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    Summary
    EudraCT Number:2016-004804-77
    Sponsor's Protocol Code Number:1262_OPBG_2018
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-09-14
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-004804-77
    A.3Full title of the trial
    A prospective study to assess safety and efficacy of the use of bone-marrow derived MESenchymal stromal cells as immunomodulatory therapy for children and young adults with severe and difficult-to-treat frequently
    relapsing or steroid-dependent idiopathic NEPHrotic syndrome: the MESNEPH study
    Studio prospettico sulla sicurezza ed efficacia dell’impiego di cellule stromali MESenchimali come terapia immunomodulatoria in pazienti pediatrici e giovani adulti affetti da forme severe e difficili di sindrome nefrosica idiopatica corticosensibile a frequenti recidive o steroido-dipendente: studio MESNEPH
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    MSC as therapy for INS: phase 1 study
    MES come terapia della sindrome nefrosica idiopatica : studio di fase I
    A.3.2Name or abbreviated title of the trial where available
    MESNEPH STUDY
    STUDIO MESNEPH
    A.4.1Sponsor's protocol code number1262_OPBG_2018
    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 SponsorBAMBINO GESU' CHILDREN'S HOSPITAL
    B.1.3.4CountryItaly
    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 supportRicerca Indipendente dell'AIFA
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBAMBINO GESU' CHILDREN'S HOSPITAL
    B.5.2Functional name of contact pointMarina Vivarelli
    B.5.3 Address:
    B.5.3.1Street AddressViale San Paolo, 15
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00165
    B.5.3.4CountryItaly
    B.5.4Telephone number00390668592393
    B.5.5Fax number00390668592602
    B.5.6E-mailmarina.vivarelli@opbg.net
    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 nameAutologous mesenchymal stromal cells (MSC)
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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.IMP: 2
    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 nameAutologous mesenchymal stromal cells (MSC)
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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
    severe multi-relapsing or steroid-dependent INS.
    sindrome nefrosica corticosensibile a frequenti recidive e/o steroido-dipendente
    E.1.1.1Medical condition in easily understood language
    INS
    sindrome nefrosica difficile
    E.1.1.2Therapeutic area Diseases [C] - Male diseases of the urinary and reproductive systems [C12]
    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
    To assess the feasibility and safety of harvesting from their bone marrow, isolating and expanding ex vivo in an approved facility and then infusing autologous MSC in children and young adults with severe multi-relapsing or steroid-dependent INS.
    L’obiettivo primario dello studio è quello di valutare la sicurezza e la fattibilità dell’impiego di cellule mesenchimali autologhe a scopo terapeutico in pazienti con sindrome nefrosica difficile.
    E.2.2Secondary objectives of the trial
    1. To evaluate whether the therapeutic use of autologous MSC is able to prevent disease relapse despite tapering of prednisone and/or other immunosuppressive treatments in children and young adults with severe multi-relapsing or steroid-dependent INS.
    2. To assess whether the therapeutic use of autologous MSC may reduce the need for prednisone and/or other immunosuppressive agents to prevent and treat further disease relapses;
    3. To evaluate whether tapering or withdrawal of immunosuppressant therapy is associated with regression of the related toxicities, such as growth retardation, hypertension, impaired glucose tolerance and obesity, infections, B cell suppression;
    4. To evaluate the immunomodulatory effect of the MSC infusion in vivo in a clinical setting.
    1. Valutare se l'uso terapeutico delle MSC autologhe è in grado di prevenire la recidiva della malattia nonostante la diminuzione del prednisone e / o di altri trattamenti immunosoppressivi nei bambini e nei giovani adulti con INS severa a frequenti recidive o steroido-dipendente.
    2. Valutare se l'uso terapeutico delle MSC autologhe può ridurre la necessità di prednisone e / o di altri agenti immunosoppressivi per la prevenzione e il trattamento di ulteriori recidive di malattia;
    3. Valutare se la riduzione o la sospensione della terapia immunosoppressiva sia associata alla regressione della tossicità correlata, quali ritardo della crescita, ipertensione, ridotta tolleranza al glucosio e obesità, infezioni, soppressione delle cellule B;
    4. Valutare l'effetto immunomodulatore dell'infusione MSC in vivo in ambito clinico
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Males and females aged 5 to 40 years.
    • Steroid-dependent or multirelapsing INS patients with 2 or more relapses in the previous year in spite of prednisone and/or one or more other immunosuppressive steroid-sparing agent. Only patients reported to invariably relapse upon treatment tapering or withdrawal who are on stable (from at least 1 month) complete (<0.3 g/24h for adults or <4 mg/h/m2 for children) or partial (<3.5 g/24h for adults or <40 mg/h/m2 for children) remission of the INS will be included. Rituximab treatment can have been previously performed but reconstitution of B cells, defined as total CD19/CD20 lymphocyte count above 5% of total lymphocytes by cytofluorimetry, must have occurred and must be recorded.
    • Histological diagnosis of MCD, FSGS, mesangial proliferative glomerulonephritis.
    • Written informed consent (or consent from parents or tutors for underage patients, as appropriate).
    • If applicable, female participants must have pregnancy test by beta-HCG dosing and be negative.
    • Patients of child-bearing or child-fathering potential must be willing to practice and must contact their physician to agree with him/her on the most appropriate approach for birth control from the time of enrollment in this study and for 3 months after receiving the latest MSC infusion.
    • Soggetti di genere maschile o femminile di età compresa tra 5 e 40 anni.
    • Pazienti affetti da forme di INS a fequenti recidive o steroido-dipendenti che abbiano presentato 2 o più recidive nell’ultimo anno nonostante terapia immunosoppresiva con prednisone e/o uno o più farmaci immunosoppressivi di seconda linea.
    • Diagnosi istologica renale di malattia a lesioni minime, glomerulosclerosi focale e segmentale o proliferazione mesangiale diffusa.
    • Firma di consenso informato da parte del paziente o da parte dei genitori/tutori legali, secondo le norme di legge.
    • Esecuzione test di gravidanza (dosaggio beta-HCG ematiche) per soggetti di genere femminile in età fertile;
    • Soggetti di sesso maschile e femminile in età fertile devono consultare il medico specialista al fine di concordare la modalità più opportuna per non procreare dal momento dell’arruolamento in questo studio fino a 3 mesi dopo aver ricevuto l’ultima infusione.
    E.4Principal exclusion criteria
    • Advanced renal failure (creatinine clearance less than 20 ml/min/1.73m2), calculated using the Schwartz formula or the Cockroft-Gault formula, as appropriate;
    • Refractory or persistent NS;
    • Genetic mutations associated with intrinsic abnormalities of the glomerular barrier;
    • Pregnancy or lactating;
    • Women of childbearing potential without following a scientifically accepted form of contraception;
    • Infectious pathogen testing positive for active infection;
    • Legal incapacity;
    • Evidence of an uncooperative attitude;
    • Previous diagnosis of: intellectual disability/mental retardation, dementia, schizophrenia.
    • Any evidence that patient will not be able to complete the trial follow-up.
    • Insufficienza renale cronica di grado severo (clearance della creatinina inferiore a 20 ml/min/1.73m2)
    • INS refrattaria o persistente
    • Presenza di mutazioni genetiche associate ad anomalie intrinsiche della barriera di filtrazione glomerulare
    • Gravidanza o allattamento
    • Segni di infezione attiva allo screening
    • Incapacità legale
    • Evidenza di un atteggiamento scarsamente cooperativo
    • Diagnosi di disabilità cognitiva/ritardo mentale, demenza, schizofrenia
    • Evidenza che il paziente non sarà in grado di portare a termine lo studio.
    E.5 End points
    E.5.1Primary end point(s)
    Safety outcomes will include serious and non-serious adverse events, including acute reactions during MSC infusion, infectious episodes and malignancies.
    La frequenza di eventi avversi gravi e non gravi, incluse reazioni acute durante infusione di MSC, episodi infettivi e tumori maligni.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at 12 months
    a 12 mesi
    E.5.2Secondary end point(s)
    - Recurrence of INS, defined as 3+ or more positive Albustix dipsticks for proteinuria on 3 consecutive days or by positive dipsticks (1+ to 3+) for 7 consecutive days, in the 12 months before and in the 12 months following MSC infusions.
    - The dose of immunosuppressive therapy required to prevent further INS relapses
    - Adverse effects of immunosuppressive therapy, such as arterial hypertension and need for antihypertensive therapy, obesity and impaired glucose tolerance, dyslipidemia, renal dysfunction, stunted statural growth, infections, immunocompetence;
    - Kidney function at baseline and at one year after MSC administration.
    - Ricorrenza di INS, definita come 3 + o più dipstick di Albustix positivo per proteinuria per 3 giorni consecutivi o per dipstick positivi (da 1 a 3+) per 7 giorni consecutivi, nei 12 mesi precedenti e nei 12 mesi successivi alle infusioni di MSC.
    - La dose di terapia immunosoppressiva necessaria per prevenire ulteriori ricadute dell'INS
    - Effetti avversi della terapia immunosoppressiva, quali ipertensione arteriosa e necessità di terapia antipertensiva, obesità e ridotta tolleranza al glucosio, dislipidemia, disfunzione renale, crescita statica rachitica, infezioni, immunocompetenza;
    - Funzione renale al basale e ad un anno dalla somministrazione di MSC.
    E.5.2.1Timepoint(s) of evaluation of this end point
    at 12 months
    a 12 mesi
    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) 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
    First administration to subjects with INS
    Prima somministrazione nell'uomo per questa indicazione
    E.7.2Therapeutic exploratory (Phase II) No
    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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 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 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
    last visit of the last patient + 6 months for data collection, data analysis and preparation of the scientific publication.
    LVLS + 6 mesi per analisi e pubblicazione
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months4
    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.1.1Number of subjects for this age range: 12
    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: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    infants
    minori
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    n.a.
    n.a.
    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 Decision2018-07-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-03-21
    P. End of Trial
    P.End of Trial StatusCompleted
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