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    Summary
    EudraCT Number:2024-000261-24
    Sponsor's Protocol Code Number:ECT-001-CB.007
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2024-06-24
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2024-000261-24
    A.3Full title of the trial
    A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in pediatric and young adult (<21year) Patients with High-Risk and Very High-Risk Myeloid Malignancies (ECT-001-CB.007)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in pediatric and young adult (<21year) Patients with High-Risk and Very High-Risk Myeloid Malignancies (ECT-001-CB.007)
    A.4.1Sponsor's protocol code numberECT-001-CB.007
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/544/2023
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCordex Biolohics Inc
    B.1.3.4CountryCanada
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCordex Biologics Inc
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCordex Biologics Inc
    B.5.2Functional name of contact pointPierre Caudrelier
    B.5.3 Address:
    B.5.3.1Street Address2950 chemin de Polytechnique
    B.5.3.2Town/ cityMontreal
    B.5.3.3Post codeH3T 1J4
    B.5.3.4CountryCanada
    B.5.4Telephone number15144181827
    B.5.6E-mailpierre.caudrelier@excellthera.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/3/20/2271
    D.3 Description of the IMP
    D.3.2Product code ECT-001-CB
    D.3.4Pharmaceutical form Dispersion for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 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) Yes
    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 and Very High-Risk Paediatric Myeloid Malignancies
    E.1.1.1Medical condition in easily understood language
    Acute Myeloid Leukaemia
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1) Examine the safety and feasibility of infusing a single ECT-001-expanded cord blood in pediatric and young adult patients with high and very high-risk myeloid malignancies
    2) Evaluate incidence of relapse at 1-year post-transplant
    E.2.2Secondary objectives of the trial
    1) Evaluate leukemia free survival at 1- and 2-years after transplantation
    2) Kinetics of hematologic engraftment
    3) Non-Relapse Mortality at Day 100 and at 1-year
    4) Incidence of acute and chronic GVHD by NIH criteria at 1- and 2-years post-transplant
    5) Incidence of grade 3 or higher infectious complications
    6) Hospitalization events: Duration of transplant admission and number of days in hospital in 1st 100 days, and last day of fever (>38°C) prior to engraftment.
    7) Incidence of pre-engraftment/engraftment syndrome (PES/ES) requiring therapy
    8) Immune reconstitution: CD4 counts, CD8 counts, IgG levels
    9) Quality of Life evaluation
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Acute Myeloid Leukemia
    a) Chemo-refractory relapse (MRD+)
    b) Primary induction failure (no CR or CRi after >= 2 courses of intensive induction therapy): < 30% blasts in evaluable marrow.
    c) Relapse after previous allogeneic (or autologous) transplant (Note: ECB-001-CB transplant to take place > 4 months from previous transplant)
    d) Secondary or therapy-related MDS/AML
    e) Poor response to induction (5-30% blasts) or MRD+ after induction
    2) Myelodysplastic syndrome (MDS)
    a) Relapse after allogeneic or autologous transplant (Note: ECB-001-CB transplant to take place >4 months from previous transplant)
    b) ≥10 % blasts within 30 days of start of conditioning regimen
    c) Poor and very poor cytogenetics abnormalities and very high-risk molecular abnormalities (e.g., TP53)
    3) Chronic myelogenous leukemia: Patients with history of blast crisis or accelerated phase
    4) Mixed Phenotype Acute Leukemia: MRD+ or relapse after previous transplant (>4 months).
    5) JMML (Juvenile Myelo-Monocytic Leukemia)
    6) 0 – 21 years
    7) Availability of 2 ≥ 4/8 HLA matched CBU (allele level: A, B, C and DRB1)
    a) Cord to be expanded: CD34+ cell count ≥ 0.5 x 10E5/kg and TNC ≥ 1.5 x 10E7/kg (pre-cryo)
    b) Back up cord: Pre-freeze TNC ≥ 2 x 10E7/kg with CD34+ cells ≥ 1 x 10E5/kg. If a single cord does not meet this criterion 2 back up cords will be an acceptable alternative with a minimum for each of 1.5 x 10E7 TNC/kg and 1 x 10E5/kg CD34+ cells. Another acceptable HSC back up source could be a haploidentical with medical clearance prior to starting conditioning regimen.
    7) Lansky / Karnofsky >60%
    8) Bilirubin < 2 x upper limit of normal (ULN) unless felt to be related to Gilbert’s disease or hemolysis; AST and ALT < 3 x ULN; alkaline phosphatase < 5 x ULN
    9) Estimated or measured creatinine clearance ≥ 50 ml/min/1.73m2
    10) Left ventricular ejection fraction of ≥ 40%
    11) FVC, FEV1 and DLCO (corrected for hemoglobin) >= 50% of predicted. In young children unable to perform pulmonary function testing: pulse oximetry >92% in room air and a normal CT of the chest
    12) Signed written informed consent (parents or legal guardians for minor patients).
    13) Female patients of childbearing potential must have a negative serum pregnancy test within 7 days of enrolment and mush be willing to use an effective contraceptive method while enrolled in the study.
    E.4Principal exclusion criteria
    1) Previous allogeneic transplantation within 4 months.
    2) Uncontrolled infection.
    3) Presence of other malignancy other than the one for which the CB transplant is being performed, with an expected survival to be less than 75% at 5 years
    4) Seropositive for HIV.
    5) Hep B and C infection with measurable viral load.
    6) Liver cirrhosis.
    7) Active CNS disease.
    8) Chloroma > 2cm.
    9) >30% blasts in marrow in evaluable marrow sample.
    10) Pregnancy, breastfeeding or unwillingness to use appropriate contraception
    11) Participation in a trial with an investigational agent within 20-30 days prior to the date of transplant (infusion of the expanded CD34+ cells): for investigation agents with half-life < 1 day: patient must be off the Investigational agent for 20 days; for investigation agents with half-life > 1 day: patient must be off the Investigational agent for 30 days.
    12) No signed informed consent.
    13) Any abnormal condition or lab result that is considered by the PI capable or altering patient’s condition or study outcome.
    E.5 End points
    E.5.1Primary end point(s)
    1) Safety and feasibility of infusing a single ECT-001-expanded blood cord (AEs, ADRs, SAEs)
    2) Relapse at 1-year post-transplant
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) Throughout trial
    2) 1-year post-transplant
    E.5.2Secondary end point(s)
    1) LFS, OS
    2) Kinetics of hematopoietic engraftment (neutrophil engraftment, platelet engraftment, graft failuire)
    3) NRM
    4) Acute and chronic GVHD
    5) Severe infectious complications
    6) Hospitalization events
    7) ES/PES requiring therapy
    8) Immune reconstitution
    9) Quallity of life
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) 1- and 2-year post-transplant
    2) Day 45 for neuts, Day 100 for platelets
    3) Day 100 and 1-year post-transplant
    4) Day 100 and 1-year post-transplant (aGVHD), 1- and 2-year post-transplant (cGVHD)
    5) Througout trial
    6) Throughout trial
    7) Throughout trial
    8) Days 30, 60 and Months 3, 6, 12 post-transplant
    9) Months 3, 6, 12, 24 post-transplant
    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 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 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    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: 15
    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: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) No
    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 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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 15
    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
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Memorial Sloan Kettering Cancer Center
    G.4.3.4Network Country United States
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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