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The purpose of this protocol is to provide access to the CliniMACS® System to hematopoietic cell transplant (HSCT) patients who do not have a matched related donor. The CliniMACS system is currently approved for use in patients who have AML, and a genetically matched sibling donor. Through this protocol, the investigators will be able to offer potentially life-saving transplants to patients who have genetically mis-matched donor, who have no other options for treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ARM A Malignant TBI | Experimental | Malignant diseases Conditioning including total body irradiation and chemotherapy |
|
| ARM B Malignant Non-TBI | Experimental | Malignant diseases chemotherapy based conditioning |
|
| ARM C Non-malignant | Experimental | Non-malignant diseases Chemotherapy based conditioning |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CliniMACS CD34+ cell enrichment and T-cell depletion | Device |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD) | GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient. | Day +100 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Graft Failure | Failure of donor stem cells to make neutrophils | Up to Day +42 after stem cell transplant |
| Length of Time to Engraftment | Absolute neutrophil count (ANC) >500 for 3 consecutive days and >80% donor cells in blood. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Rajni Agarwal, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Children's Hospital | Palo Alto | California | 94305 | United States |
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| Label | URL |
|---|---|
| Stanford Stem Cell Transplantation website | View source |
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Enrollment was open to participants with malignant or non-malignant disorders receiving mismatched related donor hematopoietic stem cell transplants who could benefit from augmented CD34+ cells and T cell-depleted products to prevent severe (grade III/IV) acute Graft vs Host Disease (GVHD). Participants were withdrawn from the study at the time of graft failure due to need for exclusionary concurrent treatment (per PI discretion)
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| ID | Title | Description |
|---|---|---|
| FG000 | ARM A Malignant TBI | Malignant diseases Conditioning including total body irradiation and chemotherapy CliniMACS CD34+ cell enrichment and T-cell depletion |
| FG001 | ARM B Malignant Non-TBI |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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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 | Apr 2, 2018 |
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| up to +1 year post-transplant |
| Chimerism of Donor Cells | The percentage of donor cells for all evaluable (without disease progression) patients | Day +100 post-transplant |
| Immune Recovery (CD4) | The time to CD4 count >100 | up to +1 year post-transplant |
| Number of Participants With Immune Recovery (CD4 >200) by Year 1 | up to +1 year post-transplant |
| Immune Recovery Shown as Phytohemagglutin (PHA) | Immune recovery defined as achieving normal levels of PHA (53,000-200,000 CPM) | 6 months and 1 year post-transplant |
| Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD) | Post-transplant lymphoproliferative disorder (PTLD) is a well-known, life-threatening complication of organ transplantation, predominantly occurring after solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT). | up to +1 year post-transplant |
| Number of Patients With Severe Toxicities | Incidence of transplant-related toxicities | up to +1 year post-transplant |
| Number of Participants Experiencing Post-transplant Infections | Post-transplant infections will be described by incidence and type. Participants may have had more than one type of infection. | up to +1 year post-transplant |
| Transplant-related Mortality (TRM) | Death related to transplant | at Day +100 and +1 year post-transplant |
Malignant diseases chemotherapy based conditioning
CliniMACS CD34+ cell enrichment and T-cell depletion
| FG002 | ARM C Non-malignant | Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion |
| Achieved Engraftment |
|
| COMPLETED |
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| NOT COMPLETED |
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Malignant Arms did not recruit due to other available studies for these patients.
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| ID | Title | Description |
|---|---|---|
| BG000 | ARM A Malignant TBI | Malignant diseases Conditioning including total body irradiation and chemotherapy CliniMACS CD34+ cell enrichment and T-cell depletion |
| BG001 | ARM B Malignant Non-TBI | Malignant diseases chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion |
| BG002 | ARM C Non-malignant | Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | 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 Patients With Severe (Grade III/IV) Acute Graft vs Host Disease (GVHD) | GVHD is a condition that occurs when donor bone marrow or stem cells attack the recipient. | Posted | Count of Participants | Participants | Day +100 |
|
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| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Graft Failure | Failure of donor stem cells to make neutrophils | Posted | Count of Participants | Participants | Up to Day +42 after stem cell transplant |
|
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| |||||||||||||||||||||||||||
| Secondary | Length of Time to Engraftment | Absolute neutrophil count (ANC) >500 for 3 consecutive days and >80% donor cells in blood. | Patients who had primary graft failure were withdrawn from the study are excluded from the analysis. | Posted | Mean | Standard Deviation | days | up to +1 year post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Chimerism of Donor Cells | The percentage of donor cells for all evaluable (without disease progression) patients | Patients who had primary graft failure were withdrawn from the study are excluded from the analysis. | Posted | Mean | Standard Deviation | Percentage of cells from donor | Day +100 post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Immune Recovery (CD4) | The time to CD4 count >100 | Patients who had primary graft failure and were withdrawn from the study are excluded from the analysis. | Posted | Mean | Standard Deviation | days | up to +1 year post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants With Immune Recovery (CD4 >200) by Year 1 | Patients who had primary graft failure and were withdrawn from the study are excluded from the analysis. | Posted | Count of Participants | Participants | up to +1 year post-transplant |
|
| ||||||||||||||||||||||||||||
| Secondary | Immune Recovery Shown as Phytohemagglutin (PHA) | Immune recovery defined as achieving normal levels of PHA (53,000-200,000 CPM) | Participants who had primary graft failure were withdrawn from the study and unable to be evaluated for this outcome measure. | Posted | Mean | Standard Deviation | Net CPM | 6 months and 1 year post-transplant |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Patients With Post-transplant Lymphoproliferative Disease (PTLD) | Post-transplant lymphoproliferative disorder (PTLD) is a well-known, life-threatening complication of organ transplantation, predominantly occurring after solid organ transplantation (SOT) and hematopoietic stem cell transplantation (HSCT). | Posted | Count of Participants | Participants | up to +1 year post-transplant |
|
| ||||||||||||||||||||||||||||
| Secondary | Number of Patients With Severe Toxicities | Incidence of transplant-related toxicities | Posted | Count of Participants | Participants | up to +1 year post-transplant |
|
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| |||||||||||||||||||||||||||
| Secondary | Number of Participants Experiencing Post-transplant Infections | Post-transplant infections will be described by incidence and type. Participants may have had more than one type of infection. | Posted | Count of Participants | Participants | up to +1 year post-transplant |
|
| ||||||||||||||||||||||||||||
| Secondary | Transplant-related Mortality (TRM) | Death related to transplant | Posted | Count of Participants | Participants | at Day +100 and +1 year post-transplant |
|
|
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Up to 1 year post-transplant.
Recipients of allogeneic hematopoietic cell transplantation face significant risks. The risk of treatment related morbidity and mortality following allogeneic transplant is related to the underlying disease, disease status at the time of transplant, prior therapies and other coexisting health problems. Therefore, in accordance with the study protocol, only AE's found to be directly related to the study-mandated intervention were captured.
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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 | ARM C Non-malignant | Non-malignant diseases Chemotherapy based conditioning CliniMACS CD34+ cell enrichment and T-cell depletion | 2 | 3 | 0 | 3 | 0 | 3 |
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This study did not meet its planned enrollment. Statistical power was not achieved for any analysis.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Erica Goodwin | Stanford University | 253-508-3556 | egood55@stanford.edu |
| Apr 27, 2023 |
| Prot_SAP_000.pdf |
| Between 18 and 65 years |
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| >=65 years |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Categories |
|---|
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Whole Blood |
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| CD3 Cells |
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| CD15 Cells |
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| CD19 |
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| CD34 |
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| CD56 |
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| Title | Denominators | Categories |
|---|
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| Title | Denominators | Categories |
|---|
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| 6 months |
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| 1 year |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
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| Categories |
|---|
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| Title | Denominators | Categories |
|---|
| BK viremia |
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| BK viruria |
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| Norovirus |
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| HHV6 viremia |
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| EBV viremia |
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| Rhinovirus/Enterovirus |
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| Parainfluenza 3 upper respiratory infection |
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| Adenovirus (low level reactivation) |
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| Cytomegalovirus (low level reactivation) |
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| Day 100 |
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| Between Day 100 and 1 Year |
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