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    Summary
    EudraCT Number:2015-002622-39
    Sponsor's Protocol Code Number:PEDFOI
    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:2021-05-27
    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 number2015-002622-39
    A.3Full title of the trial
    Experimental Study Protocol on Treatment with plasma transfusions in Patients affected by type VI Osteogenesis Imperfecta
    STUDIO SPERIMENTALE PILOTA SUL TRATTAMENTO CON TRASFUSIONI DI PLASMA NEI PAZIENTI CON OSTEOGENESI IMPERFETTA TIPO VI
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Experimental Study Protocol on Treatment with plasma transfusions in Patients affected by type VI Osteogenesis Imperfecta
    STUDIO SPERIMENTALE SUL TRATTAMENTO CON TRASFUSIONI DI PLASMA NEI PAZIENTI CON OSTEOGENESI IMPERFETTA TIPO VI
    A.3.2Name or abbreviated title of the trial where available
    Experimental Study Protocol on Treatment with plasma transfusions in Patients affected by type VI Os
    STUDIO SPERIMENTALE SUL TRATTAMENTO CON TRASFUSIONI DI PLASMA NEI PAZIENTI CON OSTEOGENESI IMPERFETT
    A.4.1Sponsor's protocol code numberPEDFOI
    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 SponsorAZIENDA OSPEDALIERA UNIVERSITARIA INTEGRATA VERONA
    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 supportAOUI Verona
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAzienda Ospedaliera Universitaria Integrata Verona
    B.5.2Functional name of contact pointUnit¿ Supporto alla Ricerca e Biost
    B.5.3 Address:
    B.5.3.1Street AddressPiazzale Stefani 1
    B.5.3.2Town/ cityVerona
    B.5.3.3Post code37126
    B.5.3.4CountryItaly
    B.5.4Telephone number0458127043
    B.5.5Fax number0458122814
    B.5.6E-mailsupporto.noprofit@ospedaleuniverona.it
    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 Yes
    D.2.1.1.1Trade name PLASMASAFE - SOLUZIONE PER INFUSIONE 1 SACCA DA 200 ML
    D.2.1.1.2Name of the Marketing Authorisation holderKEDRION S.P.A.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 namePLASMASAFE
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INN00244301
    D.3.9.2Current sponsor codeplasmasafe
    D.3.10 Strength
    D.3.10.1Concentration unit ml millilitre(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number12 to 15
    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) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Yes
    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
    type VI Osteogenesis Imperfecta
    OSTEOGENESI IMPERFETTA TIPO VI
    E.1.1.1Medical condition in easily understood language
    Osteogenesis Imperfecta
    Osteogenesi Imperfetta
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10031243
    E.1.2Term Osteogenesis imperfecta
    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 study the efficacy and safety of PEDF administration, using pharmaceutical grade plasma (Plasmasafe), in Patients affected by type VI OI, genetically and biochemically confirmed.
    Lo scopo dello studio ¿ di creare evidenze preliminari circa l¿efficacia e la sicurezza della somministrazione di PEDF, attraverso la somministrazione di plasma umano di grado farmaceutico (PLASMASAFE) in pazienti affetti da Osteogenesi Imperfetta tipo VI geneticamente determinata.
    E.2.2Secondary objectives of the trial
    NA
    NA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Children between 1 and 18 years of age, affected by type VI OI, genetically and biochemically confirmed.
    Bambini di età superiore ad un anno, affetti da Osteogenesi Imperfetta tipo VI geneticamente determinata.
    E.4Principal exclusion criteria
    - Previous transfusional adverse events.
    - Need of transfusions for other reasons
    - Precedenti reazioni trasfusionali.
    - Necessità trasfusionali di altra natura.
    E.5 End points
    E.5.1Primary end point(s)
    a) to measure the concentration of PEDF in subjects suffering from OI type VI at the end of the transfusion of a standard dose of plasma (12-15 ml / kg in single infusion), after 1 day, 7 days, 1 month plasma transfusion and before each transfusion, and to evaluate the time interval in which remains a quantity of PEDF equal to the levels of heterozygous subjects for the genetic defect (estimated about 1/5 of the normal values, about 1 mg / ml) establishing, at the end of the transfusions, the best dose of plasma to be administered and the best interval between any subsequent infusions of plasma.
    b) to evaluate at 1 month after the sixth plasma transfusion, the markers of bone apposition and reabsorption, the bone histomorphometry and densitometric parameters.
    c) to monitor the occurrence of any adverse events, particularly hemodynamic overload and transfusion reactions or allergic-type reactions febrile non hemolytic transfusion evaluating vital signs before, during and at the end of plasma transfusions, or within the following three hours.
    d) to monitor the possible occurrence of infectious diseases through the repetition of serology and NAT for HBV, HCV and HIV and serology for LUE at 6 months after the last transfusion
    a) misurare la concentrazione di PEDF nei soggetti affetti da OI tipo VI al termine della trasfusione di una dose standard di plasma (12-15 ml/Kg in unica infusione), dopo 1 giorno, 7 giorni, 1 mese dalla trasfusione di plasma e prima di ogni trasfusione, in modo tale da valutare l’intervallo di tempo in cui permanga una quantità di PEDF pari ai livelli dei soggetti eterozigoti per il difetto genetico (stimato circa 1/5 dei valori normali, circa 1 µg/ml) e stabilire, al termine delle trasfusioni, la miglior dose di plasma da somministrare e il miglior intervallo fra eventuali successive infusioni di plasma a scopo di successivo trattamento.
    b) valutare a distanza di 1 mese dalla sesta trasfusione di plasma i markers di riassorbimento e apposizione ossei, l’istomorfometria ossea e i parametri densitometrici.
    c) monitorare la comparsa di eventuali eventi avversi alla trasfusione, in particolare sovraccarico emodinamico e reazioni trasfusionali di tipo allergico o reazioni febbrili non emolitiche, valutando parametri vitali prima della trasfusione, durante e al termine della stessa e nelle prime tre ore immediatamente successive.
    d) monitorare l’eventuale comparsa di malattie infettive attraverso la ripetizione delle sierologie e NAT per HBV, HCV ed HIV e sierologie per LUE a distanza di 6 mesi dall’ultima trasfusione
    E.5.1.1Timepoint(s) of evaluation of this end point
    a) 1 day, 7 days, 1 month plasma transfusion and before each transfusion
    b) 1 month after the sixth plasma transfusion
    c) before, during and at the end of plasma transfusions, or within the following three hours.
    d) 6 months after the last transfusion
    a) 1 giorno, 7 giorni, 1 mese dalla trasfusione di plasma e prima di ogni trasfusione
    b) 1 mese dalla sesta trasfusione di plasma
    c) durante la trasfusione e al termine della stessa e nelle prime tre ore immediatamente successive.
    d) 6 mesi dall’ultima trasfusione
    E.5.2Secondary end point(s)
    NA
    NA
    E.5.2.1Timepoint(s) of evaluation of this end point
    NA
    NA
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA1
    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 Information not present in EudraCT
    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 years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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) 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: 4
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) No
    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 No
    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
    Children older than 1 year
    Bambini di et¿ superiore ad 1 anno
    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 10
    F.4.2.2In the whole clinical trial 10
    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)
    In the case of positive results in the short period, the study protocol could be continued as the Patient's and Families request.
    In caso di risultati positivi nel breve periodo, su richiesta del paziente e dei genitori, il protocollo di studio potr¿ essere continuato
    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 Decision2015-09-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-18
    P. End of Trial
    P.End of Trial StatusCompleted
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