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    Clinical Trial Results:
    Eine offene, nicht randomisierte, einarmige Pilotstudie zur Beurteilung der Wirksamkeit von Erythropoetin bei Friedreich Ataxie.

    Summary
    EudraCT number
    		 2005-005938-12
    Trial protocol
    		 AT  
    Global end of trial date
    01 Nov 2007

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Dec 2021
    First version publication date
    13 Dec 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    29-09-1963
    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 MSc, Medical University Innsbruck, University Hospital for Neurology, Anichstrasse 35, +43 (0)512-504-26285, sylvia.boesch@tirol-kliniken.at
    Scientific contact
    Priv.Doz.Dr.Sylvia Bösch MSc, Medical University Innsbruck, University Hospital for Neurology, Anichstrasse 35, +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 Nov 2007
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Nov 2007
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Nov 2007
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Basierend auf eigenen in-vitro Voruntersuchungen soll nun im Rahmen einer Pilotstudie die Sicherheit und Wirksamkeit von Erythropoetin in der Behandlung von Patienten mit Friedreich Ataxie untersucht werden. Diese Untersuchung stellt nach bisher frustranen Therapieversuchen (z.B. Desoxyferramin, Idebenone) einen gänzlich neuen Therapieansatz dar. HAUPTZIELKRITERIUM: Stabile Aufregulierung von FRATAXIN bei Patienten mit Friedreich Ataxie über 8 Wochen. Recently, we showed that rHuEPO increases frataxin levels in isolated lymphocytes from FRDA patients in vitro. Based on these findings, we initiated a clinical pilot trial to investigatethe effect of rHuEPO on frataxin levels in FRDA patients.
    Protection of trial subjects
    Safety was assessed by weekly measurement of hematocrit (Hc), hemoglobin, erythrocyte counts, reticulocytes, and thrombocytes. Blood pressure using Riva Rocci technique was monitored weekly. Electrocardiogram was performed at baseline and last visit.
    Background therapy
    		 -
    Evidence for comparator
    		 There was no evidence for a comparator.
    Actual start date of recruitment
    08 Jun 2006
    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: 12
    Worldwide total number of subjects
    12
    EEA total number of subjects
    12
    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)
    12
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patients with definite FRDA who were 18 years or older were included.

    Pre-assignment
    Screening details
    		 Twelve patients with a definite diagnosis of FRDA were included. All patients had ataxia, two patients suffered from mild cardiomyopathy, and no patient had diabetes.

    Period 1
    Period 1 title
    Treatment (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 rHuEPO
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Neo-Recormon
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    FRDA patients received 5,000IU rHuEPO (Neorecormone; Roche, Vienna, Austria) three times weekly subcutaneously for a period of 8 weeks.

    Number of subjects in period 1
    		rHuEPO
    Started
    12
    Completed
    11
    Not completed
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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

    Reporting group values
    		 Treatment Total
    Number of subjects
    		 12 12
    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)
    		 12 12
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 34.9 (18 to 55) -
    Gender categorical
    Units: Subjects
        Female
    		 4 4
        Male
    		 8 8

    End points

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

    Primary: Frataxin levels

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    End point title
    		 Frataxin levels [1]
    End point description
    Frataxin levels at baseline (BL) and after 8 weeks of treatment with recombinant human erythropoietin (rHuEPO) were measured. Individual frataxin levels at baseline were set as 100% and compared to fratraxin levels after 8 weeks of treatment.
    End point type
    Primary
    End point timeframe
    Day 0- end of week 8
    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: Changes of frataxin levels at week 8 versus baseline were performed using the paired t test (n=10; p < 0.01).
    End point values
    		 rHuEPO
    Number of subjects analysed
    10
    Units: change of frataxin levels in %
    arithmetic mean (full range (min-max))
        change in % at week 8
    27 (15 to 63)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Day 0 - end of week 8
    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
    		 -

    Serious adverse events
    		 rHuEPO
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (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 / 12 (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 during 8 weeks, 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/17702040
    http://www.ncbi.nlm.nih.gov/pubmed/18759345
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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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