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    The EU Clinical Trials Register currently displays   38451   clinical trials with a EudraCT protocol, of which   6312   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2016-004427-24
    Sponsor's Protocol Code Number:CCP-020-301
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-05-30
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedAustria - BASG
    A.2EudraCT number2016-004427-24
    A.3Full title of the trial
    An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study]
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An International Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)
    A.4.1Sponsor's protocol code numberCCP-020-301
    A.5.4Other Identifiers
    Name:INDNumber:131 384
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCastle Creek Pharmaceuticals, LLC
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCastle Creek Pharmaceuticals, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace Germany GmbH
    B.5.2Functional name of contact pointVasiliki Iassonidou
    B.5.3 Address:
    B.5.3.1Street AddressTheresienhöhe 30
    B.5.3.2Town/ cityMünchen
    B.5.3.3Post code80339
    B.5.3.4CountryGermany
    B.5.4Telephone number+49(89) 89 55 718 99 ex24960
    B.5.5Fax number+442033183827
    B.5.6E-mailV.Iassonidou@medpace.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1236
    D.3 Description of the IMP
    D.3.1Product nameDiacerein
    D.3.2Product code Diacerein
    D.3.4Pharmaceutical form Ointment
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDIACEREIN
    D.3.9.1CAS number 13739-02-1
    D.3.9.2Current sponsor codeCCP-020, AC-203
    D.3.9.3Other descriptive nameDiacerein
    D.3.9.4EV Substance CodeSUB07060MIG
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOintment
    D.8.4Route of administration of the placeboTopical use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Epidermolysis Bullosa Simplex (EBS)
    E.1.1.1Medical condition in easily understood language
    Epidermolysis Bullosa Simplex (EBS)
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10014989
    E.1.2Term Epidermolysis bullosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the efficacy of Diacerein 1% Ointment to Control Ointment based on reduction in body surface area (BSA) of EBS lesions being treated when applied once-daily for 8 weeks in subjects with EBS.
    E.2.2Secondary objectives of the trial
    To compare the effects of Diacerein 1% Ointment to Control Ointment in
    subjects with EBS in:
    • Changes in Investigator Global Assessment (IGA) scores
    • Pain
    • Pruritus
    • Mobility
    • Safety and tolerability
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is male or female at least 4 years of age at Visit 1
    2. Subject has a documented genetic mutation consistent with EBS. A blood or saliva sample will be collected for genetic confirmation if no documented gene mutation data is available. Gene mutations acceptable for inclusion are as follows: KRT5, KRT14, PLEC1, TGM5, PKP1, DSP, FERMT1, EXPH5, DST, KLHL24.
    3. Subject has an Assessment Area of EBS lesions to be treated, that is ≥2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:
    - Localized: plantar and/or palmar areas (plantar areas where >25% of the area has hyperkeratosis that has been present for greater than 12 weeks cannot be included as part of the Assessment Area)
    - Generalized: arms, legs, torso, hands and feet (scalp, groin and areas where, in the investigator's opinion, the study medication might become
    occluded cannot be included as part of the Assessment Area
    4. Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of ≥3
    5. Subject/caregiver agrees to not use any topical therapies other than the study medication that, might influence the status of the EBS lesions
    during the duration of the study (e.g., medicated cleansers, CBD oil, MediHoney, Silvadine cream 1%); the Investigator should consult the
    Medical Monitor regarding therapies not specified in the protocol
    6. Subject/caregiver agrees to follow topical product application instructions during the treatment period
    7. If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of
    birth control, as defined by this protocol (see Section 10.6), for the duration of the study.
    8. Subject is non-pregnant, non-lactating and is not planning for pregnancy during the study period
    9. Subject is in good general health and free of any known disease state or physical condition which, in the investigator's opinion, might impair
    evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation
    10. Subject is willing and able to follow all study instructions and to attend all study visits
    11. Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form.
    E.4Principal exclusion criteria
    1. Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require therapy to treat an infection)
    2. Subject has used any diacerein containing product within 6 months prior to Visit 1
    3. Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
    4. Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to
    Visit 2 (Note: inhaled and ophthalmic products containing steroids are allowed)
    5. Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Visit 1
    6. Subject is currently using systemic analgesics and/or anti-histamine therapy for treatment of EBS lesions, unless on a stable regimen (i.e., the same dosing regimen) for at least 4 weeks prior to Visit 1. Note: As needed (PRN) use of acetaminophen/paracetamol or NSAIDs within the 4 weeks prior to Visit 1 are permitted provided the treatment was unrelated to EBS symptom relief.
    7. Subject has used any systemic diuretics or cardiac glycosides or any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments within 30 days prior to Visit 1
    8. Subject has used any topical product containing allantoin on the EBS lesions to be treated within 30 days prior to Visit 1
    9. Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to Visit 1 (Note: does not include nonmelanoma
    skin cancer)
    10. Subject currently has diabetes mellitus (HbA1c ≥6.5%). Note: controlled diabetes (HbA1c < 6.5%) is also considered exclusionary
    11. Subject has a history of cardiac, hepatic (ALT and or AST >2x ULN, Total bilirubin >1.5x ULN at Visit 1), or renal disease (eGFR<30 ml/min/1.73 m2) that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application of the study assessments
    12. Subject has a non-EBS skin disease (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the
    study assessments
    13. Subject has a history of sensitivity to any of the ingredients in the study medications.
    14. Subject has participated in any investigational drug/device trial in which administration of an investigational study medication occurred within 30 days prior to Visit 1.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is based on the ITT population, and is the proportion of subjects who achieve ≥40% reduction in BSA of EBS lesions from Visit 2 (Week 0) to Visit 8 (Week 16).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline (D1) and Visit 8 (D113)
    E.5.2Secondary end point(s)
    • The proportion of subjects achieving success on the IGA, where success is defined as at least a 2-point reduction, from Visit 2 (Week 0)
    to Visit 8 (Week 16).
    • The proportion of subjects who achieve ≥ 40% reduction in BSA of EBS lesions from Visit 2 (Week 0) to Visit 6 (Week 8).
    • The proportion of subjects achieving success on the IGA, where success is defined as at least a 2-point reduction, from Visit 2 (Week 0)
    to Visit 6 (Week 8).
    • The proportion of subjects with a reduction in overall pain intensity from Visit 2 (Week 0) to Visit 6 (Week 8).
    • The proportion of subjects with a reduction in overall pruritus intensity from Visit 2 (Week 0) to Visit 6 (Week 8).

    If the comparison for the primary endpoint is significant (two-sided pvalue < 0.05), then testing will continue for the key secondary endpoints. The key secondary endpoints will be analyzed in a similar manner as the primary endpoint. To control the type I error rate for testing multiple key secondary endpoints, a fixed-sequence approach for the key secondary endpoints will be used.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All except PK: Baseline (D1) and Visit 8 (D113)
    PK: Visit 4 (D29); Visit 6 (D57); Visit 8 (D113)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Austria
    France
    Germany
    Israel
    Italy
    Netherlands
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 80
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Standard of Care
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-10-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-04
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-10-30
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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