Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   44294   clinical trials with a EudraCT protocol, of which   7351   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-861 for the Treatment of Narcolepsy With Cataplexy (Narcolepsy Type 1)

    Summary
    EudraCT number
    2022-001654-38
    Trial protocol
    FR   NO   FI   SE   NL   ES  
    Global end of trial date
    14 Dec 2023

    Results information
    Results version number
    v1(current)
    This version publication date
    29 Dec 2024
    First version publication date
    29 Dec 2024
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    TAK-861-2001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT05687903
    WHO universal trial number (UTN)
    U1111-1277-4261
    Other trial identifiers
    jRCT: jRCT2031220644
    Sponsors
    Sponsor organisation name
    Takeda Development Center Americas, Inc.
    Sponsor organisation address
    95 Hayden Avenue, Lexington, Massachusetts, United States, 02421
    Public contact
    Study Director, Takeda, TrialDisclosures@takeda.com
    Scientific contact
    Study Director, Takeda, TrialDisclosures@takeda.com
    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 Dec 2023
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Dec 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this study was to assess the effect of TAK-861 on excessive daytime sleepiness (EDS) as measured by sleep latency from the Maintenance of Wakefulness Test (MWT).
    Protection of trial subjects
    All study participants or their guardians were required to read and sign an Informed Consent Form (ICF).
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    09 Jan 2023
    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
    United States: 25
    Country: Number of subjects enrolled
    Finland: 1
    Country: Number of subjects enrolled
    France: 17
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Italy: 22
    Country: Number of subjects enrolled
    Netherlands: 4
    Country: Number of subjects enrolled
    Norway: 2
    Country: Number of subjects enrolled
    Spain: 23
    Country: Number of subjects enrolled
    Sweden: 1
    Country: Number of subjects enrolled
    Switzerland: 5
    Country: Number of subjects enrolled
    Australia: 4
    Country: Number of subjects enrolled
    Japan: 5
    Worldwide total number of subjects
    112
    EEA total number of subjects
    73
    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)
    112
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    Participants took part in the study at 33 investigative sites globally from 09 January 2023 to 14 December 2023.

    Pre-assignment
    Screening details
    Participants with a diagnosis of narcolepsy type 1 (NT1) were enrolled in the study to receive either TAK-861 or placebo.

    Period 1
    Period 1 title
    Overall Study (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
    No

    Arm title
    Placebo
    Arm description
    Participants received placebo tablets matching TAK-861, orally, BID, from Days 1 to 56.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo tablets matching TAK-861, orally, twice daily (BID) from Days 1 to 56.

    Arm title
    TAK-861 0.5 mg BID
    Arm description
    Participants received TAK-861 0.5 mg, orally, BID, from Days 1 to 56.
    Arm type
    Experimental

    Investigational medicinal product name
    TAK-861
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    TAK-861 at an oral dose of 0.5 mg, administered BID, from Days 1 to 56.

    Arm title
    TAK-861 2 mg BID
    Arm description
    Participants received TAK-861 2 mg, orally, BID, from Days 1 to 56.
    Arm type
    Experimental

    Investigational medicinal product name
    TAK-861
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    TAK-861 at an oral dose of 2 mg, administered BID, from Days 1 to 56.

    Arm title
    TAK-861 2 mg and 5 mg
    Arm description
    Participants received TAK-861 2 mg followed by the 5 mg dose, orally, from Days 1 to 56.
    Arm type
    Experimental

    Investigational medicinal product name
    TAK-861
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    TAK-861 at an oral dose of 2 mg and 5 mg, administered BID, from Days 1 to 56.

    Arm title
    TAK-861 7 mg QD
    Arm description
    Participants received TAK-861 7 mg, orally, QD, from Days 1 to 56. Placebo was given as the second dose.
    Arm type
    Experimental

    Investigational medicinal product name
    TAK-861
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    TAK-861 at an oral dose of 7.5 mg, administered once daily (QD), from Days 1 to 56.

    Number of subjects in period 1
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg and 5 mg TAK-861 7 mg QD
    Started
    22
    23
    21
    23
    23
    Completed
    21
    22
    21
    22
    23
    Not completed
    1
    1
    0
    1
    0
         Protocol deviation
    1
    1
    -
    1
    -

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo tablets matching TAK-861, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 0.5 mg BID
    Reporting group description
    Participants received TAK-861 0.5 mg, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 2 mg BID
    Reporting group description
    Participants received TAK-861 2 mg, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 2 mg and 5 mg
    Reporting group description
    Participants received TAK-861 2 mg followed by the 5 mg dose, orally, from Days 1 to 56.

    Reporting group title
    TAK-861 7 mg QD
    Reporting group description
    Participants received TAK-861 7 mg, orally, QD, from Days 1 to 56. Placebo was given as the second dose.

    Reporting group values
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg and 5 mg TAK-861 7 mg QD Total
    Number of subjects
    22 23 21 23 23
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    37.5 ( 11.86 ) 32.7 ( 11.06 ) 31.7 ( 11.31 ) 34.7 ( 11.48 ) 33.3 ( 11.94 ) -
    Gender categorical
    Units: Subjects
        Female
    14 11 9 14 10 58
        Male
    8 12 12 9 13 54
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    3 2 1 0 2 8
        Not Hispanic or Latino
    19 21 20 23 20 103
        Unknown or Not Reported
    0 0 0 0 1 1
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0 0 0 0
        Asian
    1 2 0 3 2 8
        Native Hawaiian or Other Pacific Islander
    0 0 0 0 0 0
        Black or African American
    2 1 2 0 1 6
        White
    19 19 19 19 20 96
        More than one race
    0 1 0 1 0 2
        Unknown or Not Reported
    0 0 0 0 0 0
    Average Sleep Latency From the Maintenance of Wakefulness Test (MWT)
    The MWT is a validated, objective measure that evaluates a participant's ability to remain awake under soporific conditions for a defined period. During each MWT session (1 session = 40 minutes), participants were instructed to sit quietly and remain awake for as long as possible. Sleep latency in each session was recorded on electroencephalography (EEG). If no sleep was observed according to these rules, then the latency was defined as 40 minutes.
    Units: minutes
        arithmetic mean (standard deviation)
    6.1 ( 8.82 ) 5.6 ( 7.89 ) 3.9 ( 5.98 ) 4.2 ( 3.63 ) 3.6 ( 4.87 ) -

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo tablets matching TAK-861, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 0.5 mg BID
    Reporting group description
    Participants received TAK-861 0.5 mg, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 2 mg BID
    Reporting group description
    Participants received TAK-861 2 mg, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 2 mg and 5 mg
    Reporting group description
    Participants received TAK-861 2 mg followed by the 5 mg dose, orally, from Days 1 to 56.

    Reporting group title
    TAK-861 7 mg QD
    Reporting group description
    Participants received TAK-861 7 mg, orally, QD, from Days 1 to 56. Placebo was given as the second dose.

    Primary: Change From Baseline in the Average Sleep Latency as Determined From the MWT at Week 8

    Close Top of page
    End point title
    Change From Baseline in the Average Sleep Latency as Determined From the MWT at Week 8
    End point description
    The MWT is a validated, objective measure that evaluates a participant's ability to remain awake under soporific conditions for a defined period. During each MWT session (1 session = 40 minutes), participants were instructed to sit quietly and remain awake for as long as possible. Sleep latency in each session was recorded on EEG. If no sleep was observed according to these rules, then the latency was defined as 40 minutes. The linear mixed effects model for repeated measures (MMRM) was used for analysis. Full Analysis Set included all participants who were randomized and received at least one dose of study drug and had at least one post-dose efficacy measurement. Subjects analysed is the number of participants with data available for analyses.
    End point type
    Primary
    End point timeframe
    Baseline, Week 8
    End point values
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg and 5 mg TAK-861 7 mg QD
    Number of subjects analysed
    21
    19
    21
    20
    23
    Units: minutes
    least squares mean (standard error)
        Week 8
    -1.16 ( 2.061 )
    12.49 ( 2.128 )
    23.50 ( 2.042 )
    25.42 ( 2.071 )
    14.96 ( 1.953 )
    Statistical analysis title
    Mean Sleep Latency
    Comparison groups
    Placebo v TAK-861 0.5 mg BID
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.001 [1]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    13.65
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    7.74
         upper limit
    19.57
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.983
    Notes
    [1] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    Mean Sleep Latency From the MWT
    Comparison groups
    Placebo v TAK-861 2 mg and 5 mg
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.001 [2]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    26.58
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    20.81
         upper limit
    32.35
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.91
    Notes
    [2] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    Mean Sleep Latency From the MWT
    Comparison groups
    Placebo v TAK-861 7 mg QD
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.001 [3]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    16.13
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    10.49
         upper limit
    21.76
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.842
    Notes
    [3] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    Mean Sleep Latency From the MWT
    Comparison groups
    Placebo v TAK-861 2 mg BID
    Number of subjects included in analysis
    42
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.001 [4]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    24.67
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    18.87
         upper limit
    30.46
    Variability estimate
    Standard error of the mean
    Dispersion value
    2.921
    Notes
    [4] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.

    Secondary: Change From Baseline in Epworth Sleepiness Scale (ESS) Total Score at Week 8

    Close Top of page
    End point title
    Change From Baseline in Epworth Sleepiness Scale (ESS) Total Score at Week 8
    End point description
    The ESS is a subjective, self-administered, validated scale (scored 0 to 3) to respond to each of the 8 questions of daily life that asks participants how likely they are to fall asleep in those situations. The scores are summed to give an overall score of 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are considered to be within the normal range. The MMRM was used for analysis. Full Analysis Set included all participants who were randomized and received at least one dose of study drug and had at least one post-dose efficacy measurement. Subjects analysed is the number of participants with data available for analyses.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 8
    End point values
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg and 5 mg TAK-861 7 mg QD
    Number of subjects analysed
    21
    21
    21
    22
    23
    Units: score on a scale
        least squares mean (standard error)
    -2.50 ( 1.109 )
    -8.92 ( 1.085 )
    -13.79 ( 1.115 )
    -12.81 ( 1.073 )
    -11.29 ( 1.064 )
    Statistical analysis title
    ESS Total Score at Week 8
    Comparison groups
    Placebo v TAK-861 0.5 mg BID
    Number of subjects included in analysis
    42
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.004 [5]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    -6.42
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -9.53
         upper limit
    -3.32
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.564
    Notes
    [5] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    Change From Baseline in ESS Total Score at Week 8
    Comparison groups
    Placebo v TAK-861 2 mg BID
    Number of subjects included in analysis
    42
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.001 [6]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    -11.3
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -14.44
         upper limit
    -8.16
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.581
    Notes
    [6] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    Change From Baseline in ESS Total Score at Week 8
    Comparison groups
    Placebo v TAK-861 2 mg and 5 mg
    Number of subjects included in analysis
    43
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.001 [7]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    -10.31
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -13.35
         upper limit
    -7.27
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.53
    Notes
    [7] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    Change From Baseline in ESS Total Score at Week 8
    Comparison groups
    Placebo v TAK-861 7 mg QD
    Number of subjects included in analysis
    44
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    < 0.001 [8]
    Method
    MMRM
    Parameter type
    Estimate of LS Mean Difference
    Point estimate
    -8.79
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    -11.84
         upper limit
    -5.75
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.535
    Notes
    [8] - The analysis used a linear MMRM with fixed effects for Baseline, age, prior use of narcolepsy medications, treatment, visit, and treatment-by-visit interaction as covariates. Reported p-value is adjusted for multiplicity.

    Secondary: Weekly Cataplexy Rate (WCR) at Week 8

    Close Top of page
    End point title
    Weekly Cataplexy Rate (WCR) at Week 8
    End point description
    Participants completed a daily patient-reported sleep diary to record self-reported narcolepsy symptoms. Participants recorded episodes of cataplexy attacks in the diary. The total number of events averaged for a week were reported. WCR = (total number of cataplexy attacks over a number of non-missing diary days for a given duration/number of non-missing diary days in that duration)*7. The generalized estimating equations (GEE) model was used for analysis. Full Analysis Set included all participants who were randomized and received at least one dose of study drug and had at least one post-dose efficacy measurement. Subjects analysed is the number of participants with data available for analyses.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg and 5 mg TAK-861 7 mg QD
    Number of subjects analysed
    20
    21
    21
    22
    23
    Units: cataplexy attacks per week
        least squares mean (confidence interval 95%)
    8.76 (5.68 to 13.51)
    4.24 (2.60 to 6.92)
    3.14 (1.65 to 5.98)
    2.48 (1.30 to 4.73)
    5.89 (3.64 to 9.53)
    Statistical analysis title
    WCR at Week 8
    Statistical analysis description
    The GEE model was used for analysis with fixed effects for visit, treatment, treatment-by-visit interaction, Baseline WCR, age, and prior use of narcolepsy medications.
    Comparison groups
    Placebo v TAK-861 0.5 mg BID
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.25 [9]
    Method
    GEE
    Parameter type
    IRR
    Point estimate
    0.48
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.25
         upper limit
    0.93
    Notes
    [9] - GEE model featuring negative binomial distribution was used for analysis where incidence rate was exponentiated LS mean & incidence rate ratio (IRR) was the exponentiated LS mean difference from placebo. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    WCR at Week 8
    Statistical analysis description
    The GEE model was used for analysis with fixed effects for visit, treatment, treatment-by-visit interaction, Baseline WCR, age, and prior use of narcolepsy medications.
    Comparison groups
    Placebo v TAK-861 7 mg QD
    Number of subjects included in analysis
    43
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.25 [10]
    Method
    GEE
    Parameter type
    IRR
    Point estimate
    0.67
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.35
         upper limit
    1.29
    Notes
    [10] - The GEE model featuring a negative binomial distribution was used for analysis where the incidence rate was the exponentiated LS mean and the IRR was the exponentiated LS mean difference from placebo. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    WCR at Week 8
    Statistical analysis description
    The GEE model was used for analysis with fixed effects for visit, treatment, treatment-by-visit interaction, Baseline WCR, age, and prior use of narcolepsy medications.
    Comparison groups
    Placebo v TAK-861 2 mg and 5 mg
    Number of subjects included in analysis
    42
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.003 [11]
    Method
    GEE
    Parameter type
    IRR
    Point estimate
    0.28
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.13
         upper limit
    0.6
    Notes
    [11] - The GEE model featuring a negative binomial distribution was used for analysis where the incidence rate was the exponentiated LS mean and the IRR was the exponentiated LS mean difference from placebo. Reported p-value is adjusted for multiplicity.
    Statistical analysis title
    WCR at Week 8
    Statistical analysis description
    The GEE model was used for analysis with fixed effects for visit, treatment, treatment-by-visit interaction, Baseline WCR, age, and prior use of narcolepsy medications.
    Comparison groups
    Placebo v TAK-861 2 mg BID
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    superiority
    P-value
    = 0.034 [12]
    Method
    GEE
    Parameter type
    IRR
    Point estimate
    0.36
    Confidence interval
         level
    95%
         sides
    2-sided
         lower limit
    0.16
         upper limit
    0.79
    Notes
    [12] - The GEE model featuring a negative binomial distribution was used for analysis where the incidence rate was the exponentiated LS mean and the IRR was the exponentiated LS mean difference from placebo. Reported p-value is adjusted for multiplicity.

    Secondary: Number of Participants with at Least One Treatment-emergent Adverse Event (TEAE)

    Close Top of page
    End point title
    Number of Participants with at Least One Treatment-emergent Adverse Event (TEAE)
    End point description
    An adverse event (AE) was defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of the study intervention, whether or not the occurrence was considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE was defined as an AE with an onset that occurred after receiving study drug. Safety Analysis Set included all participants who received at least one dose of study drug. Safety Analysis Set included all participants who received at least one dose of study drug.
    End point type
    Secondary
    End point timeframe
    From first dose of the study drug up to end of the study (up to 3 months)
    End point values
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg and 5 mg TAK-861 7 mg QD
    Number of subjects analysed
    22
    23
    21
    23
    23
    Units: participants
    7
    13
    15
    21
    21
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    From first dose of the study drug up to end of the study (up to 3 months)
    Adverse event reporting additional description
    Safety Analysis Set included all participants who received at least one dose of study drug.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Participants received placebo tablets matching TAK-861, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 0.5 mg BID
    Reporting group description
    Participants received TAK-861 0.5 mg, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 2 mg BID
    Reporting group description
    Participants received TAK-861 2 mg, orally, BID, from Days 1 to 56.

    Reporting group title
    TAK-861 2 mg/5 mg
    Reporting group description
    Participants received TAK-861 2 mg followed by the 5 mg dose, orally, from Days 1 to 56.

    Reporting group title
    TAK-861 7 mg QD
    Reporting group description
    Participants received TAK-861 7 mg, orally, QD, from Days 1 to 56. Placebo was given as the second dose.

    Serious adverse events
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg/5 mg TAK-861 7 mg QD
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    0 / 21 (0.00%)
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Ankle fracture
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    0 / 21 (0.00%)
    1 / 23 (4.35%)
    0 / 23 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Placebo TAK-861 0.5 mg BID TAK-861 2 mg BID TAK-861 2 mg/5 mg TAK-861 7 mg QD
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 22 (22.73%)
    11 / 23 (47.83%)
    15 / 21 (71.43%)
    20 / 23 (86.96%)
    20 / 23 (86.96%)
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    0 / 21 (0.00%)
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    0
    0
    0
    2
    Headache
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 23 (4.35%)
    3 / 21 (14.29%)
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    2
    1
    4
    2
    2
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 23 (8.70%)
    0 / 21 (0.00%)
    0 / 23 (0.00%)
    0 / 23 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    0 / 21 (0.00%)
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    0
    0
    0
    2
    Salivary hypersecretion
         subjects affected / exposed
    1 / 22 (4.55%)
    2 / 23 (8.70%)
    2 / 21 (9.52%)
    6 / 23 (26.09%)
    2 / 23 (8.70%)
         occurrences all number
    1
    2
    2
    6
    2
    Nausea
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    0 / 21 (0.00%)
    0 / 23 (0.00%)
    2 / 23 (8.70%)
         occurrences all number
    0
    0
    0
    0
    2
    Respiratory, thoracic and mediastinal disorders
    Epistaxis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    2 / 21 (9.52%)
    0 / 23 (0.00%)
    0 / 23 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 22 (4.55%)
    5 / 23 (21.74%)
    10 / 21 (47.62%)
    13 / 23 (56.52%)
    15 / 23 (65.22%)
         occurrences all number
    1
    5
    10
    13
    16
    Middle insomnia
         subjects affected / exposed
    0 / 22 (0.00%)
    1 / 23 (4.35%)
    0 / 21 (0.00%)
    3 / 23 (13.04%)
    0 / 23 (0.00%)
         occurrences all number
    0
    1
    0
    4
    0
    Renal and urinary disorders
    Micturition urgency
         subjects affected / exposed
    1 / 22 (4.55%)
    5 / 23 (21.74%)
    4 / 21 (19.05%)
    12 / 23 (52.17%)
    9 / 23 (39.13%)
         occurrences all number
    1
    5
    5
    14
    10
    Pollakiuria
         subjects affected / exposed
    1 / 22 (4.55%)
    3 / 23 (13.04%)
    7 / 21 (33.33%)
    7 / 23 (30.43%)
    12 / 23 (52.17%)
         occurrences all number
    1
    3
    8
    7
    14
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    2 / 21 (9.52%)
    2 / 23 (8.70%)
    2 / 23 (8.70%)
         occurrences all number
    0
    0
    3
    2
    2
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 22 (0.00%)
    0 / 23 (0.00%)
    0 / 21 (0.00%)
    1 / 23 (4.35%)
    2 / 23 (8.70%)
         occurrences all number
    0
    0
    0
    1
    2
    Increased appetite
         subjects affected / exposed
    1 / 22 (4.55%)
    0 / 23 (0.00%)
    2 / 21 (9.52%)
    1 / 23 (4.35%)
    1 / 23 (4.35%)
         occurrences all number
    1
    0
    2
    1
    1

    More information

    Close Top of page

    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
    For support, Contact us.
    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.

    European Medicines Agency © 1995-2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA