4
    5
    4
        Adults (18-64 years)
    2
    2
    2
    2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    11.2 ( 3.5 )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    Gender categorical
    Units: Subjects
        Female
    11
    8
        Male
    11
    10

    End points

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    End points reporting groups
    Reporting group title
    EryDex
    Reporting group description
    		 Six-month monthly treatment with Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes). Patients received intravenous infusion of 50 ml of autologous erythrocytes engineered to contain dexamethasone sodium phosphate encapsulated into erythrocytes that was previously taken from the same patient.

    Subject analysis set title
    EryDex - ITT population
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex.

    Subject analysis set title
    EryDex - PP population
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    The PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation].

    Subject analysis set title
    Visit 1 (Baseline) population - ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex

    Subject analysis set title
    Visit 1 (baseline) population - PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation].

    Subject analysis set title
    Visit 4 population - ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex

    Subject analysis set title
    Visit 7 population - ITT
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population included all patients enrolled into the study, who received at least one infusion of EryDex

    Subject analysis set title
    Visit 4 population - PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation]

    Subject analysis set title
    Visit 7 population - PP
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    PP Population included patients who were treated according to protocol and fulfilled the following criteria: • compliance with all entry criteria • absence of major protocol violations • adequate compliance with trial medication [at most one missed or untimely (outside the allowable ±10 days time-window) infusion of the study preparation].

    Subject analysis set title
    Screening population
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The ITT Population includes all 22 patients enrolled into the study.

    Primary: Change from baseline in mean ICARS Total score by visit

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    End point title
    		 Change from baseline in mean ICARS Total score by visit
    End point description
    Changes in neurological symptoms were assessed using the International Co-operative Ataxia Rating Scale (ICARS), which is a semiquantitative scale offering a compartmentalized quantification of the following 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6), for a possible total score of 100 points (higher score corresponds to worse status) Changes in ICARS scores were calculated for each patient by subtracting the score at V1 from the score at each of the follow-up visits.
    End point type
    Primary
    End point timeframe
    At Visit 2 (Day 30), 4 (Day 90), 7 (Day 180)
    End point values
    		 EryDex - ITT population EryDex - PP population Visit 1 (Baseline) population - ITT Visit 1 (baseline) population - PP
    Number of subjects analysed
    22 [1]
    18
    22
    18
    Units: score
    arithmetic mean (standard deviation)
        Visit 2
    -2.2 ( 5.6 )
    -2.4 ( 4.9 )
    000 ( 000 )
    000 ( 000 )
        Visit 4
    -3.7 ( 7.3 )
    -3.9 ( 7.1 )
    000 ( 000 )
    000 ( 000 )
        Visit 7
    -4.0 ( 7.5 )
    -5.2 ( 7.0 )
    000 ( 000 )
    000 ( 000 )
    Notes
    [1] - At Visit 4 N=19
    Statistical analysis title
    		 Visit 2 vs Visit 1 (baseline)
    Comparison groups
    EryDex - ITT population v Visit 1 (Baseline) population - ITT
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    		 other [2]
    P-value
    		 = 0.107
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [2] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. Changes over time in the ICARS Total score were analyzed with a Repeated Measures Analysis of Variance (RMANOVA) with possible covariates (e.g. age and ICARS at baseline). A Wilcoxon non-parametric test evaluating changes between visits was performed comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 4 vs Visit 1 (baseline)
    Comparison groups
    EryDex - ITT population v Visit 1 (Baseline) population - ITT
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    		 other [3]
    P-value
    		 = 0.054
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [3] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. Changes over time in the ICARS Total score were analyzed with a Repeated Measures Analysis of Variance (RMANOVA) with possible covariates (e.g. age and ICARS at baseline). A Wilcoxon non-parametric test evaluating changes between visits was performed since the overall analysis was significant comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 7 vs Visit 1 (baseline)
    Comparison groups
    EryDex - ITT population v Visit 1 (Baseline) population - ITT
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    		 other [4]
    P-value
    		 = 0.024
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [4] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. Changes over time in the ICARS Total score were analyzed with a Repeated Measures Analysis of Variance (RMANOVA) with possible covariates (e.g. age and ICARS at baseline). A Wilcoxon non-parametric test evaluating changes between visits was performed since the overall analysis was significant comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 2 vs Visit 1 (baseline)
    Comparison groups
    EryDex - PP population v Visit 1 (baseline) population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [5]
    P-value
    		 = 0.059
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [5] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. Changes over time in the ICARS Total score were analyzed with a Repeated Measures Analysis of Variance (RMANOVA) with possible covariates (e.g. age and ICARS at baseline). A Wilcoxon non-parametric test evaluating changes between visits was performed since the overall analysis was significant comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 4 vs Visit 1 (baseline)
    Comparison groups
    EryDex - PP population v Visit 1 (baseline) population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [6]
    P-value
    		 = 0.032
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [6] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. Changes over time in the ICARS Total score were analyzed with a Repeated Measures Analysis of Variance (RMANOVA) with possible covariates (e.g. age and ICARS at baseline). A Wilcoxon non-parametric test evaluating changes between visits was performed since the overall analysis was significant comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 7 vs Visit 1 (baseline)
    Comparison groups
    EryDex - PP population v Visit 1 (baseline) population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [7]
    P-value
    		 = 0.01
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [7] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. Changes over time in the ICARS Total score were analyzed with a Repeated Measures Analysis of Variance (RMANOVA) with possible covariates (e.g. age and ICARS at baseline). A Wilcoxon non-parametric test evaluating changes between visits was performed since the overall analysis was significant comparing V4 and V7 compared to baseline (V1).

    Secondary: Mean Investigator Global Assessment (IGA) by visit

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    End point title
    		 Mean Investigator Global Assessment (IGA) by visit
    End point description
    The IGA consisted of an evaluation of neurological signs and symptoms defined as neuromotor disorders, posture and deambulation, disturbances of the oro-pharyngeal apparatus, presence of peripheral neuropathy, neurodevelopmental disturbances, intellectual level, and behavior disturbances, which was based only on a subjective judgment of the Investigator. A 5 point qualitative scale with rating categories of “very much improved (1), slightly improved (2) no change (3), slightly worsened (4), and very much worsened (5)” was used to assess changes at V4 and V7, compared to the patient’s condition at baseline.
    End point type
    Secondary
    End point timeframe
    At Visit 4 and Visit 7
    End point values
    		 Visit 4 population - ITT Visit 7 population - ITT Visit 4 population - PP Visit 7 population - PP
    Number of subjects analysed
    19
    22
    18
    18
    Units: score
        arithmetic mean (standard deviation)
    2.2 ( 0.9 )
    2.4 ( 0.8 )
    2.0 ( 0.8 )
    2.2 ( 0.7 )
    Statistical analysis title
    		 Visit 7 vs Visit 4
    Comparison groups
    Visit 4 population - ITT v Visit 7 population - ITT
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 other [8]
    P-value
    		 = 0.44
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [8] - This is a within-arm comparison: groups examined should not be added. N=41 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test has been conducted on both ITT and PP Populations by comparing V4 vs. V7 scores.
    Statistical analysis title
    		 Visit 7 vs Visit 4
    Comparison groups
    Visit 4 population - PP v Visit 7 population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [9]
    P-value
    		 = 0.157
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [9] - This is a within-arm comparison: groups examined should not be added. N=36 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test has been conducted on both ITT and PP Populations by comparing V4 vs. V7 scores.

    Secondary: Ocular motility by visit

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    End point title
    		 Ocular motility by visit
    End point description
    Changes vs. baseline were computed through a 5-point qualitative scale as: “very much improved, slightly improved, no change, slightly worsened, very much worsened” (The Investigator will state if the condition was very much improved, slightly improved, exhibited no change, was slightly worse or very much worse).
    End point type
    Secondary
    End point timeframe
    At Visit 4 and at Visit 7
    End point values
    		 Visit 4 population - ITT Visit 7 population - ITT Visit 4 population - PP Visit 7 population - PP
    Number of subjects analysed
    19
    22
    18
    18
    Units: score
        arithmetic mean (standard deviation)
    2.6 ( 0.7 )
    2.4 ( 0.7 )
    2.6 ( 0.7 )
    2.2 ( 0.7 )
    Statistical analysis title
    		 Visit 7 vs Visit 4
    Comparison groups
    Visit 4 population - ITT v Visit 7 population - ITT
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 other [10]
    P-value
    		 = 0.014
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [10] - This is a within-arm comparison: groups examined should not be added. N=41 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test has been conducted on both ITT and PP Populations by comparing V4 vs. V7 scores.
    Statistical analysis title
    		 Visit 7 vs Visit 4
    Comparison groups
    Visit 4 population - PP v Visit 7 population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [11]
    P-value
    		 = 0.014
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [11] - This is a within-arm comparison: groups examined should not be added. N=36 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test has been conducted on both ITT and PP Populations by comparing V4 vs. V7 scores.

    Secondary: Change from baseline in Vineland Adaptive Behaviour Scale (VABS) by visit

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    End point title
    		 Change from baseline in Vineland Adaptive Behaviour Scale (VABS) by visit
    End point description
    The VABS complete form consists of 540 items, 261 of which were taken from the short form, and is administered by a semistructured interview by a trained interviewer with a parent or operator who takes care of the patient. The adaptive behaviour is measured according to four scales, each subdivided into eleven subscales. Each subscale is further divided into clusters (2 to 8 items) listed in evolutionary order; each cluster is sorted to a target item. A score can be assigned to the items: this score is intended to indicate whether the subjects perform that activity “usually”, “sometimes” or “never”; answers as “no chance” or “I do not know” are also provided. Scales and Subscales. VABS are divided into four scales and eleven subscales: 1) Communication: Receptive, Expressive, Written; 2) Daily Living Skills: Personal, Domestic, Community; 3) Socialization: Interpersonal Relationships, Play & Leisure Time, Coping Skills; 4) Motor Skills: Gross, Fine.
    End point type
    Secondary
    End point timeframe
    At Visit 4 (Day 90) and Visit 7 (Day 180).
    End point values
    		 EryDex - ITT population EryDex - PP population Visit 1 (Baseline) population - ITT Visit 1 (baseline) population - PP
    Number of subjects analysed
    22 [12]
    18
    22
    18
    Units: score
    arithmetic mean (standard deviation)
        Visit 4
    1.0 ( 0.9 )
    1.1 ( 0.9 )
    000 ( 000 )
    000 ( 000 )
        Visit 7
    1.3 ( 1.2 )
    1.5 ( 1.1 )
    000 ( 000 )
    000 ( 000 )
    Notes
    [12] - At visit 4 N=19
    Statistical analysis title
    		 Visit 4 vs Visit 1 (baseline)
    Comparison groups
    EryDex - ITT population v Visit 1 (Baseline) population - ITT
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    		 other [13]
    P-value
    		 < 0.0001
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [13] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test was performed comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 7 vs Visit 1 (baseline)
    Comparison groups
    Visit 1 (Baseline) population - ITT v EryDex - ITT population
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    		 other [14]
    P-value
    		 < 0.0001
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [14] - This is a within-arm comparison: groups examined should not be added. N=44 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test was performed comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 4 vs Visit 1 (baseline)
    Comparison groups
    EryDex - PP population v Visit 1 (baseline) population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [15]
    P-value
    		 < 0.0001
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [15] - This is a within-arm comparison: groups examined should not be added. N=36 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test was performed comparing V4 and V7 compared to baseline (V1).
    Statistical analysis title
    		 Visit 7 vs Visit 1 (baseline)
    Comparison groups
    EryDex - PP population v Visit 1 (baseline) population - PP
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 other [16]
    P-value
    		 < 0.0001
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Notes
    [16] - This is a within-arm comparison: groups examined should not be added. N=36 is an innate error of the EudraCT database system. A Wilcoxon non-parametric test was performed comparing V4 and V7 compared to baseline (V1).

    Secondary: Hematology values by visit

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    End point title
    		 Hematology values by visit
    End point description
    Absolute values are reported.
    End point type
    Secondary
    End point timeframe
    At screening and at Visit 7
    End point values
    		 Visit 7 population - ITT Screening population
    Number of subjects analysed
    22
    22
    Units: units
    arithmetic mean (standard deviation)
        hemoglobin (g/dl)
    12.8 ( 1.0 )
    13.5 ( 0.9 )
        hematocrit (%)
    39.0 ( 3.0 )
    41.0 ( 2.6 )
        RBC (x10^12/L)
    4.6 ( 0.4 )
    4.9 ( 0.4 )
        WBC (x10^9/L)
    5.7 ( 1.6 )
    6.5 ( 2.3 )
        neutrophils (x10^9/l)
    3.3 ( 1.3 )
    4.0 ( 2.1 )
        lymphocytes (x10^9/l)
    1.4 ( 0.4 )
    1.5 ( 0.6 )
        monocytes (x10^9/l)
    0.6 ( 0.2 )
    0.7 ( 0.2 )
        eosinophils (x10^9/l)
    0.3 ( 0.2 )
    0.2 ( 0.1 )
        basophils (x10^9/l)
    0.04 ( 0.02 )
    0.05 ( 0.01 )
        platelets (x10^9/l)
    341.2 ( 55.2 )
    373.9 ( 75.8 )
    No statistical analyses for this end point

    Secondary: Blood chemistry values by visit

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    End point title
    		 Blood chemistry values by visit
    End point description
    Absolute values are reported.
    End point type
    Secondary
    End point timeframe
    At screening and at Visit 7
    End point values
    		 Visit 7 population - ITT Screening population
    Number of subjects analysed
    22
    22
    Units: units
    arithmetic mean (standard deviation)
        blood glucose (mg/dL)
    83.7 ( 6.2 )
    86.6 ( 6.9 )
        BUN (mmol/L)
    4.3 ( 1.3 )
    4.40 ( 1.1 )
        GOT/AST (U/L)
    29.5 ( 9.3 )
    31.2 ( 10.5 )
        GPT/ALT (U/L)
    30.9 ( 20.6 )
    32.8 ( 18.6 )
        alkaline phosphatase (U/L)
    251.6 ( 85.9 )
    279.4 ( 104.1 )
        total proteins (g/dL)
    6.6 ( 0.4 )
    7.0 ( 0.4 )
        albumin (g/dL)
    4.0 ( 0.2 )
    4.30 ( 0.3 )
        creatinine (mg/dL)
    0.4 ( 0.1 )
    0.4 ( 0.1 )
        bilirubin (mg/dL)
    0.4 ( 0.3 )
    0.4 ( 0.2 )
        serum iron (μg/dL)
    66.2 ( 30.5 )
    82.9 ( 41.2 )
        sodium (mmol/L)
    141.4 ( 1.4 )
    142.3 ( 2.5 )
        potassium (mmol/L)
    4.5 ( 0.4 )
    4.7 ( 0.3 )
        calcium (mmol/L)
    2.4 ( 0.1 )
    2.5 ( 0.1 )
        phosphorus (mg/dL)
    5.1 ( 0.3 )
    5.1 ( 0.5 )
        chloride (mol/L)
    104.0 ( 1.8 )
    104.6 ( 2.5 )
    No statistical analyses for this end point

    Secondary: Urine analysis by visit

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    End point title
    		 Urine analysis by visit
    End point description
    Absolute values are reported.
    End point type
    Secondary
    End point timeframe
    at screening and at Visit 7
    End point values
    		 Visit 7 population - ITT Screening population
    Number of subjects analysed
    22
    22
    Units: units
    arithmetic mean (standard deviation)
        specific gravity
    1022.4 ( 7.3 )
    1024.8 ( 5.9 )
        pH
    5.6 ( 0.7 )
    5.8 ( 0.6 )
        glucose (mg/dL)
    0.0 ( 0.0 )
    0.0 ( 0.0 )
        protein (mg/dL)
    6.8 ( 11.1 )
    11.5 ( 9.2 )
        hemoglobin (mg/dL)
    0.0 ( 0.0 )
    0.0 ( 0.0 )
        ketones (mg/dL)
    0.5 ( 2.1 )
    0.0 ( 0.0 )
        bilirubin (mg/dL)
    0.0 ( 0.0 )
    0.0 ( 0.0 )
    No statistical analyses for this end point

    Secondary: Special laboratory analysis by visit

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    End point title
    		 Special laboratory analysis by visit
    End point description
    Absolute values are reported.
    End point type
    Secondary
    End point timeframe
    at Visit 1 and at Visit 7
    End point values
    		 Visit 1 (Baseline) population - ITT Visit 7 population - ITT
    Number of subjects analysed
    22
    22
    Units: units
    arithmetic mean (standard deviation)
        total cholesterol (mmol/L)
    4.3 ( 0.7 )
    4.3 ( 0.8 )
        HDL cholesterol (mmol/L
    1.2 ( 0.3 )
    1.3 ( 0.3 )
        LDL cholesterol (mmol/L)
    2.7 ( 0.6 )
    2.6 ( 0.7 )
        HbA1c (%)
    5.2 ( 0.3 )
    5.3 ( 0.3 )
        CD4+ lymphocytes (count/mm3)
    379.7 ( 152.0 )
    431.5 ( 147.2 )
        a-fetoprotein (μg/L)
    226.1 ( 161.8 )
    234.6 ( 180.7 )
        blood cortisol (μg/dL)
    15.9 ( 7.7 )
    13.8 ( 6.4 )
        urinary cortisol (μg/24h)
    39.9 ( 17.5 )
    29.4 ( 20.3 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    At screening visit (day -30 --> 0), V1 (day 0), V2 (day 30), V3 (day 60), V4 (day 90), V5 (day 120), V6 (day 150), V7 (day 180).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    14.0
    Reporting groups
    Reporting group title
    EryDex - Safety population
    Reporting group description
    		 The safety population includes the 22 patients who have received at least one dose of planned trial treatment.

    Serious adverse events
    		 EryDex - Safety population
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 22 (9.09%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Respiratory, thoracic and mediastinal disorders
    Bronchiectasis
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Bronchopneumonia
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 4.5%
    Non-serious adverse events
    		 EryDex - Safety population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 22 (68.18%)
    Investigations
    Low serum iron levels
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences all number
    1
    Vascular disorders
    Diastolic hypertension
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences all number
    1
    General disorders and administration site conditions
    Fever
         subjects affected / exposed
    3 / 22 (13.64%)
         occurrences all number
    3
    Immune system disorders
    Low serum CD4+ lymphocytes levels
         subjects affected / exposed
    2 / 22 (9.09%)
         occurrences all number
    2
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 22 (13.64%)
         occurrences all number
    3
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences all number
    1
    Bronchitis
         subjects affected / exposed
    2 / 22 (9.09%)
         occurrences all number
    3
    Otitis
         subjects affected / exposed
    3 / 22 (13.64%)
         occurrences all number
    4
    Urinary infection
         subjects affected / exposed
    2 / 22 (9.09%)
         occurrences all number
    2
    Herpes labialis
         subjects affected / exposed
    2 / 22 (9.09%)
         occurrences all number
    2
    Flu syndrome
         subjects affected / exposed
    3 / 22 (13.64%)
         occurrences all number
    3
    Gastroenteritis
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Hypercholesterolemia
         subjects affected / exposed
    1 / 22 (4.55%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Jul 2011
    During the study, there was an Amendment approved by the Ethic Committe of the Coordinating Centre on 11 July 2011. This amendment instituted the following procedure: only for the patients from the Department of Infantile Pediatrics and Neuro-Psychiatry - University La Sapienza, Rome, an additional 10 mL of blood was to be collected at the final visit (V7) to define the expression of mRNAs from several gene products, and to evaluate the cytokines and chemokines levels, the lymphocyte sub-populations and the RBC morphology, stability and biochemical properties.

    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.
    No limitations or caveats are applicable to this summary of results.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/24405665
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