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    Clinical Trial Results:
    A Multicenter, Open-Label Study to Evaluate the Pharmacokinetics, Tolerability, and Safety of a Single Dose of Staccato Alprazolam in Adolescent Study Participants With Epilepsy

    Summary
    EudraCT number
    2022-002523-36
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    05 Apr 2022

    Results information
    Results version number
    v1(current)
    This version publication date
    28 Sep 2022
    First version publication date
    28 Sep 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    UP0100
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04857307
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, 1070
    Public contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Scientific contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-003043-PIP01-21
    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
    05 May 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    05 Apr 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Apr 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • To evaluate the pharmacokinetic (PK) of alprazolam in adolescent study participants with epilepsy following single inhaled dose of Staccato alprazolam • To evaluate the safety and tolerability of Staccato alprazolam in adolescent study participants with epilepsy
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    Background therapy as permitted in the protocol.
    Evidence for comparator
    Not applicable
    Actual start date of recruitment
    28 Apr 2021
    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 States: 14
    Worldwide total number of subjects
    14
    EEA total number of subjects
    0
    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)
    14
    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
    The study started to enroll study participants in April 2021 and concluded in April 2022.

    Pre-assignment
    Screening details
    Participant Flow refers to the Safety Set.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Staccato Alprazolam
    Arm description
    Participants received staccato alprazolam inhalation powder on Day 1. Participants were followed up to Day 9.
    Arm type
    Experimental

    Investigational medicinal product name
    Staccato Alprazolam
    Investigational medicinal product code
    UCB7538
    Other name
    Pharmaceutical forms
    Inhalation powder
    Routes of administration
    Inhalation use
    Dosage and administration details
    Participants received staccato alprazolam on Day 1.

    Number of subjects in period 1
    Staccato Alprazolam
    Started
    14
    Completed
    14

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Staccato Alprazolam
    Reporting group description
    Participants received staccato alprazolam inhalation powder on Day 1. Participants were followed up to Day 9.

    Reporting group values
    Staccato Alprazolam Total
    Number of subjects
    14 14
    Age Categorical
    Units: participants
        <=18 years
    14 14
        Between 18 and 65 years
    0 0
        >=65 years
    0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    15.1 ± 1.9 -
    Sex: Female, Male
    Units: participants
        Female
    12 12
        Male
    2 2

    End points

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    End points reporting groups
    Reporting group title
    Staccato Alprazolam
    Reporting group description
    Participants received staccato alprazolam inhalation powder on Day 1. Participants were followed up to Day 9.

    Primary: Maximum plasma concentration (Cmax) following single inhaled dose of Staccato alprazolam

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    End point title
    Maximum plasma concentration (Cmax) following single inhaled dose of Staccato alprazolam [1]
    End point description
    Cmax was defined as the maximum observed plasma concentration. Pharmacokinetic Set (PKS) included all study participants who received at least 1 dose of IMP and had at least 1 observable PK measurement.
    End point type
    Primary
    End point timeframe
    Plasma samples were taken on Day 1, predose and then at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose
    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 hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Staccato Alprazolam
    Number of subjects analysed
    14
    Units: nanogram/milliliter
        geometric mean (geometric coefficient of variation)
    35.50 ± 57.8
    No statistical analyses for this end point

    Primary: Area under the plasma concentration-time curve from zero to the last quantifiable concentration (AUC(0-t)) following single inhaled dose of Staccato alprazolam

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    End point title
    Area under the plasma concentration-time curve from zero to the last quantifiable concentration (AUC(0-t)) following single inhaled dose of Staccato alprazolam [2]
    End point description
    AUC(0-t) was defined as area under the plasma concentration-time curve from zero to the last quantifiable concentration. Pharmacokinetic Set included all study participants who received at least 1 dose of IMP and had at least 1 observable PK measurement.
    End point type
    Primary
    End point timeframe
    Plasma samples were taken on Day 1, predose and then at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose
    Notes
    [2] - 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 hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Staccato Alprazolam
    Number of subjects analysed
    14
    Units: hour*nanogram/milliliter
        geometric mean (geometric coefficient of variation)
    278.2 ± 36.2
    No statistical analyses for this end point

    Primary: Area under the plasma concentration-time curve from time 0 to infinity (AUC) following single inhaled dose of Staccato alprazolam

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    End point title
    Area under the plasma concentration-time curve from time 0 to infinity (AUC) following single inhaled dose of Staccato alprazolam [3]
    End point description
    AUC was defined as area under the plasma concentration-time curve from time 0 to infinity. Pharmacokinetic Set included all study participants who received at least 1 dose of IMP and had at least 1 observable PK measurement. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
    End point type
    Primary
    End point timeframe
    Plasma samples were taken on Day 1, predose and then at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose
    Notes
    [3] - 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 hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Staccato Alprazolam
    Number of subjects analysed
    12
    Units: hour*nanogram/milliliter
        geometric mean (geometric coefficient of variation)
    280.0 ± 35.8
    No statistical analyses for this end point

    Primary: Apparent total body clearance (CL/F) following single inhaled dose of Staccato alprazolam

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    End point title
    Apparent total body clearance (CL/F) following single inhaled dose of Staccato alprazolam [4]
    End point description
    CL/F was defined as apparent total body clearance. Pharmacokinetic Set included all study participants who received at least 1 dose of IMP and had at least 1 observable PK measurement. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
    End point type
    Primary
    End point timeframe
    Plasma samples were taken on Day 1, predose and then at 2 minutes, 10 minutes, 30 minutes, 1 hour, 2 hours, 6 hours, 24 hours, and 36 hours postdose
    Notes
    [4] - 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 hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Staccato Alprazolam
    Number of subjects analysed
    12
    Units: Liter/hour
        geometric mean (geometric coefficient of variation)
    7.144 ± 35.8
    No statistical analyses for this end point

    Primary: Percentage of participants with treatment-emergent adverse events (TEAEs)

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    End point title
    Percentage of participants with treatment-emergent adverse events (TEAEs) [5]
    End point description
    A TEAE was defined as any AE with a start date/time on or after the dose of treatment or any unresolved event already present before administration of treatment that worsened in intensity following exposure to the treatment. Safety Set consisted of all study participants who received at least 1 dose of IMP.
    End point type
    Primary
    End point timeframe
    From Baseline (Day 1) till end of Safety Follow-up (up to Day 9)
    Notes
    [5] - 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 hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Staccato Alprazolam
    Number of subjects analysed
    14
    Units: percentage of participants
        number (not applicable)
    21.4
    No statistical analyses for this end point

    Primary: Percentage of participants with serious treatment-emergent adverse events (serious TEAEs)

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    End point title
    Percentage of participants with serious treatment-emergent adverse events (serious TEAEs) [6]
    End point description
    A TEAE was defined as any AE with a start date/time on or after the dose of treatment or any unresolved event already present before administration of treatment that worsened in intensity following exposure to the treatment. A serious adverse event (SAE) was defined as any untoward medical occurrence that, at any dose: a. Resulted in death, b. Is life-threatening, c. Required inpatient hospitalization or prolongation of existing hospitalization, d. Resulted in persistent disability/incapacity, e. Is a congenital anomaly/birth defect, f. Important medical events. Safety Set consisted of all study participants who received at least 1 dose of IMP.
    End point type
    Primary
    End point timeframe
    From Baseline (Day 1) till end of Safety Follow-up (up to Day 9)
    Notes
    [6] - 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 hypothesis testing was planned for this study. Results were summarized in tables as descriptive statistics only.
    End point values
    Staccato Alprazolam
    Number of subjects analysed
    14
    Units: percentage of participants
        number (not applicable)
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline (Day 1) till end of Safety Follow-up (up to Day 9)
    Adverse event reporting additional description
    A TEAE was defined as any AE with a start date/time on or after the dose of treatment or any unresolved event already present before administration of treatment that worsened in intensity following exposure to the treatment. Safety Set was analyzed for TEAEs.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Staccato Alprazolam
    Reporting group description
    Participants received staccato alprazolam inhalation powder on Day 1. Participants were followed up to Day 9.

    Serious adverse events
    Staccato Alprazolam
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 14 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Staccato Alprazolam
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 14 (21.43%)
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Hiccups
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Nervous system disorders
    Dysgeusia
         subjects affected / exposed
    3 / 14 (21.43%)
         occurrences all number
    3
    Somnolence
         subjects affected / exposed
    3 / 14 (21.43%)
         occurrences all number
    4
    Dizziness
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Jan 2022
    Protocol Amendment 3, dated 12 Jan 2022, was a substantial amendment that was implemented after 6 participants had been enrolled in the study. The main purpose of this amendment was to decrease the burden on study participants, their families, and clinical sites by allowing study participants to leave the clinic after the 6-hour postdose safety assessments and PK blood sampling and return to the clinic for the 24- and 36-hour postdose assessments, at the discretion of the Investigator. This change in the study conduct was supported by results from the first set of study participants (N=6, including at least 2 adolescent participants with body weight <50kg) enrolled in UP0100. Careful review of these results by the study safety monitoring committee (SMC) did not reveal any safety concerns that would preclude discharge of UP0100 study participants after the 6-hour postdose assessments. Edits were made to clarify removal of the 12-hour time point for assessment of vital signs, peripheral oxygen saturation (SpO2), and sedation/sleepiness (by visual analog scale [VAS]) from the Schedule of Assessments and other applicable sections of the protocol.

    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.
    The PK endpoint measures of geometric mean are presented with percentage geometric coefficients of variation.
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