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    Clinical Trial Results:
    Statin Treatment of Oxysterol Pathology in SPG5: a Randomized Controlled Trial -Proof of Principle

    Summary
    EudraCT number
    		 2015-000978-35
    Trial protocol
    		 DE  
    Global end of trial date
    01 Mar 2016

    Results information
    Results version number
    		 v1(current)
    This version publication date
    24 Oct 2021
    First version publication date
    24 Oct 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    STOP-SPG5
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    University Hospital Tuebingen
    Sponsor organisation address
    Hoppe - Seyler - Straße 3, Tuebingen, Germany, 72076
    Public contact
    PI, Prof. Dr. L. Schöls, University Hospital Tübingen, 0049 070712982057, ludger.schoels@uni-tuebingen.de
    Scientific contact
    PI, Prof. Dr. L. Schöls, University Hospital Tübingen, 0049 070712982057, ludger.schoels@uni-tuebingen.de
    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
    25 Oct 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    01 Mar 2016
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Mar 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Change of biomarker 27-hydroxycholesterol (27-OHC) level in serum from baseline to day 56 after treatment with atorvastatin 20/40 mg/d for 8 weeks compared to placebo treatment
    Protection of trial subjects
    The study was conducted in accordance with the Declaration of Helsinki and the ICH Guidelines in Good Clinical Practice. The study was not started before the competent ethics committee had given a favorable opinion. Written informed consent was obtained from all patients and the study was only conducted as approved by the Medical Ethic Boards of the Univeristy of Tübingen and the national regulatory institution (Bundesamt für Arzneimittel und Medizinprodukte - BfArM).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    09 Nov 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Germany: 14
    Worldwide total number of subjects
    14
    EEA total number of subjects
    14
    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)
    14
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 A total of 34 subjects from 28 families with a clinical diagnosis of HSP and genetically confirmed SPG5 were recruited in clinical centres in Antwerp, Athens, Conegliano, Lecco, Milano, Munich, Naples, Tübingen, Porto Alegre and Sao Paulo. Additionally 11 uneffected family members, carrying heterozygous CYP7B1 mutations, were included.

    Pre-assignment
    Screening details
    		 A total of 34 subjects with a clinical diagnosis of HSP and genetically confirmed SPG5 were recruited . Additionally 11 uneffected family members, carrying heterozygous CYP7B1 mutations were included. Patients fulfilling the inclusion and exclusion criterias were enrolled into the study. Randomization was performed by www.randomization.com .

    Period 1
    Period 1 title
    Atorvastatin (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Verum
    Arm description
    		 7 patients received Atorvastatin.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Atorvastatin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Atorvastatin was given at a dosage of 40mg/die for 8 weeks in adults and 20 mg/die in children under 18 years of age.

    Arm title
    		 Placebo
    Arm description
    		 7 patients received Placebo.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Atorvastatin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Atorvastatin was given at a dosage of 40mg/die for 8 weeks in adults and 20 mg/die in children under 18 years of age.

    Number of subjects in period 1
    		Verum Placebo
    Started
    7
    7
    Completed
    7
    7

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Verum
    Reporting group description
    		 7 patients received Atorvastatin.

    Reporting group title
    Placebo
    Reporting group description
    		 7 patients received Placebo.

    Reporting group values
    		 Verum Placebo Total
    Number of subjects
    		 7 7 14
    Age categorical
    According to protocol 7 patients received Atorvastatin and were compared to a second group of 7 patients receiving placebo.
    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)
    		 0 0 0
        From 65-84 years
    		 7 7 14
        85 years and over
    		 0 0 0
        Subjects
    		 0 0 0
    Gender categorical
    Verum group: 4 males, 3 females Placebo group: 2 males, 5 females
    Units: Subjects
        Female
    		 3 5 8
        Male
    		 4 2 6

    End points

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    End points reporting groups
    Reporting group title
    Verum
    Reporting group description
    		 7 patients received Atorvastatin.

    Reporting group title
    Placebo
    Reporting group description
    		 7 patients received Placebo.

    Primary: Biomarker 27 OHC

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    End point title
    		 Biomarker 27 OHC [1]
    End point description
    End point type
    Primary
    End point timeframe
    Change of biomarker 27-hydroxycholesterol (27-OHC) level in serum from baseline to day 56 after treatment with atorvastatin once daily for 8 weeks compared to placebo treatment.
    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: The statistical analysis can be found in the publication.
    End point values
    		 Verum Placebo
    Number of subjects analysed
    7
    7
    Units: ng/ml
        median (inter-quartile range (Q1-Q3))
    -307 (-409 to -144)
    -1 (-27 to 40)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    The side effects were monitored during the observation period by lab tests after and while treatment on visit 1 – 2 and in week 4 by the family doctor.
    Adverse event reporting additional description
    Table of Adverse Events can be found in the publication
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20
    Reporting groups
    Reporting group title
    Verum
    Reporting group description
    		 a total of five adverse events were reported, including two adverse events in the verum group (n=1 GGT elevation from 109 to 119 U/l; n=1 prolonged menstrual bleeding)

    Reporting group title
    Placebo
    Reporting group description
    		 a total of five adverse events were reported, including three in the placebo group (n=2 post-dural-puncture; n=1 stomach flu)

    Serious adverse events
    		 Verum Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (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: 0%
    Non-serious adverse events
    		 Verum Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 3 (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: The Adverse events can be found in the publication.

    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/29126212
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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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