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    Clinical Trial Results:
    A Phase 2 open-label, dose-escalation study to evaluate the safety, pharmacokinetics, immunogeniticy and pharmacodynamics/efficacy of EDI200, an EDA-A1 replacement protein, administered to male infants with X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).

    Summary
    EudraCT number
    2012-003561-17
    Trial protocol
    DE   GB   IT   FR  
    Global end of trial date
    17 Mar 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    27 Aug 2016
    First version publication date
    27 Aug 2016
    Other versions
    Summary report(s)
    _Edimer ECP-002 Abbrv. CSR Final_31May2016_Signed wAtt

    Trial information

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    Trial identification
    Sponsor protocol code
    ECP-002
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01775462
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Edimer Pharmaceuticals, Inc.
    Sponsor organisation address
    55 Cambridge Parkway, Suite 102W, Cambridge, United States, MA 02142
    Public contact
    Clinical Trials Information, Edimer Pharmaceuticals, Inc., +1 6177584305, ramsey@edimerpharma.com
    Scientific contact
    Clinical Trials Information, Edimer Pharmaceuticals, Inc., +1 6177584305, ramsey@edimerpharma.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    28 Sep 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    28 Sep 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Mar 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the safety, pharmacokinetics and immunogenicity of EDI200 administered to XLHED-affected neonates
    Protection of trial subjects
    Patients were always under close monitoring in the hospital by specific qualified and trained paediattic trial staff (Investigators and study nurses). All possible steps were taken to prevent the patients from any stress, pain or discomfort.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    03 Jun 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    United States: 3
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Germany: 4
    Worldwide total number of subjects
    10
    EEA total number of subjects
    7
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    10
    Infants and toddlers (28 days-23 months)
    0
    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
    -

    Pre-assignment
    Screening details
    Male neonates documented by genetic diagnosis to carry an EDA mutation associated with XLHED, and their male siblings

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

    Arms
    Arm title
    Treatment
    Arm description
    -
    Arm type
    Experimental

    Investigational medicinal product name
    EDI200
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    3/10/20 mg/kg (5 doses total) mg/kg milligram(s)/kilogram per day

    Number of subjects in period 1
    Treatment
    Started
    10
    Cohort 1
    3 [1]
    Cohort 2
    5 [2]
    Cohort 3
    2 [3]
    Completed
    10
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: 2 Subjects were enrolled in cohort 3 till study completion
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Dose was adjusted after 5 patients completed cohort 2
    [3] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: Dose was adjusted after 3 patients completed cohort 1

    Baseline characteristics

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    End points

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    End points reporting groups
    Reporting group title
    Treatment
    Reporting group description
    -

    Primary: Clinical endpoints

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    End point title
    Clinical endpoints [1]
    End point description
    End point type
    Primary
    End point timeframe
    overall
    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: Proof of concept study to be compared with a parallel natural history study
    End point values
    Treatment
    Number of subjects analysed
    10
    Units: growth and development assessments
    10
    No statistical analyses for this end point

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    16 Sep 2013 - 28 Sep 2015 Time from first patient in till last patient out visit
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    10.0
    Frequency threshold for reporting non-serious adverse events: 0%
    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 serious advers events can be found in the Appendix 16.2.7 of the CSR

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Jun 2013
    Protocol Version 3 (see CSR 9.8 page 15))
    04 Dec 2014
    Protocol AMD 5 (See CSR 9.8 page 16)
    11 Feb 2015
    Protocol AMD 6 (See CSR 9.9 page 15)

    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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