Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43861   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2018-001145-14
    Sponsor's Protocol Code Number:ALD-104
    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:2021-08-02
    Trial results View 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 number2018-001145-14
    A.3Full title of the trial
    A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects inferior or equal to 17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)
    Studio di fase 3 del prodotto farmacologico Lenti-D dopo condizionamento mieloablativo usando busulfano e fludarabina in soggetti di età minore o uguale a 17 anni affetti da adrenoleucodistrofia cerebrale (CALD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD)
    Studio clinico per valutare l'efficacia e la sicurezza della terapia genica per il trattamento della adrenoleucodistrofia cerebrale (CALD)
    A.3.2Name or abbreviated title of the trial where available
    ALD-104
    ALD-104
    A.4.1Sponsor's protocol code numberALD-104
    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.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationbluebird bio, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address60 Binney Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number0013394999300
    B.5.5Fax number00000000
    B.5.6E-mailclinicaltrials@bluebirdbio.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 numberEU/3/12/1003
    D.3 Description of the IMP
    D.3.1Product nameLenti-D Drug Product
    D.3.2Product code [not applicable]
    D.3.4Pharmaceutical form Dispersion 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 INNelivaldogene autotemcel
    D.3.9.2Current sponsor codeLenti-D Drug Product
    D.3.9.3Other descriptive nameAutologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA
    D.3.9.4EV Substance CodeSUB127987
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number50000000
    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/988313/2011
    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
    Cerebral Adrenoleukodystrophy (CALD)
    adrenoleucodistrofia cerebrale (CALD)
    E.1.1.1Medical condition in easily understood language
    Cerebral Adrenoleukodystrophy (CALD)
    adrenoleucodistrofia cerebrale (CALD)
    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 PT
    E.1.2Classification code 10051260
    E.1.2Term Adrenoleukodystrophy
    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 evaluate the efficacy and safety of Lenti-D Drug Product after myeloablative conditioning with busulfan and fludarabine in subjects with CALD
    Valutare l’efficacia e la sicurezza del prodotto farmacologico Lenti-D dopo condizionamento mieloablativo con busulfano e fludarabina in soggetti affetti da CALD
    E.2.2Secondary objectives of the trial
    Not applicable
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent is obtained from a competent custodial parent or guardian
    with legal capacity to execute a local IRB/IEC approved consent. Informed
    assent will be sought from capable subjects, in accordance with the directive
    of the IRB/IEC and with local requirements.
    2. Males aged 17 years and younger, at the time of parental/guardian consent
    and, where appropriate, subject assent.
    3. Active cerebral ALD as defined by:
    a. Elevated VLCFA values, and
    b. Active central nervous system (CNS) disease established by central
    radiographic review of brain MRI demonstrating
    i. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
    ii. Gadolinium enhancement on MRI of demyelinating lesions.
    4. Neurologic Function Score (NFS) = 1
    1. Il consenso informato è ottenuto da un genitore o da un tutore a cui è affidata la custodia con capacità giuridica di attuare un consenso approvato dal CE locale indipendente. I soggetti idonei dovranno fornire l’assenso informato, in conformità con la direttiva del CE indipendente e con tutti gli altri requisiti locali.
    2. Essere di sesso maschile e avere un’età massima di 17 anni al momento del consenso fornito dal genitore/tutore e, ove appropriato, dell’assenso fornito dal soggetto.
    3. ALD cerebrale attiva definita da:
    a. Valori elevati di acidi grassi a catena molto lunga (VLCFA), e
    b. Malattia del sistema nervoso centrale (SNC) attiva stabilito da esame radiografico centrale del cervello con RMI che dimostra un
    i. Punteggio di Loes tra 0,5 e 9 (compresi) sulla scala di 34 punti eii. Aumento di gadolinio su RMI di lesioni demielinizzanti.
    4. Punteggio di funzione neurologica (NFS) =1.
    E.4Principal exclusion criteria
    2. Use of statins, Lorenzo’s Oil, or dietary regimens used to lower VLCFA levels.
    Note: subjects must discontinue use of these medications at time of consent.
    3. Receipt of an investigational study drug or procedure within 3 months before
    Screening that might confound study outcomes. Use of investigational study drugs
    is prohibited throughout the course of the study.
    4. Any conditions that make it impossible to perform MRI studies (including allergies
    to anesthetics or contrast agents).
    5. Hematological compromise as evidenced by:
    a. Peripheral blood ANC count < 1500 cells/mm3, and either
    b. Platelet count < 100,000 cells/mm3 or
    c. Hemoglobin < 10 g/dL.
    6. Hepatic compromise as evidenced by:
    a. Aspartate transaminase (AST) value >2.5 × ULN
    b. Alanine transaminase (ALT) value >2.5 × ULN
    c. Total bilirubin value >3.0 mg/dL, except if there is a diagnosis of
    Gilbert’s Syndrome and the subject is otherwise stable
    7. Baseline estimated glomerular filtration rate <70 mL/min/1.73 m2, as determined
    using the Chronic Kidney Disease Epidemiology Collaboration creatinine equation
    (see https://www.kidney.org/professionals/KDOQI/gfr_calculatorPed)
    8. Cardiac compromise as evidenced by left ventricular ejection fraction <40%
    9. Immediate family member with a known or suspected Familial Cancer Syndrome
    (including but not limited to hereditary breast and ovarian cancer syndrome,
    hereditary non-polyposis colorectal cancer syndrome, and familial adenomatous
    polyposis).
    10. Clinically significant uncontrolled, active bacterial, viral, fungal, parasitic, or
    prionassociated
    infection.
    11. Positive for human immunodeficiency virus type 1 or 2 (HIV-1, HIV-2); hepatitis B
    virus (HBV); hepatitis C virus (HCV); human T lymphotrophic virus 1 (HTLV-1).
    (Note that subjects who have been vaccinated against HBV [positive for HBV
    surface antibodies] who are negative for other markers of prior HBV infection
    [e.g., negative for HBV core Ab] are eligible. Subjects with past exposure to
    HBV [HBcAb positive and/or HBeAb positive] are also eligible for the study
    provided they have a negative test for HBV DNA. Also note that subjects who are
    positive for anti-hepatitis C Ab are eligible as long as they have a negative hepatitis
    C viral load).
    12. Any clinically significant cardiovascular, hematological, or pulmonary
    disease, or other disease or condition that would be contraindicated for any of the other study procedures..
    13. Absence of adequate contraception for fertile subjects. Male subjects and their
    female partners are required to use two different effective methods of contraception
    from Screening through at least 6 months after Lenti-D Drug Product infusion. If
    subjects are truly sexually abstinent (where true sexual abstinence is defined as
    being in line with the preferred and usual lifestyle of the subject), no second method
    is required.
    14. Any contraindications to the use of G-CSF or plerixafor during the mobilization of
    hematopoietic stem cells, and any contraindications to the use of busulfan or
    fludarabine, including known hypersensitivity to the active substances or to any of the
    excipients in their formulations.
    15. Known hypersensitivity to protamine sulfate.
    1. Aver ricevuto in precedenza un trapianto allogenico o una terapia genetica.
    2. Uso di statine, olio di Lorenzo o regime alimentare finalizzato all’abbassamento dei
    livelli di VLCFA. Nota: i soggetti devono interrompere l’utilizzo di tali farmaci al
    momento del consenso.
    3. Aver ricevuto un farmaco in studio sperimentale o una procedura entro 3 mesi prima
    dello screening che possa alterare l’esito dello studio. L’utilizzo di farmaci in studio
    sperimentali è vietato durante tutto il corso dello studio.
    4. Qualsiasi condizione che rende impossibile effettuare studi con RMI (incluse
    allergie ad anestetici o a mezzi di contrasto).
    Compromissione ematologica come evidenziato da:
    a. Conta assoluta dei neutrofili nel sangue periferico <1500 cellule/mm3, e
    b. Conta piastrinica <100.000 cellule/mm3 oppure
    c. Emoglobina <10 g/dl.
    6. Compromissione epatica come evidenziato da:
    a. Valore transaminasi aspartica (AST) >2,5 x ULN
    b. Valore transaminasi alaninica (ALT) >2,5 x ULN
    c. Valore bilirubina totale >3,0 mg/dl, eccetto il caso in cui visia una diagnosi di
    sindrome di Gilbert e il soggetto sia altrimenti stabile
    7. Velocità di filtrazione glomerulare stimata al basale <70 ml/min/1,73 m2,
    determinata mediante l’equazione per la creatinina della CKD-EPI (Chronic Kidney Disease
    Epidemiology Collaboration) (consultare https://www.kidney.
    org/professionals/KDOQI/gfr_calculatorePed)
    8. Compromissione cardiaca come evidenziato da frazione di eiezione ventricolare
    sinistra <40%
    9. Parente prossimo con sindrome tumorale familiare nota o sospetta (tra cui, in modo
    non limitativo, sindrome del tumore mammario e ovarico ereditario, sindrome del tumore
    colorettale ereditario non poliposico e poliposi adenomatosa familiare).
    10. Infezioni batteriche non controllate, virali, micotiche, parassitarie o associate a
    prioni clinicamente significative e attive.
    11. Positività per presenza di virus dell’immunodeficienza umana di tipo 1 o 2 (HIV-1,
    HIV-2), dell’epatite B (HBV), virus dell’epatite C (HCV), virus T-linfotrofico dell’uomo
    di tipo 1 (HTLV-1). (Si noti che i soggetti che sono stati vaccinati contro il virus
    dell’epatite B [positività per anticorpi di superficie epatite B] che sono negativi per
    gli altri marcatori di una precedente infezione di epatite B [per es., negativi per l’
    antigene core dell’epatite B] sono idonei. Anche i soggetti con una pregressa
    esposizione all’epatite B [positività all’HBcAB e/o positività all’HBeAb] sono idonei
    per lo studio purché abbiano un test negativo per il DNA dell’epatite B. Si noti inoltre
    che i soggetti che risultano positivi per l’anti HCVAb sono idonei fino a quando
    continuano ad avere una carica virale negativa dell’epatite C).
    12. Qualsiasi patologia cardiovascolare, ematologica o polmonare o altre patologie o malattia clinicamente significativa sarebbe controindicata per tutte le procedure dello studio.
    13. Assenza di contraccettivi adeguati per i soggetti in età fertile. Ai soggetti di
    sesso maschile e alle loro partner di sesso femminile viene richiesto di utilizzare due
    diversi metodi contraccettivi efficaci a partire dallo screening fino ad almeno 6 mesi
    dopo l’infusione del prodotto farmacologico Lenti-D. Se i soggetti praticano
    effettivamente l’astinenza sessuale, (ove l’effettiva astinenza sessuale è intesa in
    linea con lo stile di vita di preferenza e abituale del soggetto), non è richiesto un
    secondo metodo.
    14. Eventuali controindicazioni all’uso di G-CSF o plerixafor durante la mobilizzazione
    delle cellule staminali emopoietiche e qualsiasi controindicazione all’uso di busulfano
    o fludarabina, inclusa l’ipersensibilità alle sostanze attive o a qualsiasi eccipiente.
    15. Nota ipersensibilità al solfato di protamina.
    E.5 End points
    E.5.1Primary end point(s)
    Secondary efficacy endpoints are the following:
    • Proportion of subjects without gadolinium enhancement on MRI (i.e., GdE-) at Month
    24
    • Value and change in total NFS from Baseline to protocol scheduled visits
    • MFD-free survival over time
    • Overall survival
    • Detectable vector copy number (VCN) in peripheral blood cells by
    Month 6
    The secondary safety endpoints are the following:
    • The proportion of subjects who experience either acute (=Grade II) or chronic GVHD
    at Month 24
    • Time to neutrophil engraftment after drug product infusion
    • The proportion of subjects with platelet engraftment by Month 24
    • Time to platelet engraftment post-drug product infusion
    • The proportion of subjects with loss of neutrophil engraftment postdrug product
    infusion by Month 24
    • The proportion of subjects who undergo a subsequent HSC infusion by Month 24
    • The proportion of subjects who experience transplant-related mortality through 100
    and 365 days post-drug product infusion
    • Proportion of subjects with clinical > = Grade 3 AEs, all investigational medicinal
    product (IMP)-related AEs, all SAEs, > = Grade 3 infections, and clinically
    significant changes in laboratory parameters by Month 24
    • The proportion of subjects who experience > = Grade II acute GVHD by Month 24
    • The proportion of subjects who experience chronic GVHD by Month 24
    • Number of emergency room visits (post-neutrophil engraftment) by
    Month 24
    • Number and duration of in-patient hospitalizations (post-neutrophil
    engraftment) by Month 24
    • Number and duration of ICU stays (post-neutrophil engraftment) by Month 24
    • The number of subjects in which vector-derived RCL is detected by Month 24
    • The number of subjects with insertional oncogenesis (myelodisplasia, leukemia, lymphoma, etc) by
    Month 24
    • The number of subjects with clonal predominance by Month 24
    Gli endpoint di efficacia secondari sono:
    • Parte dei soggetti senza aumento di gadolino su RMI (per es., GdE-) al mese 24
    • Valori e variazioni nelle NFS totali dal basale fino alle visite previste dal
    protocollo
    • Sopravvivenza priva delle principali invalidità funzionali nel corso del tempo
    • Sopravvivenza complessiva
    • Numero di copie del vettore (VCN) riscontrabile nelle cellule del sangue periferico
    entro il mese 6
    Gli endpoint sulla sicurezza secondari sono:
    • Percentuale di soggetti che manifestano GVHD acuta (di grado > = II) o cronica al
    Mese 24
    • Tempo di attecchimento neutrofili in seguito a infusione con il prodotto
    farmacologico
    • Percentuale di soggetti con attecchimento piastrinico entro il mese 24
    • Tempo di attecchimento piastrinico in seguito a infusione con il prodotto
    farmacologico
    • Percentuale di soggetti con perdita di attecchimento neutrofili in seguito a
    infusione con il prodotto farmacologico entro il mese 24
    • Percentuale di soggetti che si sottopongono a una ulteriore infusione di HSC entro
    il mese 24
    • Percentuale di soggetti che manifestano mortalità collegata al trapianto fino a 100
    giorni e 365 giorni in seguito a infusione con il prodotto farmacologico
    • Percentuale di soggetti con EA clinici di Grado >= 3, tutti gli EA correlati al
    prodotto medicinale sperimentale (IMP), tutti i SAE, infezioni di Grado > = 3 e
    variazioni clinicamente significative dei parametri di laboratorio entro il mese 24
    • Percentuale di soggetti che manifestano GVHD acuta di Grado >= II entro il mese 24
    • Percentuale di soggetti che manifestano GVDH cronica entro il mese 24
    • Numero di visite al pronto soccorso (in seguito ad attecchimento neutrofili) entro
    il mese 24
    • Numero e durata di ricoveri ospedalieri (in seguito ad attecchimento neutrofili)
    entro il mese 24
    • Numero e durata di permanenza nel reparto di terapia intensiva (ICU) (in seguito ad
    attecchimento neutrofili) entro il mese 24
    • Il numero di soggetti in cui è riscontrato Lentivirus competente per la replicazione
    (RCL) derivato da vettore entro il mese 24
    • Il numero di soggetti con oncogenesi da inserzione (mielodisplasia, leucemia, linfoma, ecc.)
    entro il mese 24
    • Il numero di soggetti con predominanza clonale entro il
    mese 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 24
    24 mesi
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints are the following:
    • Proportion of subjects without gadolinium enhancement on MRI (i.e.,
    GdE-) at Month 24
    • Value and change in total NFS from Baseline to protocol scheduled
    visits
    • MFD-free survival over time
    • Overall survival
    • Detectable vector copy number (VCN) in peripheral blood cells by
    Month 6
    XML File Identifier: nXcQh4t6DxIK3VxazeAZ5rtrWOU=
    Page 15/27
    The secondary safety endpoints are the following:
    • The proportion of subjects who experience either acute (=Grade II) or
    chronic GVHD at Month 24
    • Time to neutrophil engraftment after drug product infusion
    • The proportion of subjects with platelet engraftment by Month 24
    • Time to platelet engraftment post-drug product infusion
    • The proportion of subjects with loss of neutrophil engraftment
    postdrug product infusion by Month 24
    • The proportion of subjects who undergo a subsequent HSC infusion by
    Month 24
    • The proportion of subjects who experience transplant-related mortality
    through 100 and 365 days post-drug product infusion
    • The proportion of subjects who experience =Grade II acute GVHD by
    Month 24
    • The proportion of subjects who experience chronic GVHD by Month 24
    • Number of emergency room visits (post-neutrophil engraftment) by
    Month 24
    • Number and duration of in-patient hospitalizations (post-neutrophil
    engraftment) by Month 24
    • Number and duration of ICU stays (post-neutrophil engraftment) by
    Month 24
    • The number of subjects in which vector-derived RCL is detected by
    Month 24
    • The number of subjects with insertional mutagenesis leading to clonal
    dominance by Month 24
    • The number of subjects with insertional mutagenesis leading to
    leukemia by Month 24
    Gli endpoint di efficacia secondari sono:
    • Parte dei soggetti senza aumento di gadolino su RMI (per es., GdE-) al mese 24
    • Valori e variazioni nelle NFS totali dal basale fino alle visite previste dal protocollo
    • Sopravvivenza priva delle principali invalidità funzionali nel corso del tempo
    • Sopravvivenza complessiva
    • Numero di copie del vettore (VCN) riscontrabile nelle cellule del sangue periferico entro il mese 6
    L’endpoint sulla sicurezza primario è:
    • Percentuale di soggetti con attecchimento di neutrofili dopo l’infusione con il prodotto farmacologico
    Gli endpoint sulla sicurezza secondari sono:
    • Percentuale di soggetti che manifestano GVHD acuta (di grado =II) o cronica al Mese 24
    • Tempo di attecchimento neutrofili in seguito a infusione con il prodotto farmacologico
    • Percentuale di soggetti con attecchimento piastrinico entro il mese 24
    • Tempo di attecchimento piastrinico in seguito a infusione con il prodotto farmacologico
    • Percentuale di soggetti con perdita di attecchimento neutrofili in seguito a infusione con il prodotto farmacologico entro il mese 24
    • Percentuale di soggetti che si sottopongono a una ulteriore infusione di HSC entro il mese 24
    • Percentuale di soggetti che manifestano mortalità collegata al trapianto fino a 100 giorni e 365 giorni in seguito a infusione con il prodotto farmacologico
    • Percentuale di soggetti che manifestano GVHD acuta di grado =II entro il mese 24
    • Percentuale di soggetti che manifestano GVDH cronica entro il mese 24
    • Numero di visite al pronto soccorso (in seguito ad attecchimento neutrofili) entro il mese 24
    • Numero e durata di ricoveri ospedalieri (in seguito ad attecchimento neutrofili) entro il mese 24
    • Numero e durata di permanenza nel reparto di terapia intensiva (ICU) (in seguito ad attecchimento neutrofili) entro il mese 24
    • Il numero di soggetti in cui è riscontrato Lentivirus competente per la replicazione (RCL) derivato da vettore entro il mese 24
    • Il numero di soggetti con mutagenesi da inserzione che porta alla prevalenza clonale entro il mese 24
    • Il numero di soggetti con mutagenesi da inserzione che porta a leucemia entro il mese 24
    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 24
    24 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 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) 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 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.8.2.4Number 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 EEA5
    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
    United States
    France
    Germany
    Italy
    Netherlands
    United Kingdom
    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 months9
    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 months9
    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: 18
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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
    Paediatric patients
    Pazienti Pediatrici
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 11
    F.4.2.2In the whole clinical trial 35
    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)
    Subjects will be asked to enroll into a long-term follow-up study (LTF-304, EudraCT number: 2015-002805-13).
    Ai soggetti verrà chiesto di iscriversi a uno studio di follow-up a lungo termine (LTF-304, EudraCT number: 2015-002805-13).
    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 Decision2019-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-03-20
    P. End of Trial
    P.End of Trial StatusCompleted
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Thu Apr 25 17:12:37 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA