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    Clinical Trial Results:
    Clinical trial - phase II - to test safety and efficay of Etravirine's treatment in Friedreich Ataxia's patients

    Summary
    EudraCT number
    		 2019-002618-38
    Trial protocol
    		 IT  
    Global end of trial date
    17 Jan 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 Jun 2024
    First version publication date
    20 Jun 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    721
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04273165
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Associazione La Nostra Famiglia
    Sponsor organisation address
    Via Don Luigi Monza, 1, Ponte Lambro, Italy, 22037
    Public contact
    Segreteria Scientifica, Associazione La Nostra Famiglia, +39 031877330, medea@lanostrafamiglia.it
    Scientific contact
    Segreteria Scientifica, Associazione La Nostra Famiglia, +39 031877330, medea@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
    28 May 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    17 Jan 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Jan 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Test the safety of ETR treatment in patients with FRDA (two groups of patients treated with 2 different doses: 200 mg / day and 400 mg / day). The goal is to monitor all EAs that will occur during 4 months of treatment with ETR compared to EAs recorded during 4 months of observation before and 4 months after treatment.
    Protection of trial subjects
    Presence among trial documentation of a patient report form for any adverse event experienced. Presence of a dedicated phone number for safety issues available 24/7. Physical exmination during visits, including vital signs, whole body skin inspection, blood testing, urine testing.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Sep 2020
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Scientific research
    Long term follow-up duration
    		 4 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Italy: 35
    Worldwide total number of subjects
    35
    EEA total number of subjects
    35
    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)
    4
    Adults (18-64 years)
    30
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Recruitment was promoted by advertising the study through patients' associations and among patients currently followed at the clinical site. Recruitment was done at the clinical site.

    Pre-assignment
    Screening details
    		 38 patients were screened for inclusion. Of these, 35 were enrolled and 3 excluded. Of the excluded, 2 were not capable of completing the exercise test, 1 withdrew consent immediately after the screening visit.

    Period 1
    Period 1 title
    Etravirine treatment months 0 to 2
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    No blinding was done

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Etravirine 200 mg/day
    Arm description
    		 2 months treatment with etravirine 200 mg/day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Intelence
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Intelence tablets strength 200mg ; 1 tablet per day in the morning, for 2 months

    Arm title
    		 Etravirine 400 mg/day
    Arm description
    		 2 months treatment with etravirine 400 mg/day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Intelence
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Intelence tablets strength 200mg ; 2 tablets per day in the morning, for 2 months

    Number of subjects in period 1
    		Etravirine 200 mg/day Etravirine 400 mg/day
    Started
    17
    18
    Completed
    17
    17
    Not completed
    0
    1
         Adverse event, non-fatal
    -
    1
    Period 2
    Period 2 title
    Continuation of etravirine months 3 to 4
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Etravirine 200 mg/day
    Arm description
    		 2 months treatment with etravirine 200 mg/day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Intelence
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Intelence tablets strength 200mg ; 1 tablet per day in the morning, for 2 months

    Arm title
    		 Etravirine 400 mg/day
    Arm description
    		 2 months treatment with etravirine 400 mg/day
    Arm type
    		 Experimental

    Investigational medicinal product name
    Intelence
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Intelence tablets strength 200mg ; 2 tablets per day in the morning, for 2 months

    Number of subjects in period 2
    		Etravirine 200 mg/day Etravirine 400 mg/day
    Started
    17
    17
    Completed
    17
    17
    Period 3
    Period 3 title
    Follow-up without treatment mo. 5 to 8
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Etravirine 200 mg/day
    Arm description
    		 4 months without treatment
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    		 Etravirine 400 mg/day
    Arm description
    		 4 months without treatment
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 3
    		Etravirine 200 mg/day Etravirine 400 mg/day
    Started
    17
    17
    Completed
    17
    17

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Etravirine treatment months 0 to 2
    Reporting group description
    		 -

    Reporting group values
    		 Etravirine treatment months 0 to 2 Total
    Number of subjects
    		 35 35
    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)
    		 1 1
        Adolescents (12-17 years)
    		 4 4
        Adults (18-64 years)
    		 30 30
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 11.83 ( 5.7 ) -
    Gender categorical
    Units: Subjects
        Female
    		 19 19
        Male
    		 16 16

    End points

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    End points reporting groups
    Reporting group title
    Etravirine 200 mg/day
    Reporting group description
    		 2 months treatment with etravirine 200 mg/day

    Reporting group title
    Etravirine 400 mg/day
    Reporting group description
    		 2 months treatment with etravirine 400 mg/day
    Reporting group title
    Etravirine 200 mg/day
    Reporting group description
    		 2 months treatment with etravirine 200 mg/day

    Reporting group title
    Etravirine 400 mg/day
    Reporting group description
    		 2 months treatment with etravirine 400 mg/day
    Reporting group title
    Etravirine 200 mg/day
    Reporting group description
    		 4 months without treatment

    Reporting group title
    Etravirine 400 mg/day
    Reporting group description
    		 4 months without treatment

    Primary: Number of adverse drug reactions

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    End point title
    		 Number of adverse drug reactions
    End point description
    see adverse events
    End point type
    Primary
    End point timeframe
    2 months treatment 4 months treatment 4 months treatment then 4 months no treatment
    End point values
    		 Etravirine 200 mg/day Etravirine 400 mg/day Etravirine 200 mg/day Etravirine 400 mg/day Etravirine 200 mg/day Etravirine 400 mg/day
    Number of subjects analysed
    17
    17 [1]
    17
    17
    17
    17
    Units: number
    17
    18
    17
    17
    17
    17
    Notes
    [1] - 1 drop out due to non severe ADR
    Statistical analysis title
    		 Chi-squared of adverse events
    Comparison groups
    Etravirine 200 mg/day v Etravirine 400 mg/day v Etravirine 200 mg/day v Etravirine 400 mg/day v Etravirine 200 mg/day v Etravirine 400 mg/day
    Number of subjects included in analysis
    102
    Analysis specification
    Post-hoc
    Analysis type
    		 superiority
    P-value
    		 < 0.05
    Method
    		 Chi-squared
    Parameter type
    		 no estimation
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Period 1: Etravirine treatment months 0 to 2 Period 2: Continuation of etravirine months 3 to 4 Period 3: Follow-up without treatment months 5 to 8
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17
    Reporting groups
    Reporting group title
    Etravirine 200 months 0-2
    Reporting group description
    		 Etravirine 200 months 0-2

    Reporting group title
    Etravirine 400 months 0-2
    Reporting group description
    		 Etravirine 400 months 0-2

    Reporting group title
    Etravirine 200 months 3-4
    Reporting group description
    		 Etravirine 200 months 3-4

    Reporting group title
    Etravirine 400 months 3-4
    Reporting group description
    		 Etravirine 400 months 3-4

    Reporting group title
    Etravirine 200 months 5-8
    Reporting group description
    		 Etravirine 200 months 5-8 follow up without treatment

    Reporting group title
    Etravirine 400 months 5-8
    Reporting group description
    		 Etravirine 200 months 5-8 follow up without treatment

    Serious adverse events
    		 Etravirine 200 months 0-2 Etravirine 400 months 0-2 Etravirine 200 months 3-4 Etravirine 400 months 3-4 Etravirine 200 months 5-8 Etravirine 400 months 5-8
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Cardiac disorders
    Tachycardia
    Additional description: Supraventricular tachycardic arrhythmia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Etravirine 200 months 0-2 Etravirine 400 months 0-2 Etravirine 200 months 3-4 Etravirine 400 months 3-4 Etravirine 200 months 5-8 Etravirine 400 months 5-8
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    17 / 17 (100.00%)
    18 / 18 (100.00%)
    2 / 17 (11.76%)
    5 / 17 (29.41%)
    0 / 17 (0.00%)
    11 / 17 (64.71%)
    Investigations
    Blood cholesterol increased
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 18 (11.11%)
    2 / 17 (11.76%)
    2 / 17 (11.76%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    2
    2
    2
    1
    1
    Blood triglycerides increased
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 18 (0.00%)
    2 / 17 (11.76%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    3
    0
    0
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 18 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 18 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Amylase increased
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    Leukopenia
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    0
    1
    0
    1
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 17 (0.00%)
    2 / 18 (11.11%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    0
    1
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    6 / 17 (35.29%)
    10 / 18 (55.56%)
    1 / 17 (5.88%)
    2 / 17 (11.76%)
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    7
    18
    1
    3
    0
    4
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 17 (5.88%)
    2 / 18 (11.11%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    1
    3
    0
    0
    0
    2
    Oedema peripheral
    Additional description: Feeling of swollen feet
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    0
    Eye disorders
    Vision blurred
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    3 / 17 (17.65%)
    4 / 18 (22.22%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    3 / 17 (17.65%)
         occurrences all number
    5
    7
    0
    1
    0
    3
    Nausea
         subjects affected / exposed
    1 / 17 (5.88%)
    3 / 18 (16.67%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    1
    3
    0
    2
    0
    5
    Vomiting
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 18 (5.56%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    0
    1
    0
    4
    Constipation
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 17 (11.76%)
    2 / 18 (11.11%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    2
    0
    0
    0
    1
    Hyperhidrosis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Renal and urinary disorders
    Urinary incontinence
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    3
    0
    0
    0
    0
    0
    Psychiatric disorders
    Anger
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 18 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 18 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Mar 2022
    Extended recruitment and overall trial duration

    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
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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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