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    Summary
    EudraCT Number:2014-000074-19
    Sponsor's Protocol Code Number:GCP#03.01.020
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-12-03
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2014-000074-19
    A.3Full title of the trial
    Allogeneic Stem Cell Transplantation of NiCord, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells, in Adolescents and Adult Patients with Hematological Malignancies
    Allogene stamceltransplantatie van NiCord®, uit navelstrengbloed verkregen ex vivo geëxpandeerde stam- en
    progenitorcellen bij adolescente en volwassen patiënten met hematologische maligniteiten
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Transplantation of NiCord, stem cells from donated umbilical cord blood that were grown in a laboratory to expand their number, in adolescent and adult patients with cancers of the blood and lymph
    Transplantie van NiCord, stam cellen vanuit gedoneerd navelstrengbloed die zijn gekweekt in een laboratorium om in aantal te groeien, in volwassen en adolescente patienten met bloed en lymfe kanker.
    A.4.1Sponsor's protocol code numberGCP#03.01.020
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01816230
    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 SponsorGamida Cell Ltd
    B.1.3.4CountryIsrael
    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 supportGamida Cell
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSMS-oncology
    B.5.2Functional name of contact pointEllis Schipper
    B.5.3 Address:
    B.5.3.1Street AddressScience Park 408
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1098 XH
    B.5.3.4CountryNetherlands
    B.5.4Telephone number31204350580
    B.5.6E-maile.schipper@sms-oncology.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 nameNiCord (CF cultured fraction and NF non-cultured fraction)
    D.3.4Pharmaceutical form Suspension for injection
    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 INNallogeneic, umbilical cord-blood derived, ex vivo expanded, hematopoietic CD133+ cells
    D.3.9.3Other descriptive nameALLOGENEIC, UMBILICAL CORD BLOOD-DERIVED, EX VIVO EXPANDED, HEMATOPOIETIC CD133+ CELLS
    D.3.9.4EV Substance CodeSUB121492
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number1200000000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNallogeneic, umbilical cord blood-derived, non-expanded, hematopoietic CD133- cells
    D.3.9.3Other descriptive nameALLOGENEIC, UMBILICAL CORD BLOOD-DERIVED, NON-EXPANDED, HEMATOPOIETIC CD133- CELLS
    D.3.9.4EV Substance CodeSUB130958
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number500000000
    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 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
    High risk haematological malignancies
    E.1.1.1Medical condition in easily understood language
    cancer affecting blood, bone marrow, lymph nodes
    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 17.1
    E.1.2Level LLT
    E.1.2Classification code 10000845
    E.1.2Term Acute lymphoblastic leukemia
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10025316
    E.1.2Term Lymphoma NOS
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid leukemia
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10009015
    E.1.2Term Chronic myeloid leukemia
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10028534
    E.1.2Term Myelodysplastic syndrome NOS
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the cumulative incidence of patients with NiCord-derived neutrophil engraftment at 42 days following transplantation
    Assess the incidence of secondary graft failure at 180 days following transplantation of NiCord
    E.2.2Secondary objectives of the trial
    Time from infusion to neutrophil engraftment
    Time from infusion to platelet engraftment
    Incidence of platelet engraftment at 100 days
    Proportion of non-relapse mortality at 100 days
    Incidence of acute GvHD grade II-IV and III-IV at 100 days
    Incidence of chronic GvHD (limited or extensive) at 180 days and 1 year
    Incidence of secondary graft failure at 1 year following transplantation of NiCord®
    Overall survival at 180 days and 1 year
    Safety and tolerability of NiCord® transplantation
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patients must be 12-65 years of age
    2.Patients with one of the following hematologic malignancies:
    Acute lymphoblastic leukemia (ALL) at one of the following stages:
    a.High risk first complete morphologic remission (CR1), defined as one or more of the following:
    The presence of t(4;11), t(9;22), t(1;19) or MLL rearrangements t(11q23)
    Extreme leukocytosis (WBC >30,000/µl for B-ALL or >100,000/µl for T-ALL)
    Longer than 4 weeks to achieve complete remission after induction therapy
    b.Second or subsequent remission
    Acute myelogenous leukemia (AML) at one of the following stages:
    a.First complete morphologic remission (CR1) that is NOT considered as favorable-risk:
    Favorable risk is defined as having one of the following:
    t(8,21) without cKIT mutation
    inv(16) without cKIT mutation or t(16;16)
    Normal karyotype with mutated NPM1 and no FLT-3 Internal Tandem Duplication
    Normal karyotype with double mutated CEBPA
    APL in first or second molecular remission at end of consolidation
    b.Second or subsequent remission
    Chronic myelogenous leukemia (CML) at one of the following phases:
    a.Chronic phase with one or more of the following characteristics:
    Failure to achieve a primary hematologic or cytogenetic response to either nilotinib or dasatinib (following European LeukemiaNet timelines)
    Intolerance to/failure of two tyrosine kinase inhibitors (TKI)
    Any T3151 mutation
    b.Accelerated phase with one or more of the following characteristics:
    Newly diagnosed patients who do not achieve an optimal response to TKIs
    TKI-treated patients who progress from chronic phase
    c.Blast crisis (myeloid or lymphoid) with disease control
    Myelodysplastic Syndrome (MDS) with International Prognostic Scoring System (IPSS) risk category of INT-1 or greater and <5% myeloblasts in the bone marrow on morphologic analysis. MDS patients categorized as INT-1 on primary presentation must have life threatening neutropenia or thrombocytopenia.
    Lymphoma fulfilling one of the following criteria:
    a.Chemotherapy-sensitive (complete or partial response) lymphomas that have failed at least 1 prior regimen of multi-agent chemotherapy and are INELIGIBLE for an autologous transplant.
    b.Marginal zone B-cell lymphoma or follicular lymphoma that has progressed after at least two prior therapies (excluding single agent Rituxan).
    3.Patients must have a partially HLA-matched CBU: the unit must be HLA-matched at 4-6/6 HLA class I (HLA-A & HLA-B, low resolution) and II (HLA-DRB1, high resolution) loci with the patient. The CBU must have a pre-cryopreserved, total CD34+ cell dose of ≥10 x10^6 as well as a pre-cryopreserved total nucleated cell dose of ≥1.8x10^9 and ≥1.8x10^7 TNC/kg. The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation.
    4.Patients must have an additional partially HLA-matched CBU, or two CBUs, reserved as a backup in case of batch failure. The backup CBU/s must be HLA-matched at 4-6/6 HLA class I (HLA-A & HLA-B, low resolution) and II (HLA-DRB1, high resolution) loci with the patient, and must have a pre-cryopreserved, total nucleated cell dose of at least 2x10^7 per kilogram, in case of one CBU, or 3x10^7 per kilogram, in case of two CBUs.
    5.Patients must have a related, haplo-identical family member who is suitable for bone marrow or peripheral blood stem cell donation and has agreed to do so in the event of graft failure.
    6.Subjects’ Performance score ≥70% by Karnofsky/Lansky
    7.Patient has sufficient physiologic reserves including:
    a)Cardiac: Left ventricular ejection fraction (LVEF) of >40% by echocardiogram, radionuclide scan or cardiac MRI
    b)Pulmonary function tests demonstrating FVC and FEV1 of >50% of predicted for age and DLCO > 50% of predicted
    c)Renal: Creatinine clearance test (by Cockcroft-Gault equation) ≥60 mL/min
    d)Hepatic: Serum Bilirubin < 2.0 mg/dl; Hepatic transaminases (ALT and AST) < 3 x upper limit of normal range
    8.Females of childbearing potential, defined as any female who has experienced menarche and is not postmenopausal or permanently sterilized (e.g. tubal occlusion, hysterectomy, bilateral salpingectomy), agree to use an appropriate method of contraception from at least 7 days prior to myeloablative therapy until completion of follow-up procedures. An appropriate method of contraception is defined as one that results in a low failure rate (i.e., less than 1 percent per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine contraceptive devices (IUDs), sexual abstinence, or a vasectomized partner.
    9.Patient (or legal guardian) signs the written informed consent after being aware of the nature of the patient’s disease and willingly consents to the treatment program after being informed of alternative treatments, potential risks, benefits, and discomforts.
    E.4Principal exclusion criteria
    1. NK cell lymphoma, Burkitt's lymphoma
    2. CLL/SLL diagnosis
    3. MDS or CML with "marked" or "3+" fibrosis
    4. Less than 21 days have elapsed since initiation of the patient's last chemotherapy regimen and the initiation of the stem cell transplant preparative regimen (intrathecal agents, hydroxyurea, tyrosine kinase inhibitors, hypomethylating agents and rituximab, not considered chemotherapy)
    5. Persistent clinically significant toxicities from prior chemotherapy that, in the investigator's opinion, make the patient unsuitable for transplant
    6. Evidence of anti-HLA antibodies to the selected NiCord® CBU (MFI>3000)
    7. Evidence of HIV infection or HIV positive serology
    8. Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
    9. Pregnancy (βHCG +) or lactation
    10. Active malignancy other than that for which the UCB transplant is being performed within 12 months of enrollment. Fully resected cutaneous squamous cell or basal cell carcinoma or cervical carcinoma in situ within 12 months of enrollment will be permitted.
    11. Evidence of uncontrolled bacterial, fungal or viral infections or severe concomitant diseases, which in the judgment of the Principal Investigator indicate that the patient could not tolerate transplantation
    12. Patients with signs and symptoms of active central nervous system (CNS) disease, including CNS leukemia or lymphoma
    13. Patients with an 8/8 allele level HLA-matched and readily available related or unrelated donor, e.g. patients who have haploidentical related donors will not be excluded
    14. Prior allogeneic hematopoietic stem cell transplant
    15. Allergy to bovine, gentamicin, or to any other product which may interfere with the treatment
    16. Psychiatric illness and/or social situations that would limit compliance with study requirements
    17. Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by Sponsor
    E.5 End points
    E.5.1Primary end point(s)
    1. cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation
    2. incidence of secondary graft failure at 180 days following transplantation of NiCord®
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. day 42 and 2. day 180
    E.5.2Secondary end point(s)
    1. Time to Neutrophil engraftment
    2. Time to Platelet engraftment
    3. Incidence of platelet engraftment at 100 days
    4. Non-relapse mortality at 100 days
    5. Acute GvHD grade II-IV and III-IV at 100 days
    6. Chronic GvHD (limited or extensive) at 180 days and 1 year
    7. Incidence of secondary graft failure at 1 year following transplantation of NiCord®
    8. Overall survival at 180 days and 1 year
    9. Safety and tolerability if NiCord® transplantation
    Exploratory endpoints:
    1. Immune Reconstitution
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Not relevant; 2. Not relevant; 3. 100 days; 4. 100 days; 5. 100 days; 6. 180 days and 1 year; 7. 1 year; 8. 180 days and 1 year; 9. 1 year; 1. 70, 100, 180 and 365 days
    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
    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 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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Italy
    Netherlands
    Spain
    United States
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 4
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 16
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 20
    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)
    When subject completes the study visits he will be offered to participate in long term follow up study.
    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 Decision2014-11-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-06-29
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