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    Clinical Trial Results:
    A PROSPECTIVE, OPEN-LABEL, NON-COMPARATIVE STUDY TO ASSESS THE SAFETY, TOLERABILITY AND EFFICACY OF VORICONAZOLE FOR THE PRIMARY AND SALVAGE TREATMENT OF INVASIVE CANDIDIASIS, CANDIDEMIA, AND ESOPHAGEAL CANDIDIASIS IN PEDIATRIC SUBJECTS

    Summary
    EudraCT number
    2009-012848-16
    Trial protocol
    DE   HU   SK   CZ   BG   RO   Outside EU/EEA  
    Global end of trial date
    08 Jul 2013

    Results information
    Results version number
    v2(current)
    This version publication date
    02 Jun 2016
    First version publication date
    01 Aug 2015
    Other versions
    v1
    Version creation reason
    • Correction of full data set
    Reporting periods and duplicate Adverse Events in their data.

    Trial information

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    Trial identification
    Sponsor protocol code
    A1501085
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01092832
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Pfizer Inc.
    Sponsor organisation address
    235 E 42nd Street, New York, United States, NY 10017
    Public contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.com
    Scientific contact
    Pfizer ClinicalTrials.gov Call Center, Pfizer Inc., 001 800-718-1021, ClinicalTrials.gov_Inquiries@pfizer.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
    12 Aug 2014
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Jul 2013
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the safety and tolerability of voriconazole for the treatment of invasive candidiasis, including candidemia, and esophageal candidiasis in pediatric subjects 2 to less than (<)18 years of age.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    28 Oct 2010
    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
    Slovakia: 1
    Country: Number of subjects enrolled
    China: 2
    Country: Number of subjects enrolled
    Czech Republic: 7
    Country: Number of subjects enrolled
    Hong Kong: 3
    Country: Number of subjects enrolled
    Hungary: 2
    Country: Number of subjects enrolled
    Mexico: 5
    Country: Number of subjects enrolled
    Philippines: 1
    Country: Number of subjects enrolled
    Poland: 1
    Worldwide total number of subjects
    22
    EEA total number of subjects
    11
    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)
    14
    Adolescents (12-17 years)
    8
    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
    Total 22 subjects were treated from 11 centers across 8 countries. 21 subjects completed the study and 1 subject discontinued from the study, reason not specified.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Voriconazole: 2 to <12 years
    Arm description
    Subjects aged 2 to <12 years with invasive candidiasis/candidemia (ICC) received a loading dose of voriconazole, every 12 hours (q12h) for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 5 days. Subjects with esophageal candidiasis (EC) received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to oral (PO) therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
    Arm type
    Experimental

    Investigational medicinal product name
    Voriconazole
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects with ICC received a loading dose of voriconazole 9 milligram per kilogram (mg/kg), intravenously (IV), q12h for the first 24 hours, followed by maintenance dosing of voriconazole 8 mg/kg, IV, q12h for a minimum of 5 days of IV therapy. Subjects with EC received voriconazole 4 mg/kg, IV, q12h for a minimum of 5 days of IV therapy.

    Investigational medicinal product name
    Voriconazole
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in both ICC and EC, once signs and symptoms of Candida infection had resolved and was clinically stable received voriconazole (either oral suspension or tablets) 9 mg/kg, q12h (maximum dose of 350 mg).

    Arm title
    Voriconazole: 12 to <18 years
    Arm description
    Subjects aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole, q12h for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 7 days. Subjects with EC received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to PO therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.
    Arm type
    Experimental

    Investigational medicinal product name
    Voriconazole
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Subjects with ICC received a loading dose of voriconazole 6 mg/kg, IV, q12h for the first 24 hours, followed by maintenance dosing of voriconazole 4 mg/kg, IV, q12h for a minimum of 7 days of IV therapy. Subjects with EC received voriconazole 3 mg/kg, IV, q12h for a minimum of 5 days of IV therapy.

    Investigational medicinal product name
    Voriconazole
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for oral suspension, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects in both ICC and EC, once signs and symptoms of Candida infection had resolved and was clinically stable received voriconazole (either oral suspension or tablets) 200 mg, q12h.

    Number of subjects in period 1
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Started
    14
    8
    Completed
    13
    8
    Not completed
    1
    0
         Not specified
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Voriconazole: 2 to <12 years
    Reporting group description
    Subjects aged 2 to <12 years with invasive candidiasis/candidemia (ICC) received a loading dose of voriconazole, every 12 hours (q12h) for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 5 days. Subjects with esophageal candidiasis (EC) received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to oral (PO) therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.

    Reporting group title
    Voriconazole: 12 to <18 years
    Reporting group description
    Subjects aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole, q12h for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 7 days. Subjects with EC received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to PO therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.

    Reporting group values
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years Total
    Number of subjects
    14 8 22
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    6.8 ± 2.9 14.4 ± 1.7 -
    Gender categorical
    Units: Subjects
        Female
    8 6 14
        Male
    6 2 8

    End points

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    End points reporting groups
    Reporting group title
    Voriconazole: 2 to <12 years
    Reporting group description
    Subjects aged 2 to <12 years with invasive candidiasis/candidemia (ICC) received a loading dose of voriconazole, every 12 hours (q12h) for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 5 days. Subjects with esophageal candidiasis (EC) received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to oral (PO) therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.

    Reporting group title
    Voriconazole: 12 to <18 years
    Reporting group description
    Subjects aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole, q12h for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 7 days. Subjects with EC received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to PO therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.

    Primary: Percentage of Subjects With Adverse Events - Overall Summary

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    End point title
    Percentage of Subjects With Adverse Events - Overall Summary [1]
    End point description
    Percentage of subjects with adverse events (AEs), serious adverse events (SAEs), severe AEs, who discontinued due to AEs, or who had dose reduced or temporarily discontinued due to AEs. Safety population.
    End point type
    Primary
    End point timeframe
    Baseline up to 1 month follow-up
    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: Only descriptive statistics was planned to be reported for this endpoint.
    End point values
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Number of subjects analysed
    14
    8
    Units: percentage of subjects
    number (not applicable)
        With AEs
    92.9
    75
        With SAEs
    14.3
    12.5
        With severe AEs
    28.6
    37.5
        Discontinued due to AEs
    14.3
    25
        Dose reduced/temporary discontinuation due to AE
    21.4
    0
    No statistical analyses for this end point

    Secondary: Percentage of Subjects With a Global Response of Success at End of Treatment (EOT)

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    End point title
    Percentage of Subjects With a Global Response of Success at End of Treatment (EOT)
    End point description
    Global response was determined programmatically based on investigator assessment of clinical and microbiological response. Global response of success was defined as clinical cure or improvement and microbiological eradication or presumed eradication. Exact 95 percent (%) confidence interval for binomial proportions using Clopper-Pearson method. Modified Intent-to-Treat (MITT) Population: all subjects who received at least 1 dose of study medication and who have confirmed ICC, EC or subjects with EC who do not have confirmation of EC by esophagoscopy, but who had at least confirmation of oropharyngeal candidiasis.
    End point type
    Secondary
    End point timeframe
    EOT (upto Day 42)
    End point values
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Number of subjects analysed
    9
    8
    Units: percentage of subjects
        number (confidence interval 95%)
    88.9 (51.75 to 99.72)
    62.5 (24.49 to 91.48)
    No statistical analyses for this end point

    Secondary: All-Cause Mortality - Number of Subject Deaths

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    End point title
    All-Cause Mortality - Number of Subject Deaths
    End point description
    Safety population.
    End point type
    Secondary
    End point timeframe
    Day 28 and 1 Month Follow-up
    End point values
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Number of subjects analysed
    14
    8
    Units: subjects
    number (not applicable)
        Day 28
    0
    0
        1 Month Follow-up
    0
    0
    No statistical analyses for this end point

    Secondary: Time to Death

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    End point title
    Time to Death
    End point description
    No subject died within the safety reporting period, therefore time to death was not applicable.
    End point type
    Secondary
    End point timeframe
    Baseline up to 1 month follow-up
    End point values
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: months
        median (full range (min-max))
    ( to )
    ( to )
    Notes
    [2] - No subject died within the safety reporting period.
    [3] - No subject died within the safety reporting period.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Day 49 (7 days after the last dose of study drug)
    Adverse event reporting additional description
    The same event may appear as both an adverse event (AE) and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study.
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.0
    Reporting groups
    Reporting group title
    Voriconazole: 2 to <12 years
    Reporting group description
    Subjects aged 2 to <12 years with invasive candidiasis/candidemia (ICC) received a loading dose of voriconazole, every 12 hours (q12h) for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 5 days. Subjects with esophageal candidiasis (EC) received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to oral (PO) therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.

    Reporting group title
    Voriconazole: 12 to <18 years
    Reporting group description
    Subjects aged 12 to <18 years (excluding those aged 12-14 years weighing <50 kg) with ICC received a loading dose of voriconazole, q12h for the first 24 hours, followed by maintenance dose of voriconazole, q12h for a minimum of 7 days. Subjects with EC received voriconazole, q12h for a minimum of 5 days. In both ICC and EC, once signs and symptoms of Candida infection had resolved and the subject was clinically stable, subjects were switched to PO therapy and received voriconazole, q12h. Voriconazole was administered for at least 7 days (subjects with EC) or 14 days (subjects with ICC) after last positive blood culture up to a maximum of 42 days of treatment.

    Serious adverse events
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 8 (12.50%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Pneumonia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Febrile neutropenia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Splenic candidiasis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 2%
    Non-serious adverse events
    Voriconazole: 2 to <12 years Voriconazole: 12 to <18 years
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    13 / 14 (92.86%)
    6 / 8 (75.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    3
    0
    Phlebitis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Venoocclusive disease
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Device occlusion
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Hypothermia
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 8 (12.50%)
         occurrences all number
    3
    2
    Pyrexia
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 8 (12.50%)
         occurrences all number
    2
    1
    Reproductive system and breast disorders
    Testicular mass
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Drug dose omission
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Incision site pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Incorrect drug administration rate
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Refractoriness to platelet transfusion
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Transplant failure
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Underdose
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Alanine aminotransferase abnormal
         subjects affected / exposed
    3 / 14 (21.43%)
    0 / 8 (0.00%)
         occurrences all number
    3
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Aspartate aminotransferase abnormal
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Blood alkaline phosphatase abnormal
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Blood triglycerides increased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Drug level decreased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Gamma-glutamyltransferase abnormal
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Haematocrit abnormal
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Lymphocyte percentage abnormal
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Monocyte count abnormal
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Staphylococcus test positive
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Cardiac disorders
    Pericardial effusion
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Tachycardia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Atelectasis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Bronchospasm
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Cough
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Haemoptysis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Hydrothorax
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypoventilation
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Pharyngeal erythema
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Pneumothorax
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Respiratory disorder
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Epistaxis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    3
    0
    Hypothrombinaemia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Leukocytosis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Leukopenia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Neutropenia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Platelet disorder
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Thrombocytopenia
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    Nervous system disorders
    Paraesthesia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Eye disorders
    Amaurosis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Conjunctivitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Corneal opacity
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Eye pruritus
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Eyelid disorder
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Photophobia
         subjects affected / exposed
    2 / 14 (14.29%)
    1 / 8 (12.50%)
         occurrences all number
    2
    2
    Retinal disorder
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Visual acuity reduced
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Ascites
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    Nausea
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Oesophagitis
         subjects affected / exposed
    0 / 14 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    0
    2
    Tongue ulceration
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Vomiting
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    Renal and urinary disorders
    Cystitis haemorrhagic
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Haematuria
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hepatobiliary disorders
    Gallbladder disorder
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hepatosplenomegaly
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hyperbilirubinaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Jaundice
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Liver disorder
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    Skin and subcutaneous tissue disorders
    Dermatitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Dermatosis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Purpura
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Rash
         subjects affected / exposed
    3 / 14 (21.43%)
    0 / 8 (0.00%)
         occurrences all number
    3
    0
    Scab
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Musculoskeletal chest pain
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Metabolism and nutrition disorders
    Hyperglycaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    Hyperkalaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypermagnesaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypertriglyceridaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypoalbuminaemia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Hypocalcaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypochloraemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypoglycaemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypokalaemia
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 8 (0.00%)
         occurrences all number
    4
    0
    Hyponatraemia
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Hypophagia
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Hypophosphataemia
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 8 (12.50%)
         occurrences all number
    2
    1
    Infections and infestations
    Anorectal cellulitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Bone abscess
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Bronchopulmonary aspergillosis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    Cellulitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Oral herpes
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Rhinitis
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    Splenic candidiasis
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Nov 2009
    The primary reason for creation of this protocol amendment was to include additional visual safety assessments, as requested by the United States Food and Drug Administration (FDA). 1- Dilated fundoscopic examination at the EOT and at the 1-month follow-up visit. These assessments were in addition to the dilated fundoscopic examination already required at the time of Screening. 2- Confrontational and/or automated visual field test at the time of Screening and at the EOT in subjects 5 years of age and older. For subjects who were critically ill and/or clinically unstable at Baseline, visual field testing could be performed at a later time, when, in the investigator’s judgment, the subject was clinically stable and able to perform the test adequately. 3- In subjects 3 years of age and younger, visual safety monitoring was limited to visual acuity and dilated fundoscopy. In children who were too young to perform the visual acuity test, fixation should have been assessed as to whether it is central, steady or maintained in each eye. 4- Assessment of vital signs, and signs and symptoms of Candida infection (including radiologic findings) every 3 days was no longer required in subjects who have been discharged from the hospital and are continuing to receive study drug treatment on an outpatient basis. 5- In subjects who experience a treatment-emergent cardiac arrhythmia that was felt to be, in the investigator’s judgment, clinically significant, the investigator was to collect a standard 12-lead electro cardiaography (ECG) at the time of the event, or within 2 hours following the event, and send a duplicate of the ECG to the designated central ECG reader.
    08 Jul 2010
    Regarding the primary and salvage EC inclusion criteria: In addition to symptoms consistent with EC, subjects who could not undergo esophagoscopy due to neutropenia, thrombocytopenia, or HIV/advanced AIDS, were to have culture-proven oropharyngeal candidiasis in order to qualify for study entry.

    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 study was prematurely terminated due to slow enrollment. The study was not terminated due to safety issues or concerns. Interpretation of the data are limited due to the small sample size and descriptive design.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
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