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    The EU Clinical Trials Register currently displays   35510   clinical trials with a EudraCT protocol, of which   5839   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2018-001741-14
    Sponsor's Protocol Code Number:TEM-MM-101
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-06-12
    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 number2018-001741-14
    A.3Full title of the trial
    A phase I/II dose escalation study evaluating safety and activity of autologous CD34+-enriched hematopoietic progenitor cells genetically modified with a lentiviral vector encoding for the human interferon-alfa2 gene in multiple myeloma patients with early relapse after intensive front line therapy
    Sperimentazione clinica di fase I/II atta a valutare la sicurezza e l’attività di dosi crescenti di cellule staminali ematopoietiche autologhe CD34+ geneticamente modificate con un vettore lentivirale codificante per il gene umano dell’interferone-alfa2 in pazienti affetti da mieloma multiplo recidivato precocemente dopo trattamenti intensivi di prima linea
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical gene therapy study with hematopoietic stem cells for the treatment of patients suffering from a plasma cell tumor.
    Studio clinico di terapia genica con cellule staminali ematopoietiche per il trattamento di pazienti affetti da un tumore delle plasmacellule
    A.3.2Name or abbreviated title of the trial where available
    TEM-MM
    TEM-MM
    A.4.1Sponsor's protocol code numberTEM-MM-101
    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 SponsorOSPEDALE SAN RAFFAELE
    B.1.3.4CountryItaly
    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 supportGenenta Science S.r.l.
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOSPEDALE SAN RAFFAELE
    B.5.2Functional name of contact pointUfficio Ricerche Cliniche – Stem Ce
    B.5.3 Address:
    B.5.3.1Street AddressVia Olgettina, 60
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20132
    B.5.3.4CountryItaly
    B.5.4Telephone number02-26434289
    B.5.5Fax number02-26434760
    B.5.6E-mailurc.scp@hsr.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 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 nameTemferon
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTemferon
    D.3.9.2Current sponsor codeTemferon
    D.3.9.3Other descriptive nameHSPC autologhe geneticamente modificate con vettore lentivirale TIA126-LV, codificante per cDNA hIFN-2
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number2500000 to 10000000
    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 No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.2.1.1.1Trade name MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML
    D.2.1.1.2Name of the Marketing Authorisation holderCHUGAI SANOFI AVENTIS
    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 nameLENOGRASTIM
    D.3.2Product code LENOGRASTIM
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLENOGRASTIM
    D.3.9.2Current sponsor codeLENOGRASTIM
    D.3.9.3Other descriptive nameLENOGRASTIM
    D.3.10 Strength
    D.3.10.1Concentration unit million IU million international units
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number34
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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: 3
    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 MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO
    D.2.1.1.2Name of the Marketing Authorisation holderGENZYME EUROPE B.V.
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/4/227
    D.3 Description of the IMP
    D.3.1Product namePLERIXAFOR
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPLERIXAFOR
    D.3.9.2Current sponsor codePLERIXAFOR
    D.3.9.3Other descriptive namePLERIXAFOR
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 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 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: 4
    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 ENDOXAN BAXTER - 500 MG POLVERE PER SOLUZIONE INIETTABILE 1 FLACONE VETRO TIPO III 500 MG
    D.2.1.1.2Name of the Marketing Authorisation holderBAXTER 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 nameCICLOFOSFAMIDE
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCICLOFOSFAMIDE
    D.3.9.2Current sponsor codeCICLOFOSFAMIDE
    D.3.9.3Other descriptive nameCICLOFOSFAMIDE
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 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 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: 5
    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 REVLIMID - 25 MG CAPSULA RIGIDA - USO ORALE BLISTER (PCTFE/PVC/ALU) 21 CAPSULE
    D.2.1.1.2Name of the Marketing Authorisation holderCELGENE EUROPE LIMITED
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/03/177
    D.3 Description of the IMP
    D.3.1Product nameLENALIDOMIDE
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLENALIDOMIDE
    D.3.9.2Current sponsor codeLENALIDOMIDE
    D.3.9.3Other descriptive nameLENALIDOMIDE
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 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 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: 6
    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 VELCADE - 1 FLACONCINO DA 3.5 MG
    D.2.1.1.2Name of the Marketing Authorisation holderJANSSEN-CILAG INTERNATIONAL N.V.
    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 nameBORTEZOMIB
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBORTEZOMIB
    D.3.9.2Current sponsor codeBORTEZOMIB
    D.3.9.3Other descriptive nameBORTEZOMIB
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3.5
    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 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 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: 7
    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 SOLDESAM - 0.2% GOCCE ORALI, SOLUZIONE FLACONE 10 ML
    D.2.1.1.2Name of the Marketing Authorisation holderLABORATORIO FARMACOLOGICO MILANESE S.R.L.
    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 nameDESAMETASONE
    D.3.4Pharmaceutical form Oral drops, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDESAMETASONE
    D.3.9.2Current sponsor codeDESAMETASONE
    D.3.9.3Other descriptive nameDESAMETASONE
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.2
    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 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 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: 8
    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 ALKERAN - 50 MG/10 ML POLVERE E SOLVENTE PER SOLUZIONE INIETTABILE 1 FLACONCINO POLVERE + 1 FLACONCINO SOLVENTE DA 10 ML
    D.2.1.1.2Name of the Marketing Authorisation holderASPEN PHARMA TRADING LIMITED
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/15/1463
    D.3 Description of the IMP
    D.3.1Product nameMELFALAN
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMELFALAN
    D.3.9.2Current sponsor codeMELFALAN
    D.3.9.3Other descriptive nameMELFALAN
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 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 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: 9
    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 ZARZIO - 30MU (60MU/ML) - SOLUZIONE INIETTABILE O PER INFUSIONE - USO SOTTOCUTANEO O ENDOVENOSO-SIRINGA PRERIEMPITA (VETRO) 0.5 ML 1 SIRINGA PRERIEMPITA
    D.2.1.1.2Name of the Marketing Authorisation holderSANDOZ GMBH
    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 nameFILGRASTIM
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNFILGRASTIM
    D.3.9.2Current sponsor codeFILGRASTIM
    D.3.9.3Other descriptive nameFILGRASTIM
    D.3.10 Strength
    D.3.10.1Concentration unit Munit million units
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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
    Multiple myeloma in early relapse after intensive front line therapy
    Mieloma multiplo recidivato precocemente dopo trattamenti intensivi di prima linea
    E.1.1.1Medical condition in easily understood language
    Multiple myeloma in early relapse after intensive front line therapy
    Mieloma multiplo recidivato precocemente dopo trattamenti intensivi di prima linea
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10028228
    E.1.2Term Multiple myeloma
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate safety and tolerability of escalating doses of Temferon in multiple myeloma patients with early relapse after intensive front line therapy.
    Valutare la sicurezza e la tollerabilità di dosi crescenti di Temferon in pazienti con mieloma multiplo recidivato precocemente dopo terapia intensiva in prima linea.
    E.2.2Secondary objectives of the trial
    To evaluate biological activity and efficacy of escalating doses of Temferon as maintenance/consolidation treatment in multiple myeloma patients with early relapse after intensive front line therapy.
    Valutare l'attività biologica e l'efficacia di dosi crescenti di Temferon come trattamento di mantenimento/consolidamento in pazienti con mieloma multiplo recidivato precocemente dopo terapia intensiva in prima linea.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Multiple myeloma patients with early relapse after intensive front-line treatment and disease measurable by serum biomarkers that have obtained at least a VGPR after second-line salvage treatment;
    2. Able and willing to provide written informed consent;
    3. Able to comply with study protocol and procedures;
    4. Male or Female;
    5. Age 18-70 years;
    6. Fit patients: Performance status scores: ECOG <2 and Karnofsky > 70%; Life expectancy of ≥ 6 months;
    7. Adequate cardiac, renal, hepatic and pulmonary functions;
    8. Women of child-bearing potential enrolled in the study must have a negative pregnancy test at screening and agree to use acceptable methods of contraception during the trial;
    9. Men enrolled in the study with partners who are women of child bearing potential, must be willing to use an acceptable barrier contraceptive method during the trial or have undergone successful vasectomy at least 6 months prior to entry into the study.
    1. Pazienti con mieloma multiplo recidivato precocemente dopo trattamento intensivo in prima linea, malattia misurabile mediante biomarcatori sierici e che hanno ottenuto una VGPR dopo un trattamento di salvataggio in seconda linea;
    2. Pazienti in grado e disposti a fornire il consenso informato scritto;
    3. Pazienti in grado di rispettare il protocollo di studio e le procedure;
    4. Maschi e femmine;
    5. Età 18-70 anni;
    6. Pazienti in salute: ECOG < 2 e Karnofsky >70%, aspettativa di vita ≥ 6 mesi;
    7. Adeguate funzioni cardiache, renali, epatiche e polmonari;
    8. Le donne in età fertile arruolate nello studio devono sottoporsi a un test di gravidanza negativo allo screening e accettare di utilizzare metodi contraccettivi accettabili durante lo studio;
    9. Gli uomini arruolati nello studio con partner che sono donne potenzialmente fertili, devono essere disposti a utilizzare un metodo contraccettivo di barriera accettabile durante lo studio o aver subito una vasectomia di successo almeno 6 mesi prima dell'ingresso nello studio.
    E.4Principal exclusion criteria
    1. Use of other investigational agents within 4 weeks prior to experimental treatment (within 6 weeks if use of long-acting agents);
    2. Severe active viral, bacterial, or fungal infection at eligibility evaluation;
    3. Active autoimmune disease or a history of clinically relevant autoimmune manifestations requiring immunosuppressive treatment, i.e. psoriasis, systemic lupus erythematosus, rheumatoid arthritis, vasculitis, immune-mediated peripheral neuropathies;
    4. Sarcoidosis requiring active steroid or immunosuppressive treatment;
    5. Primary amyloidosis;
    6. History of neuropsychiatric illness including severe depression, schizophrenia, bipolar disorders, impaired cognitive function, dementia or suicidal tendency;
    7. Neuropathy > grade 2;
    8. History of severe cardiovascular disease such as prior stroke, coronary artery disease requiring intervention, unresolved arrhythmias;
    9. Malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or family history of familial cancer syndromes;
    10. Myelodysplasia, cytogenetic or molecular alterations specifically associated with clonal hematopoiesis of the myeloid lineage, or other serious hematological disorder other than the plasma cell dyscrasia;
    11.Other clinical conditions judged by the Investigator non-compatible with the study procedures;
    12. Positivity for HIV-1, HIV-2 (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (or negative HCV RNA but on antiviral treatment) and/or Treponema Pallidum or Mycoplasma active infection;
    13. Active alcohol or substance abuse within 6 months of the study;
    14. Pregnancy or lactation;
    15. Previous allogeneic bone marrow transplantation, kidney or liver transplant, or gene therapy;
    16. Prior to conditioning: inability to meet the target mobilization cell number after at least 2 attempts of HSPC collection.
    1. Uso di altri agenti investigativi nelle 4 settimane precedenti il trattamento sperimentale (entro 6 settimane se si usano agenti a lunga durata d'azione);
    2. Grave infezione virale, batterica o fungina attiva alla valutazione dell'idoneità;
    3. Malattia autoimmune attiva o una storia rilevante di importanti manifestazioni autoimmuni, in particolare psoriasi, sarcoidosi, lupus eritematoso sistemico, artrite reumatoide, vasculite, neuropatie periferiche immuno-mediate;
    4. Sarcoidosi che richiede trattamento attivo con steroidi o uso di agenti immunosuppressivi;
    5. Amiloidosi primaria;
    6. Storia di malattia neuropsichiatrica inclusa depressione, schizofrenia, disturbi bipolari, compromissione della funzione cognitiva, demenza o tendenza al suicidio;
    7. Neuropatia> grado 2;
    8. Anamnesi di gravi malattie cardiovascolari come un precedente ictus, malattia coronarica che richiede un intervento, aritmie non risolte;
    9. Neoplasie maligne (eccetto il tumore cutaneo locale o neoplasia intraepiteliale cervicale) o storia familiare di sindromi da cancro familiare;
    10. Mielodisplasia, alterazioni citogenetiche o molecolari specificatamente associate all'ematopoiesi clonale del lignaggio mieloide o altra grave patologia ematologica diversa dalla discrasia delle plasmacellule;
    11. Altre condizioni cliniche giudicate dallo sperimentatore non compatibili con le procedure dello studio;
    12. Positività per HIV-1, HIV-2 (sierologia o RNA) e/o HbsAg e/o HBV-DNA e/o HCV-RNA (o RNA HCV negativo ma con trattamento antivirale) e/o infezione da Treponema Pallidum o Mycoplasma;
    13. Uso attivo o abuso di alcool o altre sostanze entro 6 mesi dallo studio;
    14. Gravidanza o allattamento;
    15. Precedente trapianto di midollo osseo allogenico, trapianto di rene o fegato o terapia genica;
    16. Prima del condizionamento: incapacità, in seguito a mobilizzazione, di raggiungere il numero target di cellule HSPC dopo almeno 2 tentativi di raccolta.
    E.5 End points
    E.5.1Primary end point(s)
    - Engraftment of Temferon after reduced intensity conditioning (vector copy number [VCN] > 0.07 in myeloid cells between Day +30 and Day +100 in at least 2 patients);
    - The proportion of patients achieving hematologic recovery by Day +30 from ASCT with non-manipulated autologous HSPC and infusion of Temferon;
    - Safety of Temferon measured through the 24 months post Temferon infusion.
    - Attecchimento di Temferon dopo regime di condizionamento a ridotta intensità (numero di copie vettore [VCN]> 0,07 nelle cellule mieloidi tra il giorno +30 e il giorno +100 in almeno 2 pazienti);
    - Proporzione di pazienti che hanno ottenuto il recupero ematologico entro il giorno +30 dalla somministrazione di cellule staminali e progenitori (HSPC) autologhi non manipolati 5 e dall’infusione di Temferon.;
    - Sicurezza di Temferon valutata nei 24 mesi successivi alla sua infusione.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Multiple timepoints up to 2 years
    Multipli timepoints fino a 2 anni
    E.5.2Secondary end point(s)
    - Presence of an IFN-alfa gene signature in the bone marrow (BM) of Temferon-treated patients.
    - Presence of transduced myeloid cells in BM aspirate and peripheral blood (PB) at Day +30 and Day +100 post Temferon infusion as determined by VCN.
    - Persistence of transduced myeloid cells in PB and BM as assessed by VCN for at least 12 weeks
    - Overall response rate (ORR)
    - Fraction of patients achieving complete response (CR) with minimal residual disease (MRD) levels ≤ 10-5 and 10-6 on BM aspirate
    - Duration of maintenance of response during the 24 months post Temferon infusion
    - Progression-free survival (PFS) and overall survival (OS) at 24 months post Temferon infusion.
    - Change in health-related quality of life (HRQOL) at 6, 12 and 24 months post Temferon infusion compared to screening.
    - Change over time in performance status scores (ECOG and Karnofsky) through 24 months post Temferon infusion.
    - Induzione, nel midollo osseo (BM) dei pazienti trattati con Temferon di geni IFN-responsivi.
    - Presenza di cellule mieloidi trasdotte nell'aspirato midollare (BM) e nel sangue periferico (PB) al giorno +30 e giorno +100 dopo l'infusione di Temferon.
    - Persistenza di cellule mieloidi trasdotte in PB e BM valutata mediante VCN per almeno 12 settimane.
    - Tasso di risposta obiettiva (ORR)
    - Frazione di pazienti che raggiungono una risposta completa (CR) con malattia minima residua (MRD) ≤ 10-5 e 10-6 valutata su aspirato midollare.
    - Durata del mantenimento della risposta nei 24 mesi successivi all'infusione di Temferon
    - Sopravvivenza libera da progressione (PFS) e sopravvivenza globale (OS) a 24 mesi dall'infusione di Temferon.
    - Cambiamento della qualità della vita correlata alla salute (HRQOL) a 6, 12 e 24 mesi dall'infusione di Temferon rispetto allo screening.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Multiple timepoints up to 2 years
    Multipli timepoints fino a 2 anni
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    A dosi crescenti
    Dose escalation
    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.8.4.1Number 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 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
    LVLS
    LVLS
    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 months8
    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 months8
    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 No
    F.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 9
    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)
    Patients will be invited to participate in a long term follow-up study for an additional 6 years.
    Al termine della sperimentazione, i pazienti saranno invitati a partecipare ad uno studio di follow-up a lungo termine di 6 anni.
    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 Decision2018-11-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-13
    P. End of Trial
    P.End of Trial StatusOngoing
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