A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, Umbilical Cord Blood-derived, Stem and Progenitor Cells, versus Unmanipulated Umbilical Cord Blood for Patients with Hematological Malignancies

Gamida Cell Ltd0 个研究点目标入组 120 人开始时间: 2016年5月17日最近更新: 2016年6月20日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Gamida Cell Ltd
入组人数: 120

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

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研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

性别: All

入选标准

•1.Patients must be 16-60 years of age at the time of randomization
•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
•-Evidence of minimal residual disease (MRD) by flow cytometry
•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 or more of the following:
•-t(8,21) without either cKIT mutation or positive MRD
•-inv(16) or t(16;16) without either cKIT mutation or positive MRD
•-Normal karyotype with mutated NPM1 and no FLT-3 Internal Tandem Duplication without MRD
•-Normal karyotype with double mutated CEBPA without positive MRD
•-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 T315I mutation
•b.Accelerated phase with one or more of the following characteristics:
•-Newly diagnosed patients who do not achieve an optimal response to TKIs as outlined in the European LeukemiaNet timelines
•-TKI-treated patients who progress from chronic phase
•c.Prior blast crisis (myeloid and lymphoid) currently in complete morphologic remission
•?Myelodysplastic Syndrome (MDS) with history of International Prognostic Scoring System (IPSS) risk category of INT-1 or greater. On screening morphologic analysis patients must have no circulating myeloblasts and ?10% myeloblasts in the bone marrow. MDS patients categorized as INT-1 on primary presentation must have life threatening neutropenia (ANC < 0.5x10^9/L) or thrombocytopenia (platelets < 30x10^9/L).
•3.CBU criteria as described in the protocol; confirmatory typing must be completed for CBU#1 chosen for transplant.
•4.Patients must have an additional partially HLA-matched CBU, or two CBUs, reserved as a backup. 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.
•In case of one CBU, if the CBU is HLA-matched at 5-6/6, it must contain a pre-cryopreserved (post processing) total nucleated cell dose of ?2.5x10^7 TNC/kg, AND a pre-cryopreserved (post processing) CD34+ cell dose of ?1.2 x10^5 CD34+ cells/kg. If the CBU is HLA-matched at 4/6, it must contain a pre-cryopreserved (post processing) total nucleated cell dose of ?3.5x10^7 TNC/kg, AND a pre-cryopreserved (post processing) CD34+ cell dose of ?1.7 x10^5 CD34+ cells/kg.
•In case of two CBUs, the CBUs must have a combined pre-cryopreserved (post processing) total nucleated cell dose of at least 3x10^7 per kilogram.
•5.Patient?s Performance score ?70% by Karnofsky
•6.Patient has sufficient physiologic reserves including:
•a.Cardiac: Left ventricular ejection fraction (LVEF) of ?40% by echocardiogram, radionuclide

排除标准

•1.MDS or CML with ?marked? or ?3+? fibrosis
•2.CMMoL or MDS/CMMoL overlap
•3.Fewer than 21 days have elapsed since initiation of the patient's last chemotherapy cycle and the initiation of the stem cell transplant preparative regimen (intrathecal agents, hydroxyurea, tyrosine kinase inhibitors, hypomethylating agents, rituximab and lenalidomide not considered chemotherapy)
•4.Persistent clinically significant toxicities that, in the investigator?s opinion, make the patient unsuitable for transplant
•5.Evidence of anti-HLA antibodies to the selected CBU #1 (MFI>3000)
•6.Evidence of HIV infection or HIV positive serology
•7.Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
•8.Pregnancy, as indicated by a positive serum human chorionic gonadotrophin (HCG) test, or lactation
•9.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.
•10.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
•11.Patients with signs and symptoms of leukemic blasts in the central nervous system (CNS)
•12.Patients with an 8/8 allele level HLA-matched and readily available related or unrelated donor (whose stem cells can be collected in a timely manner without jeopardizing recipient outcome). Patients who have haploidentical related donors will not be excluded
•13.Prior allogeneic hematopoietic stem cell transplant
•14.Allergy to bovine, gentamicin, or to any other product that may interfere with the treatment
•15.Psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance and/or psychiatric illness and/or social situations that would limit compliance with study requirements
•16.Enrolled in another interventional clinical trial or received an investigational treatment 30 days prior to CBU shipment to the production site, unless documented approval obtained from Sponsor prior to randomization

研究者

发起方

Gamida Cell Ltd

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