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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43694   clinical trials with a EudraCT protocol, of which   7248   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2022-002458-26
    Sponsor's Protocol Code Number:ECT-001-CB.004
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-11-11
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2022-002458-26
    A.3Full title of the trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of expanded Cord Blood transplantation in patients with high-risk blood cancers
    A.4.1Sponsor's protocol code numberECT-001-CB.004
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04103879
    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 SponsorExCellThera inc.
    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 supportExCellThera inc.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationErasmus mC
    B.5.2Functional name of contact pointJurjen Versluis
    B.5.3 Address:
    B.5.3.1Street AddressDr. Molewaterplein 40
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3015 GD
    B.5.4Telephone number00310107040704
    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 nameECT-001-CB
    D.3.2Product code ECT-001-CB
    D.3.4Pharmaceutical form Solution for infusion in administration system
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D. cell therapy medicinal product Yes
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product Yes
    D. classification and reference numbersomatic cell therapy medicinal product (product ref.: H0005772)
    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 acute leukemia/myelodysplasia requiring an allogeneic haematopoietic stem cell transplantation
    E.1.1.1Medical condition in easily understood language
    blood cancer
    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 22.0
    E.1.2Level PT
    E.1.2Classification code 10067859
    E.1.2Term Allogenic stem cell transplantation
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1) To examine the safety (including assessment of the rate of graft failure) and feasibility of infusing a single ECT-001-expanded cord blood in patients with high and very high-risk acute leukemia/myelodysplasia
    2) Evaluate relapse-free survival at 1- and 2-years post-transplant in a group of patients with high-risk acute leukemia/myelodysplasia.
    E.2.2Secondary objectives of the trial
    1) Determine the kinetics of hematologic engraftment (including neutrophil and platelet engraftment) following infusion of a single ECT-001-expanded cord blood.
    2) Estimate the incidence of transplant related mortality at day 100 and 1-year post-transplant.
    3) Determine incidence of acute and chronic GVHD by NIH criteria at 2 years post-transplant.
    4) Determine incidence of grade 3 or higher infectious complications.
    5) Determine incidence of pre-engraftment/engraftment syndrome requiring therapy.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) 18-70 years old
    2) Presence of a high and very high-risk hematologic malignancy
    3) Availability of 2 CBs ≥ 4/6 HLA match when DRB1 is performed at the allele level and A, B at antigen
    resolution (intermediate resolution) and ≥ 4/8 HLA match when A, B, C and DRB1 are performed at
    the allele level. An acceptable alternative would be a 3/6 or 5/8 as long as there is no double DRB1
    4) Karnofsky ≥70.
    5) Left ventricular ejection fraction ≥ 40% (within 3 months unless patient has received chemotherapy
    or radiation therapy to the thorax since the last cardiac evaluation) or fractional shortening >22%
    6) Forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1) and diffusing capacity
    corrected for hemoglobin (DLCOc) ≥ 50% of predicted
    7) Bilirubin < 2 x upper limit of normal (ULN) unless felt to be related to Gilbert’s disease or hemolysis;
    AST and ALT ≤ 2.5 x ULN; alkaline phosphatase ≤ 5 x ULN.
    8) Adequate renal function defined as creatinine < 2.0 mg/dl (adults). All adults with a creatinine > 1.2
    or a history of renal dysfunction must have estimated creatinine clearance > 50 ml/min.
    9) Hematopoietic cell transplantation specific comorbidity index (HCT-CI) ≤3 if patients have ≥5%
    blasts in the bone marrow and HCT-CI ≤5 if 60-70 years old.
    E.4Principal exclusion criteria
    1) Allogeneic myeloablative transplant within 6 months.
    2) Autologous hematopoietic stem cell transplant within 6 months.
    3) Active or recent (prior 6 month) invasive fungal infection without ID consult and approval.
    4) Presence of a malignancy other than the one for which the UCB transplant is being performed
    and the expected survival related to the malignancy is estimated to be less than 75% at 5 years.
    5) HIV positivity.
    6) Hepatitis B or C infection with measurable viral load.
    7) Liver cirrhosis.
    8) Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
    9) Participation in a trial with an investigational agent within 30 days prior to entry in the study.
    10) Patient unable to give informed consent or unable to comply with the treatment protocol including
    appropriate supportive care, follow-up, and tests.
    11) Any abnormal condition or laboratory result that is considered by the principal investigator capable
    of altering patient condition or study outcome.
    12) Active central nervous system involvement.
    13) Chloroma > 2 cm.
    E.5 End points
    E.5.1Primary end point(s)
    Evaluate relapse-free survival at 1- and 2-years post-transplant.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1, and 2 years post-transplant.

    E.5.2Secondary end point(s)
    Kinetics of hematopoietic engraftment (Neutrophil engraftment and Platelet engraftment)
    Transplant related mortality (TRM)
    Incidence of acute and chronic GVHD
    Incidence of grade 3 or higher infectious complications
    Incidence of pre-engraftment and engraftment syndrome requiring therapy
    E.5.2.1Timepoint(s) of evaluation of this end point
    Neutrophil engraftment: absolute neutrophil count (ANC) ≥ 0.5 x 109/L for 3 consecutive days after the 1st post-CBT nadir. Time to ANC ≥ 0.1 x109/L
    Platelet engraftment: sustained platelet count ≥ 20 x 109/L with no platelet transfusion in the preceding 7 days as per CIBMTR standards
    Time to achieving a platelet count ≥ 50 x 109/L
    TRM will be monitored throughout the study and in particular at 100 days and 1 and 2 years post transplantation
    The incidence of acute and chronic GvHD will be evaluated throughout the study, but particularly evaluated at 1-year post-transplant for aGVHD, 1 and 2 years post-transplant for cGVHD, using NIH criteria.
    The incidence of grade 3 or higher infectious complications, pre-engraftment and engraftment syndrom will be evaluated throughout the study

    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. 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 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 No
    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
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days1
    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: 25
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 5
    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 Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    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 5
    F.4.2.2In the whole clinical trial 30
    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)
    standard of care
    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 Decision2022-11-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-11-09
    P. End of Trial
    P.End of Trial StatusOngoing
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