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    Clinical Trial Results:
    Effects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s Ataxia

    Summary
    EudraCT number
    		 2008-000040-13
    Trial protocol
    		 AT  
    Global end of trial date
    01 Aug 2012

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Feb 2022
    First version publication date
    16 Feb 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    21011075118
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Medical University Innsbruck
    Sponsor organisation address
    Christoph-Probst-Platz 1, Innrain 52, Innsbruck, Austria, 6020
    Public contact
    Priv.Doz. Dr. Sylvia Bösch, University Hospital for Neurology, Anichstrasse 35, 6020 Innsbruck, +43 (0)512-504-26285, sylvia.boesch@tirol-kliniken.at
    Scientific contact
    Priv.Doz. Dr. Sylvia Bösch, University Hospital for Neurology, Anichstrasse 35, 6020 Innsbruck, +43 (0)512-504-26285, sylvia.boesch@tirol-kliniken.at
    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
    01 Aug 2012
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Aug 2012
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Aug 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Aims of the proposed study 1) to investigate morphological and biochemical parameters of skeletal mus-cle systematically, to identify hematopoietic progenitor cells (CD34, CD133) as well as to measure baseline Frataxin expression in skeletal muscle (specimen) of FRDA patients. 2) to measure numbers of circulating CD34 and CD133 hematopoietic progenitor cells in peripheral blood of FRDA patients in response to rhuEPO treatment; 3) to study effects of rhuEPO treatment on Frataxin expression, numbers of CD34 and CD133 hematopoietic progenitor cells, satellite cells, capillary density and respiratory chain complex activi-ties of skeletal muscle tissue obtained by re-biopsy. 4) to identify changes in muscle energy metabolism (in-vivo marker) in skeletal muscle of rhuEPO treated FRDA patients applying magnetic resonance spectros-copy at baseline and study endpoint.
    Protection of trial subjects
    Safety was assessed by red blood cell count and blood pressure in two weekly intervals.
    Background therapy
    		 -
    Evidence for comparator
    		 There was no evidence for a comparator.
    Actual start date of recruitment
    11 Feb 2009
    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
    Austria: 15
    Worldwide total number of subjects
    15
    EEA total number of subjects
    15
    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)
    15
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 All patients were recruited from the ataxia outpatient clinic of the Department of Neurology (Medical University Innsbruck).

    Pre-assignment
    Screening details
    		 Screening visits to check for inclusion and exclusion criteria were carried out in February and March 2009 after final trial registration.

    Period 1
    Period 1 title
    Treatment (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    This trial was not blinded.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 rhuEPO
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Neorecormon
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    FRDA patients received 3,000 international units (IU) rhuEPO (Roche, Switzerland) thrice weekly over a study period of 8 weeks.

    Arm title
    		 Control
    Arm description
    		 -
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		rhuEPO Control
    Started
    7
    8
    Completed
    7
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    rhuEPO
    Reporting group description
    		 -

    Reporting group title
    Control
    Reporting group description
    		 -

    Reporting group values
    		 rhuEPO Control Total
    Number of subjects
    		 7 8 15
    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)
    		 7 8 15
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 40.00 ( 14.01 ) 40.06 ( 13.26 ) -
    Gender categorical
    Units: Subjects
        Female
    		 1 2 3
        Male
    		 6 6 12

    End points

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    End points reporting groups
    Reporting group title
    rhuEPO
    Reporting group description
    		 -

    Reporting group title
    Control
    Reporting group description
    		 -

    Primary: Inorganic Phosphate (resting state)

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    End point title
    		 Inorganic Phosphate (resting state)
    End point description
    Phosphorus 31 magnetic resonance spectroscopy (31P MRS) offers a non invasive investigation of human skeletal muscle bioenergetics by monitoring relative and absolute changes of phosphocreatine (PCr), inorganic phosphate (Pi) and adenosine triphosphate (ATP) during incremental exercise and recovery.
    End point type
    Primary
    End point timeframe
    Day 0- week 8
    End point values
    		 rhuEPO Control
    Number of subjects analysed
    7
    8
    Units: Pi
        arithmetic mean (standard deviation)
    3.57 ( 0.86 )
    2.85 ( 0.44 )
    Statistical analysis title
    		 Pi (resting state)
    Comparison groups
    Control v rhuEPO
    Number of subjects included in analysis
    15
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 = 0.01
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Day 0- week 8
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 CTCAE
    Dictionary version
    4.0
    Reporting groups
    Reporting group title
    rhuEPO
    Reporting group description
    		 -

    Reporting group title
    Control
    Reporting group description
    		 -

    Serious adverse events
    		 rhuEPO Control
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 rhuEPO Control
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 7 (0.00%)
    0 / 8 (0.00%)
    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: No AEs and SAEs were observed during this trial.

    More information

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

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/23922695
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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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