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    Clinical Trial Results:
    Untersuchung der Dosis-Wirk-Beziehung unterschiedlicher Erythropoetin-Dosen auf Frataxin bei Friedreich Ataxie

    Summary
    EudraCT number
    		 2007-004919-55
    Trial protocol
    		 AT  
    Global end of trial date
    01 Dec 2011

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Jan 2022
    First version publication date
    28 Jan 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    04-01-1980
    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, Medical University Innsbruck, Department of Neurology, Anichstrasse 35, 6020 Innsbruck, +43 (0)512-504-26285, sylvia.boesch@tirol-kliniken.at
    Scientific contact
    Priv.Doz. Dr. Sylvia Bösch, Medical University Innsbruck, Department of 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
    29 Apr 2011
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    29 Apr 2011
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Dec 2011
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Dosis Wirkungs Beziehung zwischen Erythropoetin und Frataxin bei Patienten mit Friedreich Ataxie
    Protection of trial subjects
    Blood samples were taken to monitor haemoglobin levels, haematocrit and platelet counts. Analyses of blood parameters were done by standard laboratory procedures in an ISO 9001:2009 certified laboratory. Blood pressure was measured regularly. ECG was performed at screening and last visit.
    Background therapy
    		 -
    Evidence for comparator
    		 There was no evidence for a comparator.
    Actual start date of recruitment
    27 Dec 2007
    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: 5
    Worldwide total number of subjects
    5
    EEA total number of subjects
    5
    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)
    5
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Five patients with definite diagnosis of FRDA were included in this study after written informed consent.

    Pre-assignment
    Screening details
    		 None of the patients had idebenone, co-enzyme Q10 or other antioxidants 6 weeks prior to the study.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Single blind
    Roles blinded
    		 Subject
    Blinding implementation details
    In this trial FRDA patients received three different single injections of rhuEPO (5,000, 10,000 and 30,000 IU; epoetin beta, Roche, Basel, Switzerland) subcutaneously in monthly intervals. Laboratory staff and patients were blinded for the amount of administered rhuEPO.

    Arms
    Arm title
    		 rhuEPO
    Arm description
    		 In this single site, one arm pilot trial FRDA patients received three different single injections of rhuEPO (5,000, 10,000 and 30,000 IU; epoetin beta, Roche, Basel, Switzerland) subcutaneously in monthly intervals.
    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
    Patients received three rhuEPO singledose injections (5,000, 10,000,30,000 IU) at intervals of 1 month.

    Number of subjects in period 1
    		rhuEPO
    Started
    5
    Completed
    5

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall study
    Reporting group description
    		 -

    Reporting group values
    		 Overall study Total
    Number of subjects
    		 5 5
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 0 0
        Adults (18-64 years)
    		 5 5
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        median (inter-quartile range (Q1-Q3))
    		 49 (31 to 52) -
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 4 4

    End points

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    End points reporting groups
    Reporting group title
    rhuEPO
    Reporting group description
    		 In this single site, one arm pilot trial FRDA patients received three different single injections of rhuEPO (5,000, 10,000 and 30,000 IU; epoetin beta, Roche, Basel, Switzerland) subcutaneously in monthly intervals.

    Primary: Monthly Frataxin Measurements

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    End point title
    		 Monthly Frataxin Measurements [1]
    End point description
    We found a 19% frataxin increase after applying 5,000 IU rhuEPO (median 119%; IQR 106–254%). Frataxin measurements after 2 months showed a boost up to 263% (IQR 142–417%) as compared to rhuEPO naïve baseline frataxin levels. Finally, frataxin levels increased to maximal levels of 310% (IQR 182–480%; p=0.03) after 3 months.
    End point type
    Primary
    End point timeframe
    Baseline - 3 months
    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: Kolmogorov–Smirnov test was used to assess normal distribution of frataxin levels.
    End point values
    		 rhuEPO
    Number of subjects analysed
    5
    Units: Percentage
    median (inter-quartile range (Q1-Q3))
        after 1 month
    119 (106 to 254)
        after 2 months
    263 (142 to 417)
        after 3 months
    310 (182 to 480)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    27.12.2007- 29.04.2011
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 CTCAE
    Dictionary version
    3.0
    Reporting groups
    Reporting group title
    rhuEPO
    Reporting group description
    		 In this single site, one arm pilot trial FRDA patients received three different single injections of rhuEPO (5,000, 10,000 and 30,000 IU; epoetin beta, Roche, Basel, Switzerland) subcutaneously in monthly intervals.

    Serious adverse events
    		 rhuEPO
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 rhuEPO
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 5 (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: rhuEPO was well tolerated, no non- serious adverse events were observed.

    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/21597884
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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
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