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    Clinical Trial Results:
    Safety and Efficacy of yIFN treatment in Friedreich ataxia

    Summary
    EudraCT number
    		 2015-002432-40
    Trial protocol
    		 IT  
    Global end of trial date
    17 Jul 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    08 Jun 2022
    First version publication date
    08 Jun 2022
    Other versions
    Summary report(s)
    		 PMID 30237783
    PMID 31930551

    Trial information

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    Trial identification
    Sponsor protocol code
    042/15
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03888664
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Associazione La Nostra Famiglia
    Sponsor organisation address
    via don Luigi Monza 20, Bosisio Parini, Italy, 23842
    Public contact
    Clinical trials unit, Associazione La Nostra Famiglia, +39 031877919, trialsclinici@lanostrafamiglia.it
    Scientific contact
    Clinical trials unit, Associazione La Nostra Famiglia, +39 031877919, trialsclinici@lanostrafamiglia.it
    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
    17 Jul 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Jul 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Jul 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To test the safety of treatment with gamma interferon in Friedreich's Ataxia patients, administered for 6 months at the initial dose of 100 micrograms three times per week (first 2 weeks of treatment) and at the final dose of 200 micrograms three times per week (for the remaining 22 weeks of treatment).
    Protection of trial subjects
    Presence among trial documentation of a patient report form for any adverse event experienced. Phone check made by investigators 15 days after start of treatment. Presence of a dedicated phone number for safety issues available 24/7.
    Background therapy
    		 None.
    Evidence for comparator
    		 Single-arm trial - no comparator.
    Actual start date of recruitment
    11 Jan 2016
    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: 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)
    1
    Adolescents (12-17 years)
    7
    Adults (18-64 years)
    4
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Screening was performed on patients referring to the clinical center for their routine treatment of FRDA. 22 patients were potentially eligible for participation. Of these, 13 patients were screened for inclusion. Of these, 12 patients were enrolled, 1 patient was excluded. Reason for exclusion: severe hypovisus occurred before study start.

    Period 1
    Period 1 title
    Pre-treatment
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Single-arm trial

    Arms
    Arm title
    		 No treatment - baseline
    Arm description
    		 Baseline evaluation without treatment
    Arm type
    		 Experimental

    Investigational medicinal product name
    Imukin
    Investigational medicinal product code
    L03AB03
    Other name
    interferon gamma-1b
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Baseline evanluation period without drug treatment

    Number of subjects in period 1
    		No treatment - baseline
    Started
    12
    Completed
    12
    Period 2
    Period 2 title
    100 micrograms thrice a week dose
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Single-arm trial

    Arms
    Arm title
    		 Gamma interferon 100 micrograms
    Arm description
    		 2 weeks with gamma interferon 100 micrograms thrice a week
    Arm type
    		 Experimental

    Investigational medicinal product name
    Imukin
    Investigational medicinal product code
    L03AB03
    Other name
    interferon gamma-1b
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    100 micrograms subcutaneous injection, three times per week, during the first 2 weeks of use

    Number of subjects in period 2
    		Gamma interferon 100 micrograms
    Started
    12
    Completed
    12
    Period 3
    Period 3 title
    200 micrograms thrice a week dose
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Single-arm trial

    Arms
    Arm title
    		 Gamma interferon 200 micrograms
    Arm description
    		 22 weeks with gamma interferon 200 micrograms thrice a week
    Arm type
    		 Experimental

    Investigational medicinal product name
    Imukin
    Investigational medicinal product code
    L03AB03
    Other name
    interferon gamma-1b
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    200 micrograms subcutaneous injection, three times per week, during the following 22 weeks of use

    Number of subjects in period 3
    		Gamma interferon 200 micrograms
    Started
    12
    Completed
    11
    Not completed
    1
         Adverse event, non-fatal
    1
    Period 4
    Period 4 title
    Follow-up without treatment
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Single-arm trial

    Arms
    Arm title
    		 No treatment - Follow up
    Arm description
    		 No treatment for the following 24 weeks
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 4
    		No treatment - Follow up
    Started
    11
    Completed
    11

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Pre-treatment
    Reporting group description
    		 -

    Reporting group values
    		 Pre-treatment Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 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)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Age in years of the enrolled subjects
    Units: years
        arithmetic mean (standard deviation)
    		 17.33 ( 4.54 ) -
    Gender categorical
    Biological sex for all enrolled subjects
    Units: Subjects
        Female
    		 5 5
        Male
    		 7 7

    End points

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    End points reporting groups
    Reporting group title
    No treatment - baseline
    Reporting group description
    		 Baseline evaluation without treatment
    Reporting group title
    Gamma interferon 100 micrograms
    Reporting group description
    		 2 weeks with gamma interferon 100 micrograms thrice a week
    Reporting group title
    Gamma interferon 200 micrograms
    Reporting group description
    		 22 weeks with gamma interferon 200 micrograms thrice a week
    Reporting group title
    No treatment - Follow up
    Reporting group description
    		 No treatment for the following 24 weeks

    Primary: Number of adverse drug reactions

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    End point title
    		 Number of adverse drug reactions
    End point description
    Sum of the number of AE reported by treated patients each day at different time points along the study: baseline before treatment; during the first 2 weeks of treatment at 100 micrograms; during the following 22 weeks of treatment at 200 micrograms; during the following 24 weeks of follow-up without treatment
    End point type
    Primary
    End point timeframe
    Baseline; week 2; week 24; week 48
    End point values
    		 Gamma interferon 100 micrograms Gamma interferon 200 micrograms No treatment - Follow up
    Number of subjects analysed
    12 [1]
    12 [2]
    11 [3]
    Units: 382
    number (not applicable)
        Fever
    1
    38
    0
        Headache
    7
    110
    0
        Shivers
    0
    49
    0
        Fatigue
    6
    44
    0
        Pain at injection site
    2
    19
    0
        Flu-like symptoms
    0
    19
    0
        Nasal congestion
    0
    5
    0
        Myalgia
    1
    0
    0
        Abdominal pain
    0
    3
    0
        Diarrhea
    1
    0
    0
        Nausea
    1
    4
    0
        Vomiting
    0
    8
    0
        Appetite decreased
    2
    31
    0
        Nosebleed
    0
    5
    0
        Bruising at injection site
    0
    4
    0
        Dyschromia at injection site
    0
    12
    0
        Depression
    0
    10
    0
    Notes
    [1] - All enrolled subjects
    [2] - All enrolled subjects
    [3] - All enrolled subjects
    Statistical analysis title
    		 Chi-squared of adverse events
    Statistical analysis description
    Chi-squared analysis of adverse events numbers collected across study periods 2 and 3. Periods 1 and 4 were not analyzed because no adverse events occurred.
    Comparison groups
    Gamma interferon 100 micrograms v Gamma interferon 200 micrograms
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 equivalence [4]
    P-value
    		 < 0.0001 [5]
    Method
    		 Chi-squared
    Confidence interval
    Notes
    [4] - Null hypothesis: no difference in the number of AE during treatment with 100 or 200 micrograms
    [5] - the number of AE is different during treatment with 100 or 200 micrograms

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    2 weeks after treatment start (100 micrograms thrice a week) 3 months after treatment start (100 micrograms thrice a week for 2 weeks then 200 micrograms thrice a week) 6 months after treatment start (as above) 12 months after treatment start (as above
    Adverse event reporting additional description
    Adverse events reporting was solicited by the administration of a pre-defined form and through phone interviews. Moreover, patients were instructed to contact a dedicated phone number available 24/7 in case of any adverse event.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17
    Reporting groups
    Reporting group title
    All patients
    Reporting group description
    		 All enrolled patients

    Serious adverse events
    		 All patients
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 12 (16.67%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Nervous system disorders
    Headache
    Additional description: Persistent headache, occurred 24h after the adminsitration of Imukin 100 micrograms and resolved with the administration of paracetamol 1g. Event occurred again with every Imukin dose until treatment discontinuation.
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    6 / 6
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Vomiting
    Additional description: Persistent vomiting, occurred 24h after the administration of Imukin 100 micrograms and resolved with the administration of paracetamol 1g. Event occurred again with every Imukin dose until treatment discontinuation.
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    6 / 6
         deaths causally related to treatment / all
    0 / 0
    Abdominal pain
    Additional description: Persistent abdominal pain, occurred 24h after the administration of Imukin 100 micrograms and resolved with the administration of paracetamol 1g. Event occurred again with every Imukin dose until treatment discontinuation.
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    6 / 6
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Myalgia intercostal
    Additional description: Episode of intense coughing, followed by intense chest pain which, upon medical examination, was diagnosed as “basal right thoracic pain of presumed muscular parietal nature"
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    6 / 6
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 All patients
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 12 (100.00%)
    Vascular disorders
    Epistaxis
    Additional description: Epistaxis
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    5
    Nervous system disorders
    Headache
    Additional description: Headache
         subjects affected / exposed
    10 / 12 (83.33%)
         occurrences all number
    117
    General disorders and administration site conditions
    Hyperpyrexia
    Additional description: Fever, increased body temperature
         subjects affected / exposed
    7 / 12 (58.33%)
         occurrences all number
    39
    Chills
    Additional description: Chills
         subjects affected / exposed
    5 / 12 (41.67%)
         occurrences all number
    49
    Fatigue
    Additional description: Sensation of fatigue
         subjects affected / exposed
    9 / 12 (75.00%)
         occurrences all number
    50
    Injection site pain
    Additional description: Pain at the injection site
         subjects affected / exposed
    5 / 12 (41.67%)
         occurrences all number
    21
    Influenza like illness
    Additional description: Flu like illness
         subjects affected / exposed
    4 / 12 (33.33%)
         occurrences all number
    19
    Injection site bruising
    Additional description: Bruising at injection site
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    4
    Injection site discolouration
    Additional description: Skin dyschromia at injection site
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    12
    Gastrointestinal disorders
    Abdominal pain
    Additional description: Abdominal pain
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    3
    Diarrhoea
    Additional description: Diarrhea
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nausea
    Additional description: Nausea
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    5
    Vomiting
    Additional description: Vomiting
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    8
    Respiratory, thoracic and mediastinal disorders
    Nasal congestion
    Additional description: Nasal congestion
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    5
    Psychiatric disorders
    Depression
    Additional description: Depressive symptoms
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    10
    Musculoskeletal and connective tissue disorders
    Myalgia
    Additional description: Muscle pain, myalgia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Decreased appetite
    Additional description: Reduced appetite
         subjects affected / exposed
    4 / 12 (33.33%)
         occurrences all number
    33

    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/31930551
    http://www.ncbi.nlm.nih.gov/pubmed/33162876
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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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