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    Summary
    EudraCT Number:2020-002936-55
    Sponsor's Protocol Code Number:MSC-EB_2020
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-08-03
    Trial 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 ConcernedCzechia - SUKL
    A.2EudraCT number2020-002936-55
    A.3Full title of the trial
    Safety and Efficacy of Allogenic Adipose Tissue-derived Mesenchymal Stromal Cells in Patients with Epidermolysis Bullosa: Clinical Trial Phase I/II
    Bezpečnost a účinnost alogenních mezenchymálních stromálních buněk odvozených z tukové tkáně u pacientů s epidermolysis bullosa: klinické hodnocení fáze I/II
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of Mesenchymal Stromal Cells Derived from Adipose Tissue of Non-related Donors in Patients with The Butterfly Disease: Clinical Trial Phase I/II
    Bezpečnost a účinnost mezenchymálních stromálních buněk z tukové tkáně nepříbuzných dárců u pacientů s nemocí motýlích křídel: klinické hodnocení fáze I/II
    A.3.2Name or abbreviated title of the trial where available
    MSC-EB
    MSC-EB
    A.4.1Sponsor's protocol code numberMSC-EB_2020
    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 SponsorMasarykova univerzita
    B.1.3.4CountryCzechia
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportDEBRA ČR, z.ú.
    B.4.2CountryCzechia
    B.4.1Name of organisation providing supportMinisterstvo školství, mládeže a tělovýchovy ČR
    B.4.2CountryCzechia
    B.4.1Name of organisation providing supportMasarykova univerzita
    B.4.2CountryCzechia
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMasarykova univerzita - LF
    B.5.2Functional name of contact pointFarmÚ- Centrum klinických hodnocení
    B.5.3 Address:
    B.5.3.1Street AddressKamenice 5
    B.5.3.2Town/ cityBrno
    B.5.3.3Post code625 00
    B.5.3.4CountryCzechia
    B.5.4Telephone number+420549497205
    B.5.6E-mailjana.kubatova@med.muni.cz
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameallogeneic adipose tissue-derived MSC
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntradermal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman allogeneic adipose tissue-derived mesenchymal stem cells expanded
    D.3.9.3Other descriptive nameALLOGENEIC ADIPOSE TISSUE-DERIVED MESENCHYMAL STEM CELLS EXPANDED
    D.3.9.4EV Substance CodeSUB181445
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10000000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    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 congenita
    Epidermolysis bullosa congenita
    E.1.1.1Medical condition in easily understood language
    Rare skin disease "The Butterfly Disease"
    Vzácné kožní onemocnění "nemoc motýlích křídel"
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    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 of the study is to assess the safety of human allogeneic mesenchymal stromal cells administered intradermally
    Primárním cílem studie je posoudit bezpečnost podávání lidských alogenních mezenchymálních stromálních buněk podávaných intradermálně
    E.2.2Secondary objectives of the trial
    The secondary objective of the study is to obtain pilot data on the efficacy of the treatment
    Sekundárním cílem studie je získat pilotní údaje o účinnosti této léčby
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Age > 1 year (incl.)
    2) Clinical diagnosis of hereditary EB supported by histological diagnosis (electron microscopy, immunofluorescence antigen mapping) and DNA analysis
    3) The extent of the disease is at least 10% of the body surface
    4) Participants in a clinical trial of childbearing potential must agree to the use of prescribed contraceptive methods for the duration of the clinical study and for at least 6 months following the last dose of the study medication:
    a) Women - Proper use of a highly reliable method of contraception, ie combined hormonal contraception (in oral, vaginal or transdermal dosage form), gestagen hormonal contraception associated with ovulation inhibition (in oral or injectable dosage form), non-hormonal intrauterine device or intrauterine device releasing hormones, or the presence of bilateral tubal occlusion, a partner's vasectomy, or adherence to sexual abstinence as part of the patient's normal lifestyle.
    b) Men - Adherence to sexual abstinence or the use of an adequate contraceptive method (ie condom) in case of sexual intercourse.
    5) Willingness and ability to adhere to restrictions in the care of study lesions and to adhere to the schedule of visits and examinations during the course of the study.
    1) Věk vyšší než 1 rok (včetně)
    2) Klinická diagnóza hereditární EB podpořená histologickou diagnózou (elektronová mikroskopie, imunofluorescenční antigenní mapování) a analýzou DNA
    3) Rozsah onemocnění alespoň 10 % povrchu těla
    4) Účastníci klinického hodnocení v plodném věku musí souhlasit s používáním předepsaných metod antikoncepce po dobu trvání klinické studie a minimálně 6 měsíců následujících po podání poslední dávky studijní medikace:
    a) Ženy – Správné použití vysoce spolehlivé antikoncepční metody, tj. kombinované hormonální antikoncepce (v perorální, vaginální nebo transdermální lékové formě), gestagenní hormonální antikoncepce spojené s inhibicí ovulace (v perorální nebo injekční lékové formě), nehormonálního nitroděložního tělíska nebo nitroděložního tělíska uvolňující hormony, ev. přítomnost oboustranné tubární okluze, vazektomie u partnera, nebo dodržování sexuální abstinence, pokud jde o součást běžného životního stylu pacientky.
    b) Muži – Dodržování sexuální abstinence nebo použití adekvátní antikoncepční metody (tj. kondomu) v případě pohlavního styku.
    5) Ochota a možnost dodržet omezení v péči o studijní léze a dodržovat rozpis návštěv a vyšetření v rámci studie.
    E.4Principal exclusion criteria
    1) Systemic infection or sepsis 7 days before MSC administration
    2) Treatment with blood transfusion (erythrocyte concentrate or whole blood transfusion) 7 days before MSC administration
    3) Clinical signs of infection in the study lesions 7 days before MSC administration
    4) Inability to tolerate repeated skin injections (especially in pediatric patients)
    5) History of basal cell or squamous cell carcinoma of the skin in the last 5 years
    6) History of other malignancies of any type at any time during life
    7) Severe lung disease that requires home oxygenation
    8) Severe kidney or liver disease
    9) Dilatation cardiomyopathy
    10) Life expectancy less than 90 days
    11) Severe immune response to allogeneic human cells
    12) Other severe somatic or psychiatric illnesses that are not adequately controlled and, in the opinion of the investigator, would not allow the study protocol to be followed
    13) Previous administration of a medicinal product in a clinical trial in the 30 days or 5 elimination half-lives (whichever is longer) before the enrolment to this trial (applies to clinical investigations of medical devices, as well)
    14) Pregnancy or breastfeeding
    15) Allergy/hypersensitivity to any component of Hypotermosol solution
    1) Přítomnost systémové infekce nebo sepse 7 dní před podáním MSC
    2) Léčba krevní transfuzí (koncentrátem erytrocytů nebo transfuzí plné krve) 7 dní před podáním MSC
    3) Klinické příznaky infekce v uvažovaných studijních lézích 7 dní před podáním MSC
    4) Neschopnost snést opakované vpichy do kůže (především u dětských pacientů)
    5) Anamnéza bazocelulárního nebo spinocelulárního karcinomu kůže v posledních 5 letech
    6) Anamnéza jiné malignity jakéhokoli typu kdykoli v průběhu života
    7) Těžké onemocnění plic, které vyžaduje domácí podávání kyslíku
    8) Těžké onemocnění ledvin a jater v osobní anamnéze
    9) Dilatační kardiomyopatie v osobní anamnéze
    10) Očekávaná délka života kratší než 90 dní
    11) Prodělaná těžká imunitní reakce na podání alogenních lidských buněk
    12) Jiná těžká somatická nebo psychiatrická onemocnění, která nejsou dostatečně kontrolována a podle názoru zkoušejícího lékaře by neumožňovala dodržet protokol studie
    13) Předchozí užití léčivého přípravku v jiném klinickém hodnocení 30 dní nebo 5 eliminačních poločasů (podle toho, která lhůta je delší) před náborem do této studie (platí také pro účast v klinické zkoušce zdravotnického prostředku)
    14) Těhotenství nebo kojení
    15) Alergie/hypersenzitivita na na jakoukoliv složku přípravku/roztoku Hypotermosol
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of adverse events of special interest (AESI): severe local reaction at the injection site other than blistering, severe systemic allergic reaction at any time after MSC administration, severe systemic or local infectious complications at any time after MSC administration (combined primary safety endpoint)
    Výskyt nežádoucích příhod zvláštního významu (AESI): těžké lokální reakce v místě injekce s výjimkou vzniku puchýře, těžké systémové alergické reakce kdykoli po podání MSC, těžké systémové nebo lokální infekční komplikace kdykoli po podání MSC (kombinovaný primární parametr bezpečnosti)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Cohort 1:
    - Day 90 after 2nd administration of the 4th adult patient
    - End of the trial (Day 290 after the 2nd administration)
    Cohort 2:
    - Day 7 after 1st administration of the 1st patient aged 12-17 years
    - Day 7 after 1st administration of the 1st patient aged 2-11 years
    - Day 7 after 1st administration of the 1st patient aged 12-23 months
    - End of the trial (Day 290 after the 2nd administration of all pediatric patients)
    Kohorta 1:
    - Den 90 po podání 2. dávky 4. dospělému pacientovi
    - Na konci studie (Den 290 po podání 2. dávky všem dospělým pacientům)
    Kohorta 2:
    - Den 7 po podání 1. dávky 1. pacientovi ve věku 12-17 let
    - Den 7 po podání 1. dávky 1. pacientovi ve věku 2-11 let
    - Den 7 po podání 1. dávky 1. pacientovi ve věku 12-23 měsíců
    - Na konci studie (Den 290 po podání 2. dávky všem pediatrickým pacientům)
    E.5.2Secondary end point(s)
    Secondary safety endpoints:
    - Incidence, type, seriousness, intensity of AEs and their relation to study medication

    Efficacy endpoints:
    - Non-healed part in the area marked in the study active and control lesions
    - Change in the area of non-healed part from baseline
    - Estimation of the total area of lesions on the patient's body (% of body surface area)
    Sekundární bezečnostní parametry:
    - Výskyt, druh, závažnost, intenzita nežádoucích příhod a jejich vztah ke studijní medikaci

    Parametry účinnosti:
    a) Plocha nezhojené rány v oblasti vyznačené ve studijních lézích
    b) Změna plochy nezhojené rány od zařazení do studie
    c) Odhad celkové plochy lézí na těle pacienta (% tělesného povrchu)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary safety endpoints: End of the trial (Day 290 after the 2nd administration)

    Efficacy endpoints:
    a) Day 4, 15, 30, and 75 after the 1st administration and Day 4, 15, 30 and 90 after the 2nd administration
    b) Day 30 after the 1st administration and Day 30 after the 2nd administration
    c) Day 1 vs. Day 30 after the 1st and 2nd administration
    Sekundární bezečnostní parametry: na konci studie (den 290 po 2. podání)

    Parametry účinnosti:
    a) Den 4, 15, 30 a 75 po 1. podání a den 4, 15, 30 a 90 po 2. podání
    b) Den 30 po 1. podání a den 30 po 2. podání
    c) Den 1 oproti Dnu 30 po 1. i 2. podání
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    exploratory trial of ATMP, IMP has already been used in human
    explorativní studie LPMT, HLP bylo již u člověka podáváno
    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 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.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 11
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 4
    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 state15
    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)
    None
    Žádná
    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 Decision2021-05-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-11
    P. End of Trial
    P.End of Trial StatusOngoing
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