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    Clinical Trial Results:
    A Multicenter, Open, Sequential Dose-Escalation Study to Investigate the Safety, Tolerability, and Pharmacokinetics of 2 Separate Doses of Caspofungin Acetate in Children Between the Ages of 3 to 24 Months With New Onset Fever and Neutropenia

    Summary
    EudraCT number
    2014-005030-54
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    31 Jul 2006

    Results information
    Results version number
    v1(current)
    This version publication date
    10 Feb 2016
    First version publication date
    15 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    MK-0991-042
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00292071
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Merck Sharp & Dohme Corp.
    Sponsor organisation address
    2000 Galloping Hill Road, Kenilworth, NJ, United States, 07033
    Public contact
    Clinical Trials Disclosure, Merck Sharp & Dohme Corp., ClinicalTrialsDisclosure@merck.com
    Scientific contact
    Clinical Trials Disclosure, Merck Sharp & Dohme Corp., ClinicalTrialsDisclosure@merck.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-000010-PIP01-07
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    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
    31 Jul 2006
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Jul 2006
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Jul 2006
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    This study was an open-label, multicenter study to evaluate the safety, tolerability, and pharmacokinetics of caspofungin in clinically stable children with fever and neutropenia between the ages of 3 to 24 months.
    Protection of trial subjects
    This study was conducted in conformance with Good Clinical Practice standards and applicable country and/or local statutes and regulations regarding ethical committee review, informed consent, and the protection of human subjects participating in biomedical research. Caspofungin was to be administered daily until the participant recovered from the neutropenic episode. However, if the participant remained febrile and neutropenic after 4 days of therapy, caspofungin was to be discontinued and the participant was to be started thereafter on a standard empirical antifungal regimen with intravenous amphotericin B (either conventional deoxycholate or a lipid formulation). Similarly, if the participant developed a proven or probable breakthrough invasive fungal infection, caspofungin was to be discontinued and an intravenous (IV) formulation of amphotericin B was to be administered.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    15 Apr 2005
    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: 9
    Worldwide total number of subjects
    9
    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)
    9
    Children (2-11 years)
    0
    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
    The study enrolled clinically stable, immunocompromised children between the ages of 3 and 24 months with a history of underlying hematological or solid organ malignancies, hematopoietic stem cell transplantation (including bone marrow or peripheral stem cell transplantation), or aplastic anemia, and new onset fever and neutropenia.

    Pre-assignment
    Screening details
    Nine participants were screened and 9 were enrolled in the study. Only the caspofungin 50 mg/m^2/day regimen was enrolled. Due to enrollment difficulties the study was halted prior to enrollment of any participants in the caspofungin 70 mg/m^2/day regimen.

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

    Arms
    Arm title
    Caspofungin 50 mg/m^2/day
    Arm description
    Participants received caspofungin 50 mg/m^2/day in a 1-hour intravenous infusion for a minimum of 4 days and a maximum of 28 days.
    Arm type
    Experimental

    Investigational medicinal product name
    Caspofungin
    Investigational medicinal product code
    Other name
    CANCIDAS™, MK-0991
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Caspofungin acetate 50 mg/m^2/day in a 1-hour intravenous infusion for a minimum of 4 days and a maximum of 28 days. Infusion employed a pediatric syringe or ambulatory pump.

    Number of subjects in period 1
    Caspofungin 50 mg/m^2/day
    Started
    9
    Completed
    8
    Not completed
    1
         Adverse event, serious fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Caspofungin 50 mg/m^2/day
    Reporting group description
    Participants received caspofungin 50 mg/m^2/day in a 1-hour intravenous infusion for a minimum of 4 days and a maximum of 28 days.

    Reporting group values
    Caspofungin 50 mg/m^2/day Total
    Number of subjects
    9 9
    Age categorical
    Units: Subjects
        7 to 12 months
    4 4
        13 to 18 months
    3 3
        19 to 24 months
    2 2
    Age continuous
    Units: months
        arithmetic mean (standard deviation)
    15 ± 4.2 -
    Gender categorical
    Units: Subjects
        Female
    3 3
        Male
    6 6

    End points

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    End points reporting groups
    Reporting group title
    Caspofungin 50 mg/m^2/day
    Reporting group description
    Participants received caspofungin 50 mg/m^2/day in a 1-hour intravenous infusion for a minimum of 4 days and a maximum of 28 days.

    Primary: Area Under the Plasma Concentration Curve of Caspofungin up to 24 Hours (AUC0-24) on Day 1

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    End point title
    Area Under the Plasma Concentration Curve of Caspofungin up to 24 Hours (AUC0-24) on Day 1 [1]
    End point description
    Plasma caspofungin concentrations were measured with a high-performance liquid chromatography method.
    End point type
    Primary
    End point timeframe
    Plasma samples for measurement of caspofungin concentrations were collected on Day 1 before dosing and at 1, 2, 4, 8, 12, and 24 hours after initiation of the 1-hour infusion.
    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: This was a single-arm study; therefore, no statistical analysis was performed for this endpoint.
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    7 [2]
    Units: µg*hr/mL
        least squares mean (confidence interval 95%)
    120.2 (100.93 to 143.15)
    Notes
    [2] - Participants who received the Day 1 infusion and had sufficient plasma concentration data
    No statistical analyses for this end point

    Secondary: Plasma Concentration of Caspofungin at One Hour (C1hr) on Day 1

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    End point title
    Plasma Concentration of Caspofungin at One Hour (C1hr) on Day 1
    End point description
    Plasma caspofungin concentrations were measured with a high-performance liquid chromatography method.
    End point type
    Secondary
    End point timeframe
    Plasma samples for measurement of caspofungin concentrations were collected on Day 1 before dosing and at 1, 2, 4, 8, 12, and 24 hours after initiation of the 1-hour infusion.
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    9 [3]
    Units: µg/mL
        least squares mean (confidence interval 95%)
    17.46 (14.75 to 20.68)
    Notes
    [3] - Participants who received the Day 1 infusion and had sufficient plasma concentration data
    No statistical analyses for this end point

    Secondary: Plasma Concentration of Caspofungin at 24 Hours (C24hr) on Day 1

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    End point title
    Plasma Concentration of Caspofungin at 24 Hours (C24hr) on Day 1
    End point description
    Plasma caspofungin concentrations were measured with a high-performance liquid chromatography method.
    End point type
    Secondary
    End point timeframe
    Plasma samples for measurement of caspofungin concentrations were collected on Day 1 before dosing and at 1, 2, 4, 8, 12, and 24 hours after initiation of the 1-hour infusion.
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    7 [4]
    Units: µg/mL
        least squares mean (confidence interval 95%)
    1.34 (1.03 to 1.76)
    Notes
    [4] - Participants who received the Day 1 infusion and had sufficient plasma concentration data
    No statistical analyses for this end point

    Secondary: Area Under the Plasma Concentration Curve of Caspofungin up to 24 Hours (AUC0-24) on Day 3 to 14

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    End point title
    Area Under the Plasma Concentration Curve of Caspofungin up to 24 Hours (AUC0-24) on Day 3 to 14
    End point description
    Plasma caspofungin concentrations were measured with a high-performance liquid chromatography method. Values reported are for Day 4 or time-average geometric mean of all values obtained between Day 3 and 14.
    End point type
    Secondary
    End point timeframe
    Plasma samples for measurement of caspofungin concentrations were collected on Day 3 through 14 before dosing and at 1, 2, 4, 8, 12, and 24 hours after initiation of the daily 1-hour infusion.
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    8 [5]
    Units: µg*hr/mL
        least squares mean (confidence interval 95%)
    130.29 (107.46 to 157.96)
    Notes
    [5] - Participants who received >=1 infusion on Days 3 - 14 and had sufficient plasma concentration data
    No statistical analyses for this end point

    Secondary: Plasma Concentration of Caspofungin at One Hour (C1hr) on Day 3 to 14

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    End point title
    Plasma Concentration of Caspofungin at One Hour (C1hr) on Day 3 to 14
    End point description
    Plasma caspofungin concentrations were measured with a high-performance liquid chromatography method. Values reported are for Day 4 or time-average geometric mean of all values obtained between Day 3 and 14.
    End point type
    Secondary
    End point timeframe
    Plasma samples for measurement of caspofungin concentrations were collected on Day 3 through 14 before dosing and at 1, 2, 4, 8, 12, and 24 hours after initiation of the daily 1-hour infusion.
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    8 [6]
    Units: µg/mL
        least squares mean (confidence interval 95%)
    17.21 (14.56 to 20.36)
    Notes
    [6] - Participants who received >=1 infusion on Days 3 - 14 and had sufficient plasma concentration data
    No statistical analyses for this end point

    Secondary: Plasma Concentration of Caspofungin at 24 Hours (C24hr) on Day 3 to 14

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    End point title
    Plasma Concentration of Caspofungin at 24 Hours (C24hr) on Day 3 to 14
    End point description
    Plasma caspofungin concentrations were measured with a high-performance liquid chromatography method. Values reported are for Day 4 or time-average geometric mean of all values obtained between Day 3 and 14.
    End point type
    Secondary
    End point timeframe
    Plasma samples for measurement of caspofungin concentrations were collected on Day 3 through 14 before dosing and at 1, 2, 4, 8, 12, and 24 hours after initiation of the daily 1-hour infusion.
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    8 [7]
    Units: µg/mL
        least squares mean (confidence interval 95%)
    1.64 (1.24 to 2.16)
    Notes
    [7] - Participants who received >=1 infusion on Days 3 - 14 and had sufficient plasma concentration data
    No statistical analyses for this end point

    Secondary: Percentage of Participants with One or More Adverse Events

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    End point title
    Percentage of Participants with One or More Adverse Events
    End point description
    An adverse experience is defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the study drug, whether or not considered related to the use of the product. Any worsening of a preexisting condition which is temporally associated with the use of the product, is also an adverse experience.
    End point type
    Secondary
    End point timeframe
    Up to 14 days after the last dose of study drug
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    9
    Units: Percentage of participants
    number (not applicable)
        Clinical Adverse Events
    77.8
        Laboratory Adverse Events
    55.6
    No statistical analyses for this end point

    Secondary: Percentage of Participants Discontinued Due to an Adverse Event

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    End point title
    Percentage of Participants Discontinued Due to an Adverse Event
    End point description
    An adverse experience is defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the study drug, whether or not considered related to the use of the product. Any worsening of a preexisting condition which is temporally associated with the use of the product, is also an adverse experience.
    End point type
    Secondary
    End point timeframe
    Up to 14 days after the last dose of study drug
    End point values
    Caspofungin 50 mg/m^2/day
    Number of subjects analysed
    9
    Units: Percentage of participants
    number (not applicable)
        Clinical Adverse Event
    0
        Laboratory Adverse Event
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 14 days after the last dose of study drug
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    10.0
    Reporting groups
    Reporting group title
    Caspofungin 50 mg/m^2/day
    Reporting group description
    Participants received caspofungin 50 mg/m^2/day in a 1-hour intravenous infusion for a minimum of 4 days and a maximum of 28 days.

    Serious adverse events
    Caspofungin 50 mg/m^2/day
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 9 (22.22%)
         number of deaths (all causes)
    1
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Drug prescribing error
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pneumonitis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory distress
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Pneumonia cytomegaloviral
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Caspofungin 50 mg/m^2/day
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    8 / 9 (88.89%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hypotension
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Immune system disorders
    Acute graft versus host disease in skin
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Psychiatric disorders
    Agitation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    4
    Aspartate aminotransferase increased
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Blood albumin decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Blood calcium decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood glucose increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    2
    Blood magnesium decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood phosphorus decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood potassium decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood sodium increased
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    3
    Blood uric acid decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    3
    Haemoglobin decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Platelet count decreased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Prothrombin time prolonged
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    4
    White blood cell count increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    6
    Cardiac disorders
    Sinus tachycardia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Respiratory distress
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Tachypnoea
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Nervous system disorders
    Hydrocephalus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Intracranial pressure increased
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    2 / 9 (22.22%)
         occurrences all number
    2
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Lip blister
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Mouth haemorrhage
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Mouth plaque
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Rectal haemorrhage
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Hepatobiliary disorders
    Hepatosplenomegaly
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Renal and urinary disorders
    Acute prerenal failure
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Dermatitis diaper
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Pruritus
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Rash maculo-papular
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Rash papular
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Skin disorder
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Metabolic acidosis
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Infections and infestations
    Candida nappy rash
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Central line infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1
    Fungal infection
         subjects affected / exposed
    1 / 9 (11.11%)
         occurrences all number
    1

    More information

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    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
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