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In this trial, we aim to improve the outcomes of haplo cord transplant. Haplo cord transplant is a novel and promising way to improve transplant outcomes. We hypothesize that identification of a graft that is at least 5/6 matched and inherited paternal antigen (IPA) targeted (i.e., cord blood grafts share one or more IPA antigens with the prospective recipient) is more important to the outcome of haplo cord transplant than the nucleated cell dose. The identification of such a graft for a large proportion of the subjects may necessitate accepting a lower umbilical cord graft dose.
In addition to a umbilical cord blood transplant, recipients will receive stem cells from a family member ( a haplo-identical donor) . After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. The subject will undergo a chemotherapy conditioning regimen prior to transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past.
This is a clinical trial for subjects with hematologic malignancies ( acute leukemia, myeloproliferative disorders, lymphoma, myeloma) who are in need of a donor stem cell transplant, and for whom an umbilical cord blood transplant is thought to be the best option. As donors for allogeneic transplant, we typically try to use related family members, such as brothers or sisters, or volunteer donors who are 'HLA matched', i.e. share similar proteins on their cells. This study is for subjects for whom such a matched sibling donor or a matched unrelated donor is not available.
For such subjects a commonly used transplant procedure is to use stem cells from one or two umbilical cords (UCB) from a newborn. These umbilical cord blood grafts, despite not completely matching the recipient, cause few problems with graft vs host disease (a common complication of transplant). But they tend to grow very slowly and subjects often have very prolonged hospital stays and are at high risk for complications due to low blood counts. Umbilical cord blood transplant will be the standard arm for this protocol.
This study uses a new method of bone marrow transplantation called combined haplo-identical cord (haplo-cord) transplantation. In this procedure, cells from a related donor who shares half of the HLA proteins ( haplo-identical) are collected from the blood, as well as cells from an umbilical cord, and then both are transplanted. It is hoped that by using cells from a haplo-identical relative, subjects will have a faster recovery and require fewer transfusions. Over time the haplo-identical cells from the relative are replaced by the cells from the cord blood. The combined transplantation of haplo-identical stem cells and cord blood has previously been used in approximately 60 subjects with very encouraging results.
Traditionally it has been felt that the most important determinant of outcome of an UCB stem cell transplant is the cord blood cell dose. The second determinant is the degree of matching between donor and recipient. Many times, we have difficulty identifying UCB units of sufficient cell dose that are well matched. Of interest,in our prior study of haplo-cord SCT indicated outcomes seemed independent of the UCB cell dose. If this preliminary observation is correct, we may be able to improve the outcomes of haplo cord transplant further by accepting lower threshold UCB doses and rather focusing on optimal matching (including matching for HLA and another characteristic called IPA). This is the primary objective of this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg | Experimental | All subjects in this cohort will receive a minimal cell dose of 2 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
|
| Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg | Experimental | All subjects in this cohort will receive a minimal cell dose of 1 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CliniMACS® CD34 Reagent System | Device | The stem cells from the haplo-identical donor will be purified by a procedure called CD34 selection before they are given to the subject. A special device called the CliniMACS® CD34 Reagent System, which is not FDA approved, will be used for this purpose. The manufacturer of the device, Miltenyi Biotec, is providing the researchers access to the device for use in this research study. Because the stem cells from the haplo-identical donor are treated using the CliniMACS CD34 selection device, they cells are considered investigational. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects Who Achieved Engraftment With De-Escalating Umbilical Cord Total Nucleated Cell (TNC) Dose | We aim to identify the lowest threshold of umbilical cord total nucleated cell (TNC) dose that can be utilized assure durable umbilical cord blood engraftment in the haplo-cord transplants. The threshold will be defined as the lowest dose which assures cord blood engraftment occurs in at least 80% of subjects. | 100 days |
| Measure | Description | Time Frame |
|---|---|---|
| Long-term Survival of Subjects Undergoing Haplo-cord Transplants | Evaluate the long term outcome of subjects undergoing haplo cord transplants using an optimally matched umbilical cord blood (UCB) graft (i.e. Survival, profession-free survival (PFS), Relapse, transplant-related mortality (TRM), toxicities, infections and GVHD) | 5 years after transplant |
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Inclusion Criteria:
Subject must have a confirmed diagnosis of:
Age ≥ 18 years
Likely to benefit from allogeneic transplant in the opinion of the transplant physician
An HLA-identical related or unrelated donor cannot be identified within an appropriate time frame.
Karnofsky (KPS) Performance status of >= 70%
Acceptable organ function as defined below: Serum bilirubin: < 2.0mg/dL ALT(SGPT): < 3 X upper limit of normal Creatinine Clearance: > 50 mL/min/1.73m2 (as estimated by the modified MDRD equation)
Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Alexandra Gomez Arteaga, MD | Weill Medical College of Cornell University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Chicago Medical Center | Chicago | Illinois | 60637 | United States | ||
| Weill Cornell Medical College |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31217161 | Derived | van Besien K, Artz A, Champlin RE, Guarneri D, Bishop MR, Chen J, Gergis U, Shore T, Liu H, Rondon G, Mayer SA, Srour SA, Stock W, Ciurea SO. Haploidentical vs haplo-cord transplant in adults under 60 years receiving fludarabine and melphalan conditioning. Blood Adv. 2019 Jun 25;3(12):1858-1867. doi: 10.1182/bloodadvances.2019000200. | |
| 26869630 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 2 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Cohort 1 - Minimum Cell Dose 2 x 10^7 TN |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 8, 2022 |
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| Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg | Experimental | All subjects in this cohort will receive a minimal cell dose of 0.5 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
|
| Cohort 4 | Experimental | All subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3) Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
|
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| Fludarabine | Drug | Administer 30 mg/m2 /day intravenously x 5 days (Day -7 to Day -3) of a total dose of 150 mg/m2. Fludarabine will be dosed according to actual body weight. |
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| Melphalan | Drug | Administer 70mg/m2/day intravenously x 2 days. Melphalan will be dosed according to actual body weight. Cryotherapy with ice chips will be administered to prevent mucositis |
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| anti-thymocyte globulin (rabbit) | Drug | Administer 1.5 mg/kg/day intravenously x 3 days, total 4.5 mg/kg. ATG will be dosed according to actual body weight. The first dose will be infused over at least six hours, and subsequent doses over at least 4 hours. Pre-medications include acetaminophen 650 mg by mouth, diphenhydramine 25-50 mg by mouth or intravenously, and methylprednisolone 2 mg/kg (1 mg/ kg at the initiation and 1 mg/kg half-way through anti-thymocyte globulin administration). |
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| Rituximab | Drug | Administer one dose of 375 mg/m2 prior to or upon admission for all patients not previously exposed to rituximab or who have not received rituximab in the six months prior to transplant. |
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| Total Body Irradiation | Radiation | Patients at high risk of CNS relapse (e.g. ALL or Burkitt's lymphoma) or patients at high risk for graft rejection (i.e., donor-specific HLA antibodies, patients with severe aplastic anemia, or hemoglobinopathies) may receive 2 doses of TBI as part of the conditioning. |
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| Mycophenolate Mofetil | Drug | Will be started on Day -2 and given at a dose of 1000 mg every 8 hours until Day 28. Mycophenolae Mofetil can be given orally or intravenously. Infection, toxicity, very low patient weight (<50 kilograms) may prompt earlier discontinuation or adjustment of doses. |
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| Tacrolimus | Drug | Administered 0.03/mg/kg/day intravenous continious infusion (CI) over 24 hours from 4pm Day -2 until engraftment or when subject is able to take orally, then tacrolimus approximately 0.09 mg/kg orally in 2 divided doses. Tacrolimus should be given at full dose to maintain levels of 5-15 ng/mL through Day 180, tapered by 20% every week thereafter. Infection, toxicity or other clinical circumstances may prompt earlier discontinuation or adjustment of doses. In the presence of Graft versus Host Disease, a clinical decision by the attending physician will determine if tacrolimus can be tapered or should be continued. Oral tacrolimus can be used when intravenous access for CI tacrolimus is unavailable. |
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| Impact of IPA Targeting on Transplant Outcome | As much as possible, UCB units will be chosen to be IPA targeted. This is not always possible. We will therefore also retrospectively analyze transplant outcomes and correlate with IPA status. | 5 years from transplantation |
| Impact of NIMA Matching on Transplant Outcome | As much as possible, UCB units will be chosen to be NIMA matched. This is not always possible. We will therefore also retrospectively analyze transplant outcomes and correlate with NIMA status. | 5 years from transplantation |
| New York |
| New York |
| 10065 |
| United States |
| van Besien K, Hari P, Zhang MJ, Liu HT, Stock W, Godley L, Odenike O, Larson R, Bishop M, Wickrema A, Gergis U, Mayer S, Shore T, Tsai S, Rhodes J, Cushing MM, Korman S, Artz A. Reduced intensity haplo plus single cord transplant compared to double cord transplant: improved engraftment and graft-versus-host disease-free, relapse-free survival. Haematologica. 2016 May;101(5):634-43. doi: 10.3324/haematol.2015.138594. Epub 2016 Feb 11. |
| FG001 | Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 1 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| FG002 | Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 0.5 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| FG003 | Cohort 4 | All subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3) Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| COMPLETED |
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| NOT COMPLETED |
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| Cohort 2 - Minimum Cell Dose 1 x 10^7 TN |
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| Cohort 3 - Minimum Cell Dose 0.5 x 10^7 |
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| Cohort 4 |
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 2 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| BG001 | Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 1 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| BG002 | Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 0.5 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| BG003 | Cohort 4 | All subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3) Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Count of Participants | Participants |
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| Primary Disease | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Subjects Who Achieved Engraftment With De-Escalating Umbilical Cord Total Nucleated Cell (TNC) Dose | We aim to identify the lowest threshold of umbilical cord total nucleated cell (TNC) dose that can be utilized assure durable umbilical cord blood engraftment in the haplo-cord transplants. The threshold will be defined as the lowest dose which assures cord blood engraftment occurs in at least 80% of subjects. | A subset of participants were assessed to analyze the lowest threshold of umbilical cord total nucleated cell (TNC) dose. Once the lowest threshold was established, the remaining participants on study were treated based on that threshold. | Posted | Number | participants | 100 days |
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| Secondary | Long-term Survival of Subjects Undergoing Haplo-cord Transplants | Evaluate the long term outcome of subjects undergoing haplo cord transplants using an optimally matched umbilical cord blood (UCB) graft (i.e. Survival, profession-free survival (PFS), Relapse, transplant-related mortality (TRM), toxicities, infections and GVHD) | Not Posted | 5 years after transplant | Participants | |||||||||||||||||||||||||||||||||
| Secondary | Impact of IPA Targeting on Transplant Outcome | As much as possible, UCB units will be chosen to be IPA targeted. This is not always possible. We will therefore also retrospectively analyze transplant outcomes and correlate with IPA status. | Not Posted | 5 years from transplantation | Participants | |||||||||||||||||||||||||||||||||
| Secondary | Impact of NIMA Matching on Transplant Outcome | As much as possible, UCB units will be chosen to be NIMA matched. This is not always possible. We will therefore also retrospectively analyze transplant outcomes and correlate with NIMA status. | Not Posted | 5 years from transplantation | Participants |
Adverse events were collected for all patients from start of transplant conditioning regimen through 1 year post-transplant or when the participant was removed from the study, whichever was sooner, assessed up to 1 year.
Per protocol, events reported were grade 2 and higher infection and graft-versus-host disease events, and all other hematopoietic or extramedullary events grade 3 or higher per CTCAE v4.0.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 - Minimum Cell Dose 2 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 2 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. | 6 | 15 | 7 | 15 | 6 | 15 |
| EG001 | Cohort 2 - Minimum Cell Dose 1 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 1 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. | 6 | 20 | 9 | 20 | 6 | 20 |
| EG002 | Cohort 3 - Minimum Cell Dose 0.5 x 10^7 TNC/kg | All subjects in this cohort will receive a minimal cell dose of 0.5 x 10^7 total nucleated cells (TNC)/kilogram (kg) for the umbilical cord blood unit. Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. | 11 | 25 | 14 | 25 | 12 | 25 |
| EG003 | Cohort 4 | All subjects in this cohort will receive the minimum required cell dose that is determined following the dose de-escalation portion of the study (cohorts 1 through 3) Haplo-cord transplantation: All subjects will receive a conditioning regimen of chemotherapy prior to stem cell transplantation. No experimental drugs are used in this study, and the combinations of drugs that will be used in the conditioning regimen are combinations that have been used in the past. For the transplant component of treatment, subject will receive umbilical cord blood. The study involves transplantation of unlicensed units of cord blood. Therefore, these are considered investigational products. In addition to the umbilical cord blood unit, recipients will receive stem cells from a family member ( a haplo-identical donor). After collection and prior to infusion, these cells will be purified using a device called a CliniMACS CD34 selection device. | 79 | 210 | 156 | 210 | 93 | 210 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute Kidney Injury | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Acute Renal Failure | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
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| Infectious Colitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Adenovirus Viremia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Adult Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Altered Mental Status | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
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| Anal Fistula | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Asthma Exacerbation | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Atrioventricular block complete | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Atypical hemolytic uremic syndrome | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Bacteremia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Cardiac disorders, other - Junctional rhythym | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Catheter related infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | CTCAE (4.0) | Systematic Assessment |
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| Chronic Inflammatory Demyelinating Polyradiculoneuropathy | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Chronic Kidney Disease | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Congestive Heart Failure | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Cord blood unit clot | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| Cord colitis | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| COVID-19 Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cytomegalovirus colitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
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| Cytomegalovirus meningoencephalitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cytomegalovirus pneumonitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cytomegalovirus Retinitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cytomegalovirus Viremia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Depressed level of consciousness | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Encephalitis | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Epstein-Barr Virus Viremia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Failure to Thrive | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Febrile Neutropenia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Fever | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Fungal Pneumonia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Fungal sinusitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Gastrointestinal bleed | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hemolytic Anemia | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Herpes Simplex Virus Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Human Herpes Virus 6 Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Hyperglycemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hyperkalemia | Metabolism and nutrition disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Infusion-related Reaction | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Intracranial Hemorrhage | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| JC Virus Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Mucoepidermoid carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | CTCAE (4.0) | Systematic Assessment |
| |
| Multi-organ Failure | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Osteomyelitis | Musculoskeletal and connective tissue disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Odynophagia | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Pericardial Effusion | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pericardial Tamponade | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pericarditis | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Peripheral sensorimotor neuropathy | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pleural Effusion | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Post-operative hemorrhage | Injury, poisoning and procedural complications | CTCAE (4.0) | Systematic Assessment |
| |
| Post-transplant Lymphoproliferative Disorder | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pulmonary edema | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pulmonary embolism | Vascular disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pulmonary Hemorrhage | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Pulmonary veno-occlusive disease | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Respiratory Failure | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Small bowel obstruction | Gastrointestinal disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Stroke | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Sweet's Syndrome | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Thrombotic Microangiopathy | Blood and lymphatic system disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Toxoplasmosis infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Upper Respiratory Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Varicella zoster infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Vascular access complication | Injury, poisoning and procedural complications | CTCAE (4.0) | Systematic Assessment |
| |
| Veno-occlusive disease | Hepatobiliary disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Acute Graft-versus-Host Disease | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| Chronic Graft-versus-Host Disease | Investigations | CTCAE (4.0) | Systematic Assessment |
| |
| Graft Failure | Investigations | CTCAE (4.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cytomegalovirus viremia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Bacteremia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| C. difficile colitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Candida esophagitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Catheter Related Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Cytomegalovirus colitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Fever | General disorders | CTCAE (4.0) | Systematic Assessment |
| |
| Hepatitis B Reactivation | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Human Herpes Virus 6 Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Herpes Simplex Virus Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Infectious Diarrhea | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Vaginal yeast infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
| |
| Upper Respiratory Infection | Infections and infestations | CTCAE (4.0) | Systematic Assessment |
|
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexandra Gomez Arteaga, MD | Weill Cornell Medicine | 646-962-7950 | alg9117@med.cornell.edu |
| Aug 5, 2024 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D019337 | Hematologic Neoplasms |
| D009196 | Myeloproliferative Disorders |
| D008223 | Lymphoma |
| D054219 | Neoplasms, Plasma Cell |
| ID | Term |
|---|---|
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
| D009370 | Neoplasms by Histologic Type |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C024352 | fludarabine |
| C042382 | fludarabine phosphate |
| D008558 | Melphalan |
| D000961 | Antilymphocyte Serum |
| C512542 | thymoglobulin |
| D000069283 | Rituximab |
| D014916 | Whole-Body Irradiation |
| D009173 | Mycophenolic Acid |
| D016559 | Tacrolimus |
| ID | Term |
|---|---|
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D058846 | Antibodies, Monoclonal, Murine-Derived |
| D000911 | Antibodies, Monoclonal |
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D018942 | Macrolides |
| D007783 | Lactones |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Acute Lymphoblastic Leukemia |
|
| Chronic Myelogenous Leukemia |
|
| Hodgkin's Lymphoma |
|
| Non-Hodgkin's Lymphoma |
|
| Myelodysplastic Syndrome |
|
| Myeloproliferative Neoplasm |
|
| Other Acute Leukemia |
|
| Other Leukemia (including Chronic Lymphocytic Leukemia) |
|
| Severe Aplastic Anemia |
|
|
| Participants Achieving Engraftment with Minimum TNC of 0.5 x 10^5/kg |
|
|