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    Clinical Trial Results:
    A Multi-center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein after Maximum Use, Topical Administration of CCP-020 (Diacerein 1% ointment) to Patients with Epidermolysis Bullosa (EB)

    Summary
    EudraCT number
    		 2018-000439-29
    Trial protocol
    		 GB   NL  
    Global end of trial date
    14 Feb 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    06 Feb 2021
    First version publication date
    06 Feb 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CCP-020-101
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03472287
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 IND: 131,384
    Sponsors
    Sponsor organisation name
    Castle Creek Pharmaceuticals, LLC
    Sponsor organisation address
    233 Mt. Airy Road, Basking Ridge, United States, 07920
    Public contact
    Dr. Mary Spellman, Castle Creek Pharmaceuticals, LLC., 001 8622860400, mspellman@castlecreekpharma.com
    Scientific contact
    Dr. Mary Spellman, Castle Creek Pharmaceuticals, LLC., 001 8622860400, mspellman@castlecreekpharma.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
    04 Sep 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    14 Feb 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Feb 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study is to characterize the single-dose and steady- state pharmacokinetics (PK) of diacerein and its active metabolite, rhein, after topical application of CCP-020 (diacerein 1% ointment) under maximum use conditions in adult, adolescent and in infants/children with EB.
    Protection of trial subjects
    The study was performed in accordance with ethical principles that have their origin in the Declaration of Helsinki and are consistent with the applicable International Council for Harmonisation (ICH)/Good Clinical Practice (GCP) regulatory requirements
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Apr 2018
    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: 11
    Worldwide total number of subjects
    11
    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)
    3
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    6
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Patients were recruited in the United States by clinical study centers, following site initiation times but no earlier than April 2018.

    Pre-assignment
    Screening details
    		 Subjects were screened for inclusion and exclusion criteria at Visit 1. Subjects must have had a documented genetic mutation consistent with EB or via a blood or saliva genetic assessment as part of the study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 (Adolescents, Adults)
    Arm description
    		 Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Diacerein
    Investigational medicinal product code
    CCP-020
    Other name
    Pharmaceutical forms
    Ointment
    Routes of administration
    Topical use
    Dosage and administration details
    During the 10-day treatment period, study drug was administered once daily to the defined application area(s). The subject/caregiver or study staff applied a sufficient quantity of study drug to cover EB lesions and uninvolved skin within the application area(s) with a thin layer and gently rubbed it in.

    Arm title
    		 Cohort 2 (Children)
    Arm description
    		 Children with EB (ages 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Diacerein
    Investigational medicinal product code
    CCP-020
    Other name
    Pharmaceutical forms
    Ointment
    Routes of administration
    Topical use
    Dosage and administration details
    During the 10-day treatment period, study drug was administered once daily to the defined application area(s). The subject/caregiver or study staff applied a sufficient quantity of study drug to cover EB lesions and uninvolved skin within the application area(s) with a thin layer and gently rubbed it in.

    Number of subjects in period 1
    		Cohort 1 (Adolescents, Adults) Cohort 2 (Children)
    Started
    8
    3
    Completed
    8
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 (Adolescents, Adults)
    Reporting group description
    		 Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.

    Reporting group title
    Cohort 2 (Children)
    Reporting group description
    		 Children with EB (ages 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.

    Reporting group values
    		 Cohort 1 (Adolescents, Adults) Cohort 2 (Children) Total
    Number of subjects
    		 8 3 11
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0
        Newborns (0-27 days)
    		 0
        Infants and toddlers (28 days-23 months)
    		 0
        Children (2-11 years)
    		 0
        Adolescents (12-17 years)
    		 0
        Adults (18-64 years)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Age at time of consent
    Units: years
        arithmetic mean (standard deviation)
    		 26.5 ( 11.74 ) 8.3 ( 1.53 ) -
    Gender categorical
    Units: Subjects
        Female
    		 5 1 6
        Male
    		 3 2 5
    EB Classification
    Major sub-type of EB per genetic diagnoses
    Units: Subjects
        epidermolysis bullosa simplex (EBS)
    		 4 3 7
        dystrophic epidermolysis bullosa (DEB)
    		 4 0 4
        junctional epidermolysis bullosa (JEB)
    		 0 0 0

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 (Adolescents, Adults)
    Reporting group description
    		 Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.

    Reporting group title
    Cohort 2 (Children)
    Reporting group description
    		 Children with EB (ages 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.

    Primary: Detectable Plasma Concentrations of Diacerein and Rhein

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    End point title
    		 Detectable Plasma Concentrations of Diacerein and Rhein [1]
    End point description
    Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods. For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose. Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only. Summary statistics for each scheduled time were only reported if at least 50% of subjects hand quantifiable concentrations.
    End point type
    Primary
    End point timeframe
    Days 1-10, at select time points per protocol.
    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 statistical analyses were performed for the descriptive exposure data.
    End point values
    		 Cohort 1 (Adolescents, Adults) Cohort 2 (Children)
    Number of subjects analysed
    8
    3
    Units: Count of Participants
        Rhein below the limit of quantification - EBS
    3
    2
        Rhein below the limit of quantification - DEB
    0
    0
        Rhein above the limit of quantification - EBS
    1
    1
        Rhein above the limit of quantification - DEB
    4
    0
        Diacerein below the limit of quantification - EBS
    4
    3
        Diacerein below the limit of quantification - DEB
    4
    0
        Diacerein above the limit of quantification - EBS
    0
    0
        Diacerein above the limit of quantification - DEB
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline to 30 days after last dose of study drug application (40 days)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Cohort 1 (Adolescents, Adults)
    Reporting group description
    		 Adolescent/adult patients with EB (aged 12 and older) received Diacerein 1% Ointment daily for 10 days

    Reporting group title
    Cohort 2 (Children)
    Reporting group description
    		 Children with EB (aged 4 to 11 years, inclusive) received Diacerein 1% Ointment daily for 10 days.

    Serious adverse events
    		 Cohort 1 (Adolescents, Adults) Cohort 2 (Children)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
    0 / 3 (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
    		 Cohort 1 (Adolescents, Adults) Cohort 2 (Children)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 3 (0.00%)
         occurrences all number
    1
    0

    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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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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