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    Summary
    EudraCT Number:2018-002935-88
    Sponsor's Protocol Code Number:VX18-CTX001-131
    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:2021-01-28
    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 number2018-002935-88
    A.3Full title of the trial
    A Long-term Follow-up Study of Subjects With ß-thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic
    Stem Cells (CTX001)
    Studio di follow-up a lungo termine in soggetti con ß-talassemia o anemia falciforme trattati con cellule staminali ematopoietiche autologhe modificate con CRISPR-Cas9 (CTX001)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Long-term Follow-up Study of Subjects With ß-thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic
    Stem Cells (CTX001)
    Studio di follow-up a lungo termine in soggetti con ß-talassemia o anemia falciforme trattati con cellule staminali ematopoietiche autologhe modificate con CRISPR-Cas9 (CTX001)
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    A.4.1Sponsor's protocol code numberVX18-CTX001-131
    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 SponsorVERTEX PHARMACEUTICALS INCORPORATED
    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 supportVertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Trials and Medical Info
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address50 Northern Avenue
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02210-1862
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016173416617
    B.5.5Fax number0000000
    B.5.6E-mailmedicalinfo@vrtx.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCTX001
    D.3.2Product code [CTX001]
    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.3.8INN - Proposed INNcellule ematopoietiche staminali e progenitrici umane (hHSPCs) autologhe, CD34+, modificate con CRISPR-Cas9
    D.3.9.2Current sponsor codeCTX001
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number3
    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 Yes
    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 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
    Transfusion-dependent ß thalassemia (TDT).
    Severe sickle cell disease (SCD).
    Beta-talassemia trasfusione dipendente (UDT)
    Anemia Falciforme grave (SCD)
    E.1.1.1Medical condition in easily understood language
    Transfusion-dependent ß thalassemia.
    Severe sickle cell disease.
    Beta-talassemia trasfusione dipendente (TDT)
    Anemia Falciforme grave (SCD)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10043391
    E.1.2Term Thalassaemia beta
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10040641
    E.1.2Term Sickle cell anaemia
    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
    Evaluate long-term safety up to 15 years after CTX001 infusion in subjects who received CTX001 for treatment of transfusion-dependent thalassemia (TDT) or severe sickle cell disease (SCD)
    Valutare la sicurezza a lungo termine fino a 15 anni dopo l’infusione di CTX001 in soggetti che hanno ricevuto CTX001 per il trattamento della talassemia trasfusione-dipendente (UDT) o dell’anemia falciforme (SCD) grave
    E.2.2Secondary objectives of the trial
    To evaluate efficacy of CTX001 up to 15 years after CTX001 infusion, in subjects who received CTX001 for treatment of TDT or SCD
    Valutare l'efficacia di CTX001 fino a 15 anni dopo l'infusione di CTX001, in soggetti che hanno ricevuto CTX001 per il trattamento di TDT o SCD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects or legal representative or guardian (if applicable) must sign and date informed consent form (ICF).
    2. Subjects must have received CTX001 infusion.
    1. I soggetti o il legale rappresentante o tutore (se applicabile) devono firmare e datare il modulo di consenso informato (ICF)
    2. I soggetti devono aver ricevuto l'infusione di CTX001
    E.4Principal exclusion criteria
    1. There are no exclusion criteria.
    1. Non ci sono criteri di esclusione
    E.5 End points
    E.5.1Primary end point(s)
    • New malignancies
    • New or worsening hematologic disorders (e.g. immune-mediated cytopenias, aplastic anemia, primary immunodeficiencies)
    • All-cause mortality
    • All serious adverse events (SAEs) occurring up to 5 years after CTX001
    infusion
    • CTX001-related AEs and SAEs
    • Nuove neoplasie
    • Disturbi ematologici nuovi o in peggioramento (ad es. citopenie immunomediate, anemia aplastica, immunodeficienze primarie)
    • Mortalità per tutte le cause
    • Tutti gli eventi avversi seri (SAE) che si verificano fino a 5 anni dopo l'infusione di CTX001
    • Eventi avversi correlati a CTX001 e SAE
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 15 years after CTX001 infusion (with exception of SAEs as noted above).
    Fino a 15 anni dopo l'infusione di CTX001 (ad eccezione dei SAE, come segnalato sopra)
    E.5.2Secondary end point(s)
    • Total Hemoglobin (Hb) concentration (pre-transfusion) over time
    • Fetal hemoglobin (HbF) concentration (pre-transfusion) over time
    • Proportion of alleles with intended genetic modification present in peripheral blood leukocytes over time
    TDT:
    • TDT-related transfusion
    • Iron overload as measured by liver iron concentration (LIC), cardiac
    • iron concentration (CIC), and ferritin
    • Proportion of subjects receiving iron chelation therapy over time
    SCD:
    • Severe vaso-occlusive crisis (VOC)
    • SCD-related transfusions; • Hemoglobin (Hb) concentrations over time
    • HbF concentrations over time
    TDT:
    • TDT-related transfusion
    • Iron overload as measured by liver iron concentration (LIC), cardiac
    iron concentration (CIC), and ferritin
    SCD:
    • Severe vaso-occlusive crisis (VOC)
    • Concentrazioni totale di emoglobina (Hb) (pre-trasfusione ) nel tempo
    • Concentrazioni di HbF (pre-trasfusione ) nel tempo
    • Proporzione di alleli con modificazione genetica prevista presente in leucociti del sangue periferico nel tempo

    TDT:
    • Trasfusione dovuta a TDT
    • Sovraccarico di ferro misurato dalla concentrazione di ferro nel fegato (LIC), concentrazione di ferro cardiaco (CIC) e ferritina
    • Proporzione di soggetti sottoposti a terapia di chelazione del ferro nel tempo
    SCD:
    • Crisi vaso-occlusiva grave (VOC)
    • SCD correlata a trasfusioni; • Concentrazioni di emoglobina (Hb) nel tempo
    • Concentrazioni di HbF nel tempo
    TDT:
    • Trasfusione dovuta a TDT
    • Sovraccarico di ferro misurato dalla concentrazione di ferro nel fegato (LIC), concentrazione di ferro cardiaco (CIC) e ferritina
    SCD:
    • Crisi vaso-occlusiva grave (VOC)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 15 years after CTX001 infusion (with exception of LIC and CIC which will be assessed for up to 5 years).; Up to 5 years after CTX001 infusion.
    Fino a 15 anni dopo l'infusione di CTX001 (ad eccezione di LIC e CIC Che saranno rilevati fino a 5 anni).; Fino a 5 dopo l'infusione di CTX001
    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) 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) 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 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
    In aperto, follow up a lungo termine
    Open, 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 EEA6
    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
    Canada
    United States
    Belgium
    France
    Germany
    Greece
    Italy
    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 years20
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years21
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 90
    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 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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 37
    F.4.2.2In the whole clinical trial 90
    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)
    Not applicable
    Non applicabile
    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 Decision2019-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-24
    P. End of Trial
    P.End of Trial StatusOngoing
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