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    Summary
    EudraCT Number:2013-002245-11
    Sponsor's Protocol Code Number:LTF-303
    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:2021-01-07
    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 number2013-002245-11
    A.3Full title of the trial
    Long-term Follow-up of Subjects with Hemoglobinopathies Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
    Follow-up a lungo termine di soggetti affetti da emoglobinopatie trattati con terapia genica ex vivo usando cellule ematopoietiche autologhe trasdotte con un vettore lentivirale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate long term effect of an experimental approach of gene therapy to treat ß-thalassemia major, know also as transfusion dependent ß-thalassemia, and the sickle cell disease.
    Studio per valutare gli effetti a lungo termine di un approccio di trasferimento genico sperimentale per trattare la beta-talassemia grave, indicata anche come ß–talassemia trasfusione dipendente, e la malattia a cellule falciformi grave .
    A.3.2Name or abbreviated title of the trial where available
    LTF-303
    LTF-303
    A.4.1Sponsor's protocol code numberLTF-303
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02633943
    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 SponsorBLUEBIRD BIO, INC.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportbluebird bio, Inc (with its wholly owned subsidiary bluebird bio (Netherlands) B.V.)
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCTI Clinical Trial and Consulting Services Italy
    B.5.2Functional name of contact pointClinical Trial Operations
    B.5.3 Address:
    B.5.3.1Street AddressLargo Richini 6
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20122
    B.5.3.4CountryItaly
    B.5.4Telephone number00390258215530
    B.5.5Fax number00390258215400
    B.5.6E-mailregulatoryitaly@ctifacts.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/146/12
    D.3 Description of the IMP
    D.3.1Product nameLentiGlobin BB305 Drug Product (autologous CD34 cells transduced w/ LentiGlobin BB305)
    D.3.2Product code [LentiGlobin BB305]
    D.3.4Pharmaceutical form Emulsion 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 INNbetibeglogene autotemcel
    D.3.9.1CAS number 1905394-85-5
    D.3.9.2Current sponsor codeLentiGlobin BB305
    D.3.9.3Other descriptive nameUTOLOGOUS CD34+ CELLS TRANSDUCED WITH BB305 VECTOR ENCODING BETA-AT87Q- GLOBIN
    D.3.9.4EV Substance CodeSUB127985
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number20000000
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberEMA/CAT/988303/2011
    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 Yes
    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
    Subjects treated with gene therapy drug products in a bluebird bio-sponsored study will be invited to participate in this long-term follow-up study to monitor the safety and efficacy of the drug products. To date, subjects in this study have been treated with bluebird bio gene therapy drug products developed to treat the following indications:

    beta-thalassaemia major
    Sickle cell disease
    Ai soggetti trattati con il prodotto medicinale in uno studio clinico sponsorizzato
    da bluebird bio sarà chiesto di partecipare a questo studio di follow-up a lungo termine per monitorare la sicurezza ed efficacia del prodotto medicinale. Ad oggi, i soggetti in questo studio sono stati trattati con il prodotto medicinale di terapia genica blubirdbio sviluppato per il trattamento delle seguente indicazioni:
    ß-talassemia grave
    malattia a cellule falciformi
    E.1.1.1Medical condition in easily understood language
    See above.
    Si veda sopra
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10055579
    E.1.2Term Sickle-cell beta thalassemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Monitor the long-term safety and efficacy of gene therapy drug products used in bluebird bio-sponsored clinical studies in treated subjects with hemoglobinopathies.
    Monitor the long term efficacy of the drug product.
    Monitorare la sicurezza a lungo termine del prodotto medicinale di
    terapia genica usato in studi clinici sponsorizzati da bluebird bio in soggetti
    trattati affetti da emoglobinopatie.
    Monitorare l’efficacia a lungo termine del prodotto medicinale.
    E.2.2Secondary objectives of the trial
    Not Applicable
    Non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provision of written informed consent for this study by subject, or as applicable, subject’s parent(s)/ legal guardian(s).
    2. Previously treated with drug product for therapy of a hemoglobinopathy in a bluebird bio-sponsored clinical study.
    3. Able to comply with study requirements
    1. Rilascio del consenso informato scritto per questo studio da parte del soggetto
    o, se pertinente, del/i genitore/i/tutore/i legale/i del soggetto
    2. Trattamento con un prodotto medicinale per la terapia di una emoglobinopatia
    in uno studio clinico sponsorizzato da bluebird bio
    3. Capacità di attenersi ai requisiti dello studio
    E.4Principal exclusion criteria
    1.Met the VCN discontinuation criterion, as follows:
    Undetectable VCN (<0.0003 copies per diploid geome) in peripheral blood cells for 2 consecutive measurements at least 1 month apart and at least 12 months after drug product infusion.
    1. Soddisfacimento del criterio di interruzione della misurazione del VCN nello
    studio originario, come segue: il soggetto presenta VCN non rilevabile
    (<0,0003 copie per genoma diploide) nelle cellule del sangue periferico per 2
    misurazioni consecutive eseguite a distanza di almeno 1 mese e almeno 12
    mesi dopo l’infusione del prodotto medicinale
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints include:
    - Overall survival
    - All drug product-related adverse events (AEs) through Year 15 post hematopoietic stem cell transplantation (HSCT)
    - All serious adverse events (SAEs) through Year 15 post-HSCT
    - Incidence of vector-derived RCL, assessed from archived samples as clinically-indicated
    - Characterization of events of insertional mutagenesis leading to clonal dominance or leukemia
    -Laboratory tests as specified

    Efficacy evaluations will be performed throughout the study and longterm efficacy will be assessed, as feasible, based on data collected in this study.
    Gli endpoint di sicurezza includono:
    • Sopravvivenza complessiva
    • Tutti gli EA correlati al prodotto medicinale fino all’Anno 15 post-HSCT
    • Tutti i SAE fino all’Anno 15 post-HSCT (indipendentemente dalla
    correlazione con il prodotto medicinale)
    • Incidenza di RCL derivati dal vettore, valutata dai campioni d’archivio
    come clinicamente indicato
    • Caratterizzazione degli eventi di mutagenesi inserzionale che conducono
    a dominanza clonale o leucemia
    • Esami di laboratorio come specificato

    Le valutazioni di efficacia saranno eseguite per tutta la durata dello studio e
    l’efficacia a lungo termine sarà valutata, se fattibile, in base ai dati raccolti durante
    questo studio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Follow-up visits are scheduled every 6 months through Year 5 (Month 60) post-transplant, and then annually thereafter through Year 15 post-transplant.
    Le visite di follow up sono pianificate ogni 6 mesi fino al 5o anno (mese 60) post-trapianto, e poi annualmente dopo il 15o anno dopo il trapianto.
    E.5.2Secondary end point(s)
    Not Applicable
    Not Applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not Applicable
    Not Applicable
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open 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 Yes
    E.8.1.7.1Other trial design description
    Long-term follow up
    Long-term follow up
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Thailand
    United States
    France
    Germany
    Greece
    Italy
    United Kingdom
    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 years13
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years13
    E.8.9.2In all countries concerned by the trial months0
    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) No
    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: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 80
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 53
    F.4.2.2In the whole clinical trial 94
    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.
    Nessuno
    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 Decision2019-05-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-26
    P. End of Trial
    P.End of Trial StatusOngoing
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