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    Summary
    EudraCT Number:2016-003226-16
    Sponsor's Protocol Code Number:BP-404
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-11-17
    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 number2016-003226-16
    A.3Full title of the trial
    Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Follow-up di studio di fase I/II sull’impiego di cellule T CaspaCide derivate da donatore familiare parzialmente compatibile sottoposto a procedura di T deplezione αβ, in pazienti pediatrici affetti da disordini ematologici dopo trapianto aploidentico.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Follow-up di studio di fase I/II volto alla valutazione della sicurezza e della efficacia della somministrazione di cellule T BPX-501 dopo trapianto aploidentico da donatore familiare parzialmente compatibile sottoposto a procedura di T deplezione αβ in pazienti pediatrici.
    A.3.2Name or abbreviated title of the trial where available
    CaspaCide TCR αβ haplo HSCT
    A.4.1Sponsor's protocol code numberBP-404
    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 SponsorBellicum Pharmaceuticals, 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 supportBellicum Pharmaceuticals
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBellicum Pharmaceuticals, Inc
    B.5.2Functional name of contact pointMartha French
    B.5.3 Address:
    B.5.3.1Street Address2130 W. Holcombe Blvd. Suite 800
    B.5.3.2Town/ cityHouston
    B.5.3.3Post codeTX 77030
    B.5.3.4CountryUnited States
    B.5.4Telephone number018323841119
    B.5.5Fax number012817687698
    B.5.6E-mailmfrench@bellicum.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/3/16/1666
    D.3 Description of the IMP
    D.3.1Product nameAP1903
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 195514-63-7
    D.3.9.2Current sponsor codeAP1903
    D.3.9.3Other descriptive nameAP1903
    D.3.9.4EV Substance CodeSUB171411
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.4
    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 No
    D.3.11.3.2Gene therapy medical product No
    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.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
    Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic
    syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies)
    Patologie neoplastiche ematologiche (LLA, LMA, Linfoma non Hodgikin, sindrome mielodisplastica, deficienza immunitaria congenita, grave anemia aplastica, anemia di Fanconi, osteopetrosi, casi selezionati di emoglobinopatia)
    E.1.1.1Medical condition in easily understood language
    Hematological disorders
    Disordini ematologici
    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 19.0
    E.1.2Level HLGT
    E.1.2Classification code 10018849
    E.1.2Term Haematological disorders NEC
    E.1.2System Organ Class 10005329 - Blood and lymphatic system 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 long-term safety of the infusion of BPX-501 gene
    modified T cells and to evaluate disease-free survival at 1 and 2 years
    Valutare la sicurezza a lungo termine dell’infusione delle cellule T BPX-501 geneticamente modificate e la sopravvivenza libera da malattia a 1 e 2 anni.
    E.2.2Secondary objectives of the trial
    Long-term safety for a total of 15 years from BPX-501
    administration
    Sicurezza a lungo termine per un totale di 15 anni dalla somministrazione di BPX-501.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed written informed consent by the patient or the patient’s guardian for children who are minors
    2. enrolled on BP-004 protocol, received BPX-501 infusion and completed 6 months of active treatment
    1. Acquisizione per iscritto del consenso informato da parte del paziente o del tutore legale del paziente in caso di minori
    2. Paziente che è stato arruolato per il trattamento secondo il protocollo BP-004, ha ricevuto l’infusione di BPX-501 e ha completato 6 mesi di trattamento attivo
    E.4Principal exclusion criteria
    1. Lack of parents’/guardian’s informed consent for children who are minors.
    1. Mancanza del consenso informato dei genitori/tutori legali dei pazienti minorenni.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of disease-free survival in both malignant and non-malignant
    patients at 1 and 2 years.
    Incidenza della sopravvivenza libera da malattia sia in pazienti affetti da patologie maligne sia in pazienti affetti da patologie non maligne a 1 e 2 anni.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 2 years post-transplant
    Fino a 2 anni dopo il trapianto
    E.5.2Secondary end point(s)
    Long-term safety for a total of 15 years from BPX-501
    administration
    Sicurezza a lungo termine per un totale di 15 anni dalla somministrazione di BPX-501.
    E.5.2.1Timepoint(s) of evaluation of this end point
    15 years
    15 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 No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase I/II
    Fase I/II
    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 No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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 No
    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
    The study will evaluate disease free survival for all patients enrolled at
    1 year, 2 years. Patients will be followed for a total of 15 years for gene
    therapy safety events. The study will be closed after the completion of
    the 15 years of follow-up.
    Lo studio valuterà la sopravvivenza libera da malattia per tutti pazienti arruolati ad 1 anno e 2 anni. I pazienti saranno seguiti per un totale di 15 anni per eventi di sicurezza legati alla terapia genica. Lo studio sarà chiuso dopo il completamento dei 15 anni di follow-up.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years15
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 170
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 60
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 80
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children
    Bambini
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state155
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 175
    F.4.2.2In the whole clinical trial 175
    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
    Non previsto
    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 Decision2017-02-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-18
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-04-24
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