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    Summary
    EudraCT Number:2015-002805-13
    Sponsor's Protocol Code Number:LTF-304
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-05-12
    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 number2015-002805-13
    A.3Full title of the trial
    Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
    Follow-up a lungo termine di soggetti con adrenoleucodistrofia cerebrale che sono stati trattati con il prodotto medicinale Lenti-D
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
    Follow-up a lungo termine di soggetti con adrenoleucodistrofia cerebrale che sono stati trattati con il prodotto medicinale Lenti-D
    A.3.2Name or abbreviated title of the trial where available
    LTF-304
    LTF-304
    A.4.1Sponsor's protocol code numberLTF-304
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02698579
    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.5.6E-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 [N/A]
    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 CD34+ CELLS TRANSDUCED WITH LENTI-D VECTOR ENCODING 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 numberClassification: Gene Therapy Medicinal Product Reference Number: EMA/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] - Nutritional and Metabolic Diseases [C18]
    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
    - Monitor for long-term safety of the Lenti-D Drug Product (also known as elivaldogene autotemcel; hereafter referred to as eli-cel) administered in parent clinical studies.
    - Monitor for long-term efficacy of eli - cel administered in parent clinical studies.
    • Monitorare la sicurezza a lungo termine del prodotto medicinale Lenti-D (noto anche come elivaldogene autotemcel; di seguito indicato come eli-cel) somministrato negli studi clinici originari.
    • Monitorare l’efficacia a lungo termine di eli-cel somministrato negli studi clinici originari.
    E.2.2Secondary objectives of the trial
    Not Applicable
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Provision of written informed consent for this study by the subject or subject’s parent(s)/ legal guardian(s) and written informed assent by subject, if applicable
    2.Have received eli-cel Drug Product in a parent clinical study.
    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 e assenso informato scritto da parte del soggetto
    2. Aver ricevuto eli-cel in uno studio clinico originario
    E.4Principal exclusion criteria
    There are no exclusion criteria for this Study.
    Non vi sono criteri di esclusione per questo studio.
    E.5 End points
    E.5.1Primary end point(s)
    • The number of subjects who experience graft versus host disease (GVHD) through 15 years post-drug product infusion
    • The number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) through 15 years post drug product infusion
    • The number of subjects with new or worsening hematologic disorders through 15 years post-drug product infusion
    • The number of subjects with new or worsening neurologic disorders through 15 years post-drug product infusion
    • The number of subjects with malignancies through 15 years post-drug product infusion
    • Major functional disability (MFD)-free survival over time through Year 15 post-drug product infusion
    • Numero di soggetti che manifestano malattia del trapianto contro l’ospite (GVHD) per 15 anni dopo l’infusione del prodotto medicinale
    • Numero di soggetti con EA immuno-correlati (per es. disturbi autoimmuni, GVHD, infezioni opportunistiche, HIV) per 15 anni dopo l’infusione del prodotto medicinale
    • Numero di soggetti con disturbi ematologici nuovi o in peggioramento per 15 anni dopo l’infusione del prodotto medicinale
    • Numero di soggetti con disturbi neurologici nuovi o in peggioramento per 15 anni dopo l’infusione del prodotto medicinale
    • Numero di soggetti con neoplasie maligne per 15 anni dopo l’infusione del prodotto medicinale
    • Sopravvivenza libera da disabilità funzionale maggiore (MFD) nel tempo, fino all’Anno 15 dopo l’infusione del prodotto medicinale
    E.5.1.1Timepoint(s) of evaluation of this end point
    In Study LTF-304, follow-up visits are scheduled every 6 months through Year 5 post-drug product infusion, and then annually thereafter through Year 15 post-drug product infusion.
    Le visite di follow-up nello studio LTF-304 sono programmate almeno ogni 6 mesi fino all’Anno 5 successivamente all’infusione del prodotto medicinale e in seguito ogni anno fino all’Anno 15 successivamente all’infusione del prodotto medicinale.
    E.5.2Secondary end point(s)
    • Change from Baseline (defined in parent study) through Year 15 post-drug product infusion in neurologic function score (NFS)
    • The number of subjects without gadolinium enhancement (GdE) on MRI over time through Year 15 post-drug product infusion
    • The number of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) through 15 years post-drug product infusion
    • Variazione dal basale (definita nello studio originario) fino all’Anno 15 dopo l’infusione del prodotto medicinale nel punteggio della funzione neurologica (NFS)
    • Numero di soggetti che non presentano potenziamento del gadolinio (GdE) alla RM nel tempo, fino all’Anno 15 dopo l’infusione del prodotto medicinale
    • Numero di soggetti sottoposti a successivo trapianto di cellule staminali (ovvero, seconda infusione di CSE) per 15 anni dopo l’infusione del prodotto medicinale
    E.5.2.1Timepoint(s) of evaluation of this end point
    In Study LTF-304, follow-up visits are scheduled every 6 months through Year 5 post-drug product infusion, and then annually thereafter through Year 15 post-drug product infusion.
    Le visite di follow-up nello studio LTF-304 sono programmate almeno ogni 6 mesi fino all’Anno 5 successivamente all’infusione del prodotto medicinale e in seguito ogni anno fino all’Anno 15 successivamente all’infusione del prodotto medicinale.
    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 Yes
    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 EEA4
    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
    Argentina
    Australia
    Brazil
    Canada
    United States
    France
    Netherlands
    Germany
    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: 50
    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) 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 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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 11
    F.4.2.2In the whole clinical trial 60
    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
    None
    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 Decision2022-05-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-06
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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