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    Clinical Trial Results:
    A phase II, randomized, double-blind, placebo-controlled, parallel group study to evaluate the safety, efficacy, and pharmacodynamics of 52 weeks of treatment with basmisanil in participants aged 2 to 14 years old with Dup15q syndrome followed by a 2-year optional open-label extension

    Summary
    EudraCT number
    2021-003791-13
    Trial protocol
    IT   ES   PT   PL   NL  
    Global end of trial date
    04 Mar 2024

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

    Trial information

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    Trial identification
    Sponsor protocol code
    BP42992
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT05307679
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    F. Hoffmann-La Roche AG
    Sponsor organisation address
    Grenzacherstrasse 124, Basel, Switzerland, 4058
    Public contact
    F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.com
    Scientific contact
    F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 May 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Mar 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the effects of 52 weeks of treatment with basmisanil on core symptom domains of Dup15q syndrome (language and social skills) and daily functioning
    Protection of trial subjects
    All participants were required to sign an Informed Consent Form.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    27 May 2022
    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
    United Kingdom: 1
    Country: Number of subjects enrolled
    United States: 3
    Country: Number of subjects enrolled
    Spain: 3
    Worldwide total number of subjects
    7
    EEA total number of subjects
    3
    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)
    7
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Aged 6-14 years with documented maternal duplication (3 copies) or triplication (4 copies) of the chromosome 15q11.2-q13.1 region including the Prader-Willi/Angelman critical region defined as [BP2-BP3] segment, and a Dup15q syndrome Clinician Global Impression of Severity scale (Dup15q CGI S)overall severity score >/= 4 (at least moderately ill).

    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
    Yes

    Arm title
    Placebo
    Arm description
    Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.
    Arm type
    Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Participants received oral placebo BID on Day 1, then TID on Days 2-365.

    Arm title
    Basmisanil
    Arm description
    Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.
    Arm type
    Experimental

    Investigational medicinal product name
    Basmisanil
    Investigational medicinal product code
    Other name
    RO5186582
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Part 1: Participants received oral basmisanil BID on Day 1, then TID on Days 2-365. Part 2: Participants were to receive oral basmisanil for up to 2 years.

    Number of subjects in period 1
    Placebo Basmisanil
    Started
    2
    5
    Completed
    0
    0
    Not completed
    2
    5
         Study terminated by sponsor or physician decision
    2
    5

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.

    Reporting group title
    Basmisanil
    Reporting group description
    Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.

    Reporting group values
    Placebo Basmisanil Total
    Number of subjects
    2 5 7
    Age categorical
    Units: Subjects
        Children (2-11 years)
    2 5 7
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    8.0 ± 1.4 8.8 ± 2.2 -
    Sex: Female, Male
    The values for male and female participants are reported together due to an unacceptable risk of participant re-identification.
    Units: Participants
        Male and Female
    2 5 7
    Ethnicity (NIH/OMB)
    Participants were not consented on the collection of race and ethnicity data.
    Units: Subjects
        Unknown or Not Reported
    2 5 7
    Race (NIH/OMB)
    Participants were not consented on the collection of race and ethnicity data.
    Units: Subjects
        Unknown or Not Reported
    2 5 7

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.

    Reporting group title
    Basmisanil
    Reporting group description
    Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.

    Primary: Vineland-3 adaptive behavior composite scores

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    End point title
    Vineland-3 adaptive behavior composite scores [1]
    End point description
    The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills. Items are either scored as 2 = Usually, 1 = Sometimes, 0 = Never; or scored as 2 = Yes, 0 = No in the case of items that require a binary response. Lower scores indicate lower adaptive behavior abilities. Only baseline data have been reported. The low n for remaining timepoints (Day 183, Day 365) leads to an unacceptable risk of participant re-identification.
    End point type
    Primary
    End point timeframe
    Baseline, Day 183, Day 365
    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: No formal statistical analyses were performed due to the low number of trial participants.
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    30.50 (30.0 to 31.0)
    36.00 (27.0 to 52.0)
    No statistical analyses for this end point

    Secondary: Vineland-3 gross and fine motor subdomains scores

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    End point title
    Vineland-3 gross and fine motor subdomains scores
    End point description
    The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills. Items are either scored as 2 = Usually, 1 = Sometimes, 0 = Never; or scored as 2 = Yes, 0 = No in the case of items that require a binary response. Lower scores indicate lower adaptive behavior abilities. Only baseline data have been reported. The low n for remaining timepoints (Day 183, Day 365) leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 183, Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    2 [2]
    Units: Units on a scale
    median (full range (min-max))
        Gross Motor V-Scale Score - Baseline
    1.00 (1.0 to 1.0)
    4.50 (1.0 to 8.0)
        Fine Motor V-Scale Score - Baseline
    1.00 (1.0 to 1.0)
    3.00 (1.0 to 5.0)
    Notes
    [2] - Data not reported for participants older than 9 years; normative data only available for ages 0-9y
    No statistical analyses for this end point

    Secondary: Vineland 3 expressive and receptive communication subdomains

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    End point title
    Vineland 3 expressive and receptive communication subdomains
    End point description
    The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills. Items are either scored as 2 = Usually, 1 = Sometimes, 0 = Never; or scored as 2 = Yes, 0 = No in the case of items that require a binary response. Lower scores indicate lower adaptive behavior abilities. Only baseline data have been reported. The low n for remaining timepoints (Day 183, Day 365) leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 183, Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Expressive Communication V-Scale Score - Baseline
    1.00 (1.0 to 1.0)
    1.00 (1.0 to 11.0)
        Receptive Communication V-Scale Score - Baseline
    1.00 (1.0 to 1.0)
    1.00 (1.0 to 6.0)
    No statistical analyses for this end point

    Secondary: Vineland-3 play and leisure time and interpersonal relationships subdomains

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    End point title
    Vineland-3 play and leisure time and interpersonal relationships subdomains
    End point description
    The Vineland-3 is an instrument that measures communication, daily living skills, socialization, and motor skills. Items are either scored as 2 = Usually, 1 = Sometimes, 0 = Never; or scored as 2 = Yes, 0 = No in the case of items that require a binary response. Lower scores indicate lower adaptive behavior abilities. V-scale scores are presented. Only baseline data have been reported. The low n for remaining timepoints (Day 183, Day 365) leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 183, Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Play and Leisure - Baseline
    1.00 (1.0 to 1.0)
    1.00 (1.0 to 9.0)
        Interpersonal Relationships - Baseline
    1.50 (1.0 to 2.0)
    1.00 (1.0 to 9.0)
    No statistical analyses for this end point

    Secondary: Mullen Scales of Early Learning (MSEL) gross and fine motor domains

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    End point title
    Mullen Scales of Early Learning (MSEL) gross and fine motor domains
    End point description
    The MSEL are designed for a certified rater to provide an assessment of cognitive ability and motor development of typically developing children from birth through age 68 months. It was administered to all participants in this study irrespective of their chronological age. The instrument consists of 124 items across five scales measuring Gross Motor, Visual Reception, Fine Motor, Expressive Language, and Receptive Language. The scoring for each item ranges from 0 to 5 points, with lower scores indicating lower developmental abilities. Only baseline data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 183, Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Gross Motor - Baseline
    20.50 (14.0 to 27.0)
    19.00 (13.0 to 35.0)
        Fine Motor - Baseline
    15.50 (13.0 to 18.0)
    15.00 (6.0 to 30.0)
    No statistical analyses for this end point

    Secondary: MSEL visual reception domain scores

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    End point title
    MSEL visual reception domain scores
    End point description
    The MSEL are designed for a certified rater to provide an assessment of cognitive ability and motor development of typically developing children from birth through age 68 months. It was administered to all participants in this study irrespective of their chronological age. The instrument consists of 124 items across five scales measuring Gross Motor, Visual Reception, Fine Motor, Expressive Language, and Receptive Language. The scoring for each item ranges from 0 to 5 points, with lower scores indicating lower developmental abilities. Only baseline data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 183, Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    14.00 (11.0 to 17.0)
    4.00 (2.0 to 42.0)
    No statistical analyses for this end point

    Secondary: MSEL expressive and receptive language subdomains

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    End point title
    MSEL expressive and receptive language subdomains
    End point description
    The MSEL are designed for a certified rater to provide an assessment of cognitive ability and motor development of typically developing children from birth through age 68 months. It was administered to all participants in this study irrespective of their chronological age. The instrument consists of 124 items across five scales measuring Gross Motor, Visual Reception, Fine Motor, Expressive Language, and Receptive Language. The scoring for each item ranges from 0 to 5 points, with lower scores indicating lower developmental abilities. Only baseline data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline, Day 183, Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Expressive Language - Baseline
    12.50 (12.0 to 13.0)
    17.00 (6.0 to 29.0)
        Receptive Language - Baseline
    10.00 (7.0 to 13.0)
    16.00 (9.0 to 28.0)
    No statistical analyses for this end point

    Secondary: Dup15q syndrome Clinician Global Impression of Severity (CGI-S) scale scores

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    End point title
    Dup15q syndrome Clinician Global Impression of Severity (CGI-S) scale scores
    End point description
    The Dup15q CGI-S is a 10-domain clinician-rated measure on a 6-point scale that measures global severity of illness at a given point in time. The ten domains are seizures, expressive communication difficulties, fine motor skills difficulties, gross motor skills difficulties, cognitive/intellectual impairment, impairment in activities of daily living/self-care, socialization, maladaptive behavior, sleep difficulties, and overall severity. Response options are: 1 = none 2 = very mild 3 = mild 4 = moderate 5 = severe 6 = very severe. Only baseline and Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    4.00 (4.0 to 4.0)
    4.00 (4.0 to 6.0)
        Day 28
    4.00 (4.0 to 4.0)
    4.00 (3.0 to 6.0)
    No statistical analyses for this end point

    Secondary: Dup15q syndrome Clinician Global Impression of Change scale (CGI-C) scores

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    End point title
    Dup15q syndrome Clinician Global Impression of Change scale (CGI-C) scores
    End point description
    The Dup15q CGI-C is a 10-domain clinician-rated measure on a 7-point scale that assesses the clinician’s impression of change in illness compared with baseline. The ten domains are seizures, expressive communication difficulties, fine motor skills difficulties, gross motor skills difficulties, cognitive/intellectual impairment, impairment in activities of daily living/self-care, socialization, maladaptive behavior, sleep difficulties, and overall severity. Response options are: 1 = very much improved 2 = much improved 3 = minimally improved 4 = no change 5 = minimally worse 6 = much worse 7= very much worse Only Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Day 28 - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    4
    Units: Units on a scale
    median (full range (min-max))
        Day 28
    3.50 (3.0 to 4.0)
    3.00 (2.0 to 3.0)
    No statistical analyses for this end point

    Secondary: Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) domain scores - Irritability

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    End point title
    Aberrant Behavior Checklist - Second Edition - Community Version (ABC-2-C) domain scores - Irritability
    End point description
    The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). Subscale scores and a total score can be calculated with higher scores indicating greater severity. Only baseline and Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    11.00 (11.0 to 11.0)
    15.00 (3.0 to 40.0)
        Day 28
    10.00 (6.0 to 14.0)
    15.00 (5.0 to 18.0)
    No statistical analyses for this end point

    Secondary: ABC-2-C domain scores - Social Withdrawal

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    End point title
    ABC-2-C domain scores - Social Withdrawal
    End point description
    The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). Subscale scores and a total score can be calculated with higher scores indicating greater severity. Only baseline and Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    14.50 (11.0 to 18.0)
    16.00 (2.0 to 25.0)
        Day 28
    7.50 (6.0 to 9.0)
    14.00 (7.0 to 17.0)
    No statistical analyses for this end point

    Secondary: ABC-2-C domain scores - Stereotypic Behavior

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    End point title
    ABC-2-C domain scores - Stereotypic Behavior
    End point description
    The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). Subscale scores and a total score can be calculated with higher scores indicating greater severity. Only baseline and Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    8.00 (8.0 to 8.0)
    6.00 (0.0 to 15.0)
        Day 28
    5.50 (5.0 to 6.0)
    13.00 (7.0 to 16.0)
    No statistical analyses for this end point

    Secondary: ABC-2-C domain scores - Hyperactivity/Non-Compliance

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    End point title
    ABC-2-C domain scores - Hyperactivity/Non-Compliance
    End point description
    The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). Subscale scores and a total score can be calculated with higher scores indicating greater severity. Only baseline and Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    32.00 (23.0 to 41.0)
    19.00 (13.0 to 38.0)
        Day 28
    12.00 (6.0 to 18.0)
    22.00 (15.0 to 33.0)
    No statistical analyses for this end point

    Secondary: ABC-2-C domain scores - Inappropriate Speech

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    End point title
    ABC-2-C domain scores - Inappropriate Speech
    End point description
    The ABC-2 is an updated, empirically derived, validated 58-item caregiver-completed rating scale that measures the severity of a range of maladaptive behaviors commonly observed in children, adolescents, and adults with intellectual and developmental disabilities. The Community version of the scale (ABC-2-C) will be used. It is designed for use in individuals who are not residing in institutional settings. The checklist assesses symptoms across five domains: irritability, social withdrawal, stereotypic behavior, hyperactive/noncompliance, and inappropriate speech. Items are scored on a 4-point scale from 0 (never) to 3 (severe problem). Subscale scores and a total score can be calculated with higher scores indicating greater severity. Only baseline and Day 28 data have been reported. The low n for remaining timepoints leads to an unacceptable risk of participant re-identification.
    End point type
    Secondary
    End point timeframe
    Baseline - Day 365
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Units on a scale
    median (full range (min-max))
        Baseline
    5.00 (2.0 to 8.0)
    2.00 (1.00 to 4.00)
        Day 28
    1.50 (0.0 to 3.0)
    0.00 (0.0 to 2.0)
    No statistical analyses for this end point

    Secondary: Plasma concentration of basmisanil

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    End point title
    Plasma concentration of basmisanil [3]
    End point description
    Data for certain timepoints (Day 1 Hour 7, Day 1 Hour 8, Day 14 Hour -1, Day 183 Hour 0) has been excluded due to an unacceptable risk of patient re-identification.
    End point type
    Secondary
    End point timeframe
    Day 1 - Day 365
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was specific to the basmisanil arm as the placebo arm did not receive basmisanil.
    End point values
    Basmisanil
    Number of subjects analysed
    5
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 1 - Hour 1.5
    2520 ± 965
        Day 1 - Hour 3.5
    2220 ± 955
        Day 1 - Hour 5.5 (n=4)
    1640 ± 756
        Day 1 - Hour 7.5 (n=4)
    1040 ± 392
        Day 1 - Hour 9 (n=3)
    791 ± 368
        Day 2 - Hour 0
    731 ± 535
        Day 2 - Hour 1.5 (n=4)
    2380 ± 576
        Day 2 - Hour 3.5
    2210 ± 864
        Day 14 - Hour -1.5 (n=2)
    1210 ± 1230
        Day 14 - Hour 0 (n=4)
    902 ± 973
        Day 14 - Hour 1.5 (n=4)
    2480 ± 355
        Day 14 - Hour 3.5
    2160 ± 698
        Day 92 - Hour 0 (n=3)
    896 ± 689
    No statistical analyses for this end point

    Secondary: Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
    End point description
    An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: Count of participants
        AEs and SAEs
    2
    5
    No statistical analyses for this end point

    Secondary: Plasma concentration of the basmisanil metabolite M1

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    End point title
    Plasma concentration of the basmisanil metabolite M1 [4]
    End point description
    Data for certain timepoints (Day 1 Hour 7, Day 1 Hour 8, Day 14 Hour -1, Day 183 Hour 0) has been excluded due to an unacceptable risk of patient re-identification.
    End point type
    Secondary
    End point timeframe
    Day 1 - Day 365
    Notes
    [4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: This endpoint was specific to the basmisanil arm as the placebo arm did not receive basmisanil.
    End point values
    Basmisanil
    Number of subjects analysed
    5
    Units: ng/mL
    arithmetic mean (standard deviation)
        Day 1 - Hour 1.5
    1410 ± 585
        Day 1 - Hour 3.5
    1510 ± 417
        Day 1 - Hour 5.5 (n=4)
    985 ± 335
        Day 1 - Hour 7.5 (n=4)
    675 ± 221
        Day 1 - Hour 9 (n=3)
    535 ± 173
        Day 2 - Hour 0
    664 ± 568
        Day 2 - Hour 1.5 (n=4)
    1330 ± 358
        Day 2 - Hour 3.5
    1410 ± 506
        Day 14 - Hour -1.5 (n=2)
    1400 ± 1410
        Day 14 - Hour 0 (n=4)
    1060 ± 1470
        Day 14 - 1.5 (n=4)
    1510 ± 413
        Day 14 - Hour 3.5
    1250 ± 540
        Day 92 - Hour 0 (n=3)
    648 ± 522
    No statistical analyses for this end point

    Secondary: Quantitative EEG (qEEG) beta-band power

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    End point title
    Quantitative EEG (qEEG) beta-band power
    End point description
    End point type
    Secondary
    End point timeframe
    Baseline, Day 14
    End point values
    Placebo Basmisanil
    Number of subjects analysed
    2
    5
    Units: µV2
    arithmetic mean (standard deviation)
        Baseline
    9.47 ± 0.21
    11.39 ± 1.55
        Day 14
    9.44 ± 0.37
    9.74 ± 1.05
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    Baseline - Day 365
    Adverse event reporting additional description
    All subjects were affected by non-serious adverse events (NSAEs). One subject was affected by serious adverse events (SAEs). There were no deaths. The exact NSAEs and SAEs are not reported due to the risk of patient re-identification.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    Basmisanil
    Reporting group description
    Part 1: Participants received oral basmisanil BID on the first day of treatment, then TID to Day 365. Part 2: Participants who completed Part 1 were to receive oral basmisanil for approximately 2 years.

    Reporting group title
    Placebo
    Reporting group description
    Part 1: Participants received oral placebo twice (BID) on the first day of treatment, then three times daily (TID) to Day 365.

    Serious adverse events
    Basmisanil Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 2 (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
    Basmisanil Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
    0 / 2 (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: All participants reported at least one AE. The exact nature of the AEs has not been disclosed due to the high risk of patient re-identification due to the low number of participants.

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Jan 2022
    Changes to the schedule of activities, including additional assessments; updates to inclusion and exclusion criteria.
    25 Jul 2023
    Addition of optional open-label extension of two years; modification of age criteria; changes to study assessments.

    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.
    Data for certain timepoints with only 1 analyzed participant is not reported due to an unacceptable risk of participant re-identification.
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