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    Clinical Trial Results:
    DOUBLE BLIND RANDOMIZED STUDY TO ASSESS THE EFFICACY OF BF2.649 COMPARED TO PLACEBO IN ADD-ON TO SODIUM OXYBATE IN THE TREATMENT OF NARCOLEPTIC PATIENTS WITH RESIDUAL EXCESSIVE DAYTIME SLEEPINESS (EDS) DURING 8 WEEKS.

    Summary
    EudraCT number
    		 2011-000084-27
    Trial protocol
    		 DE   ES   FI  
    Global end of trial date
    12 Aug 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    21 Jul 2016
    First version publication date
    21 Jul 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    P10-01/BF2.649
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01789398
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Bioprojet
    Sponsor organisation address
    9 rue Rameau , Paris, France, 75005
    Public contact
    Bioprojet clinical department , Bioprojet, +33 01 47 03 66 33,
    Scientific contact
    Bioprojet clinical department , Bioprojet, +33 01 47 03 66 33,
    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
    14 Sep 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Aug 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Aug 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To show relevant beneficial effect of BF2.649 on EDS compared to placebo in add on to sodium oxybate in narcoleptic patients with residual EDS. To characterize the efficacy of BF2.649 compared to placebo in showing an incremental improvement to the situation achieved by the use of sodium oxybate particularly in terms of a reduction of EDS as measured by the ESS scale. In addition the change in the average number of cataplexy attacks per week was assessed.
    Protection of trial subjects
    Tolerability as measured by Treatment Emergent Adverse Events (TEAE), changes in physical examination and vital signs.
    Background therapy
    		 The study treatment is compared to placebo in add-on to sodium oxybate.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    20 Sep 2012
    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
    Spain: 6
    Country: Number of subjects enrolled
    Finland: 10
    Country: Number of subjects enrolled
    France: 4
    Country: Number of subjects enrolled
    Germany: 12
    Country: Number of subjects enrolled
    Italy: 16
    Worldwide total number of subjects
    48
    EEA total number of subjects
    48
    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)
    45
    From 65 to 84 years
    3
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Main inclusion criteria: Diagnosis of narcolepsy (ICSD-2) Patients complaining of residual EDS Main non-inclusion criteria: Untreated sleep apnea disorder

    Pre-assignment
    Screening details
    		 Study duration for each patient: 12 weeks (1 week of wash-out + 2 weeks for baseline + 8 weeks under double blind study treatment + 1 week for study treatment wash-out). Selected : 51 patients Full Analysis Set (FAS): 48 patients Intent To Treat set (ITT): 46 patients Per Protocol Set: 45 patients

    Pre-assignment period milestones
    Number of subjects started
    		 51 [1]
    Number of subjects completed
    		 48

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Not met entry criteria: 2
    Reason: Number of subjects
    		 Not willing to participate in the study: 1
    Notes
    [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: 51 subjects were screened. 48 subjects were enrolled. There was 3 screen failures
    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    Active treatments and placebo were manufactured according to random code list. No distinction was performed regarding the final batch number.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Placebo
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Buccal use
    Dosage and administration details
    Placebo

    Arm title
    		 BF2.649
    Arm description
    		 BF2.649
    Arm type
    		 Experimental

    Investigational medicinal product name
    BF2.649
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Buccal use
    Dosage and administration details
    Uptitration: 5 the 10mg Stable dose: 10; 20 or 40 mg

    Number of subjects in period 1
    		Placebo BF2.649
    Started
    22
    26
    Completed
    20
    26
    Not completed
    2
    0
         Protocol deviation
    1
    -
         Lack of efficacy
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Reporting group title
    BF2.649
    Reporting group description
    		 BF2.649

    Reporting group values
    		 Placebo BF2.649 Total
    Number of subjects
    		 22 26 48
    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
    FAS and Total safety population (N=48)
    Units: years
        arithmetic mean (standard deviation)
    		 42.7 ( 12.01 ) 42.57 ( 12.92 ) -
    Gender categorical
    FAS and Safety population (N=48)
    Units: Subjects
        Female
    		 7 7 14
        Male
    		 15 19 34
    Subject analysis sets

    Subject analysis set title
    Full Analysis Set (FAS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Full Analysis Set (FAS)

    Subject analysis set title
    Safety Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Safety Set

    Subject analysis set title
    Intent To Treat set (ITT)
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Intent To Treat set (ITT)

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Per Protocol Set

    Subject analysis sets values
    		 Full Analysis Set (FAS) Safety Set Intent To Treat set (ITT) Per Protocol Set
    Number of subjects
    48
    48
    46
    45
    Age categorical
    Units: Subjects
        In utero
        Preterm newborn infants (gestational age < 37 wks)
        Newborns (0-27 days)
        Infants and toddlers (28 days-23 months)
        Children (2-11 years)
        Adolescents (12-17 years)
        Adults (18-64 years)
        From 65-84 years
        85 years and over
    Age continuous
    FAS and Total safety population (N=48)
    Units: years
        arithmetic mean (standard deviation)
    42.63 ( 12.38 )
    42.63 ( 12.38 )
    ( )
    ( )
    Gender categorical
    FAS and Safety population (N=48)
    Units: Subjects
        Female
    14
    14
        Male
    34
    34

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Reporting group title
    BF2.649
    Reporting group description
    		 BF2.649

    Subject analysis set title
    Full Analysis Set (FAS)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Full Analysis Set (FAS)

    Subject analysis set title
    Safety Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Safety Set

    Subject analysis set title
    Intent To Treat set (ITT)
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    Intent To Treat set (ITT)

    Subject analysis set title
    Per Protocol Set
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Per Protocol Set

    Primary: Epworth Sleepiness Scale

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    End point title
    		 Epworth Sleepiness Scale [1]
    End point description
    End point type
    Primary
    End point timeframe
    Change from baseline. Baseline (Mean of pre-treatment measures at V1 and V2), End of double-blind phase (Mean of post-treatment measures at V5 and V6).
    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: Not specified.
    End point values
    		 Placebo BF2.649 Intent To Treat set (ITT)
    Number of subjects analysed
    20
    26
    46
    Units: N-P ANCOVA
        number (not applicable)
    -2.08
    -2.6
    -2.37
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Throughout the study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    17.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 -

    Reporting group title
    BF2.649
    Reporting group description
    		 -

    Serious adverse events
    		 Placebo BF2.649
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 26 (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: 5%
    Non-serious adverse events
    		 Placebo BF2.649
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 22 (9.09%)
    6 / 26 (23.08%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 22 (9.09%)
    6 / 26 (23.08%)
         occurrences all number
    3
    9
    Dizziness
         subjects affected / exposed
    0 / 22 (0.00%)
    1 / 26 (3.85%)
         occurrences all number
    0
    1
    General disorders and administration site conditions
    Gastrointestinal disorder
         subjects affected / exposed
    0 / 22 (0.00%)
    1 / 26 (3.85%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    15 Dec 2011
    Adding of 3 weeks of follow-up [1 extra week (at baseline period) & 2 weeks at the end of study to assess the occurrence or not of withdrawal symptoms]. Patient diary recorded and collected during all the study duration.
    05 Apr 2012
    Adding of 2 visits: a screening visit V0, three weeks prior to the study treatment randomization for patient under psychotropic medication and a control treatment visit at V7 to ensure patient monitoring. Reducing to one week the withdrawal assessment period and providing placebo to all patients during that period. Eliminating SART assessment. Eliminating BF2.649 plasma dosage. Adding an ECG at visit V4.
    10 Dec 2012
    Increase of BF2.649 (Pitolisant) dose up to 40 mg per day.

    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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