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    Summary
    EudraCT Number:2014-005679-96
    Sponsor's Protocol Code Number:SD-006
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-09-09
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2014-005679-96
    A.3Full title of the trial
    An Open Label, Multi-center, Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients with Epidermolysis Bullosa
    Estudio de extensión, multicéntrico y abierto para evaluar la seguridad a largo plazo de ZorblisaTM (SD-101-6.0) en pacientes con epidermólisis ampollosa
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Investigation into the Long-term Safety of Zorblisa (SD-101-6.0) cream used by Patients with Epidermolysis Bullosa.
    Una investigación a largo plazo sobre la seguridad de la crema Zorblisa (SD-101-6.0) usada por pacientes con epidermólisis ampollosa
    A.3.2Name or abbreviated title of the trial where available
    Study to Evaluate the Long-term Safety of Zorblisa in Patients with Epidermolysis Bullosa
    A.4.1Sponsor's protocol code numberSD-006
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/266/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorScioderm, INC
    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 supportScioderm, INC.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationScioderm, INC.
    B.5.2Functional name of contact pointJamie Gault
    B.5.3 Address:
    B.5.3.1Street Address4601 Creekstone Drive Suite 160
    B.5.3.2Town/ cityDurham
    B.5.3.3Post codeNC 27703
    B.5.3.4CountryUnited States
    B.5.4Telephone number0034620983130
    B.5.5Fax number001919294-4416
    B.5.6E-mailjamie.gault@sderm.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 numberEMA/OD/145/13
    D.3 Description of the IMP
    D.3.1Product nameZorblisa
    D.3.2Product code SD-101
    D.3.4Pharmaceutical form Cream
    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 INNALLANTOIN
    D.3.9.1CAS number 97-59-6
    D.3.9.3Other descriptive nameALLANTOIN
    D.3.9.4EV Substance CodeSUB12779MIG
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/V) percent weight/volume
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Epidermolysis Bullosa
    Epidermólisis bullosa
    E.1.1.1Medical condition in easily understood language
    Genetic skin fragility disorder
    enfermedad genética de fragilidad de la piel
    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 19.0
    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
    The primary objective is to demonstrate the long-term safety of ZORBLISA in patients, with Simplex, Recessive Dystrophic and Junctional non-Herlitz Epidermolysis Bullosa.
    El objetivo principal es demostrar la seguridad a largo plazo de ZORBLISA en pacientes con epidermólisis ampollosa simple, distrófica recesiva y juntural tipo no-Herlitz.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to assess the efficacy of ZORBLISA in terms of the change in Body Surface Area (BSA) of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study
    Los objetivos secundarios son evaluar la eficacia de ZORBLISA en cuanto a los cambios en la superficie corporal (SC) afectada por lesiones y en la carga asociada a las heridas, así como el cierre de las heridas no cicatrizadas durante el estudio SD-005.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed Consent form signed by the patient or patient's legal
    representative; if the patient is under the age of 18 but
    capable of providing assent, signed assent from the patient.
    2. Patient (or caretaker) must be willing to comply with all protocol
    requirements.
    3. Patient who completed the SD-005 study (on study drug at Visit 5)
    1. Consentimiento informado firmado por el sujeto o por el representante legal del sujeto; si el sujeto es menor de 18 años de edad pero es capaz de dar su asentimiento, asentimiento firmado por el sujeto.
    2. El sujeto (o su cuidador) debe estar dispuesto a cumplir todos los requisitos del protocolo.
    3. Pacientes que hayan completado el estudio SD-005 (con administración del fármaco del estudio en la visita 5).
    E.4Principal exclusion criteria
    1. Patients who do not meet the entry criteria outlined in the Inclusion criteria.
    2. Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed at the final visit for SD-005 for female patients of childbearing potential.)
    3. Females of childbearing potential who are not abstinent or not practicing a medically acceptable method of contraception.
    1. Pacientes que no cumplan los criterios de inclusión especificados con anterioridad.
    2. Embarazo o lactancia durante el estudio. (En el caso de las pacientes en edad fértil, se realizará una prueba de embarazo en orina en la visita final del estudio SD-005).
    3. Mujeres en edad fértil que no se abstengan de tener relaciones sexuales o no empleen un método de anticoncepción aceptable
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the long-term safety of ZORBLISA in patients with Simplex, Recessive Dystrophic, and Junctional non-Herlitz Epidermolysis Bullosa
    El criterio principal de valoración es la seguridad a largo plazo de ZORBLISA en pacientes con epidermólisis ampollosa simple, distrófica recesiva y juntural tipo no-Herlitz.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety is assessed via monitoring of local tolerability at the application sites, occurrence of adverse events and physical examinations at Months 1, 3, 6, 9 and 12
    La seguridad se evaluará mediante monitorización de la tolerabilidad a nivel local, acontecimientos adversos y exploraciones físicas los meses 1, 3, 6, 9 y 12
    E.5.2Secondary end point(s)
    The secondary endpoints include the change from baseline in BSA coverage of lesional skin and wound burden; as well as closure of unhealed target wounds from the SD-005 study.
    los objetivos secundarios incluyen el cambio desde basal en la cobertura SC de las lesiones y el nivel de lesiones, así como el cierre de lesiones diana que no han curado desde el ensayo SD-005
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Change in lesional skin based on BSA estimates at Months 1, 3, 6, 9 and 12 compared to Baseline
    - Change in total body wound coverage based on BSA estimates at Months 1, 3, 6, 9 and 12 compared to Baseline
    - For target wounds that are not closed by the end of Study SD-005, the target wound area at the final visit for Study SD-005 will be used as the baseline
    area size of the target wound for SD-006. Target wounds will be assessed at each subsequent visit until the wound is documented as closed.
    - Cambio de la piel afectada en base a los cálculos de SC en los meses 1, 3, 6, 9 y 12 en coparación con el periodo basal
    - Cambio en la cobertura de la superficie corporal afectada por lesiones, en base a estimaciones de la SC en los meses 1, 3, 6, 9 y 12 en coparación con el periodo basal
    - Para lesiones diana que no habían cerrado en el estudio SD-005, el area de la lesión diana en la visita final del estudio SD-005 se usará como basal para el estudio SD-006. Las lesiones diana se evaluarán en cada visita hasta que se documente el cierre
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 EEA8
    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
    Austria
    France
    Germany
    Italy
    Netherlands
    Poland
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 130
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 23
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 26
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 22
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 39
    F.1.3Elderly (>=65 years) No
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 98
    F.4.2.2In the whole clinical trial 130
    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)
    SD-006 will run for one year until marketing application approval as we would like to continue to provide patients with topical therapy without interruption as this could be detrimental to patients showing a response to treatment in the SD-005 study. Three month check-ups will be organized whereby Scioderm will collect long term safety data on SD-101. This will be assessed by monitoring the local tolerability at the application sites, occurrence of adverse events and physical examinations.
    El ensayo SD-006 durará un año hasta tener la autorización de comercialización con el objetivo de continuar proporcionando el medicamento a los pacientes sin interrupción. Se harán revisiones cada 3 meses para recoger datos de seguridad de SD-101 a largo plazao. La evaluación se hará por la tolerabilidad local en las zonas de aplicación, acontecimientos adversos y exploraciones físicas
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-11-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-11-03
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-09-03
    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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