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Post-transplant cyclophosphamide and abatacept can be used as standard-of-care approaches to GVHD prevention in the HLA mismatched donor-recipient setting and are simpler to use since study enrollment is not required.
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The purpose of this study is to investigate the safety and efficacy of TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) for malignant and non-malignant disorders in children and adolescent/young adult patients using the CliniMACS® immunomagnetic selection device (Miltenyi Biotec).
Acute graft versus host disease (GVHD) remains a significant cause of morbidity and mortality and is the biggest barrier to successful allogeneic hematopoietic cell transplantation (HSCT) outcomes. Improved methods of acute GVHD prevention are needed. TCRαβ+/CD19+ depletion of allogeneic hematopoietic stem cell products offers an opportunity to limit the risk of acute GVHD by removing TCRαβ+ T cells and CD19+ B cells which participate in acute GVHD initiation and perpetuation. The purpose of this study is to investigate the safety and efficacy of TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) for malignant and non-malignant disorders in children and adolescent/young adult patients using the CliniMACS® immunomagnetic selection device (Miltenyi Biotec).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TCRαβ+/CD19+ depleted HSCT | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) | Drug | The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Infusion-related Reactions | Number of patients who experienced infusion reactions including rash, fever, difficulty breathing, and blood pressure abnormalities at the time of infusion of stem cells. | 100 days |
| Measure | Description | Time Frame |
|---|---|---|
| Engraftment and Sustained Donor Chimerism | Initial neutrophil engraftment prior to day +28 was determined by monitoring for neutrophil count recovery post-transplant and performing blood tests to confirm presence of donor cells. Sustained donor chimerism at 1 year post transplant was determined again by performing blood tests to confirm presence of donor cells. | 28 days and 1 year |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | TCRαβ+/CD19+ Depleted HSCT | TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold. |
| 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 | Jan 27, 2021 |
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| Number of Participants With Acute GVHD | Patients were monitored for symptoms of acute graft versus host disease including rash, diarrhea, and increased bilirubin using the Modified Glucksberg Criteria. | 100 days |
| Number of Participants With Chronic GVHD | Patients were monitored for symptoms of chronic graft versus host disease using the NIH Consensus Criteria. | 1 year |
| GVHD-free Survival | GVHD-free survival was determined based on the presence or not of acute or chronic GVHD at 1 year. | 1 year |
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| ID | Title | Description |
|---|---|---|
| BG000 | TCRαβ+/CD19+ Depleted HSCT | TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold. |
| Units | Counts |
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| 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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| Race/Ethnicity, Customized | 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 | ||||||||||||||||||||||
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| Primary | Incidence of Infusion-related Reactions | Number of patients who experienced infusion reactions including rash, fever, difficulty breathing, and blood pressure abnormalities at the time of infusion of stem cells. | Posted | Number | number of infusion reactions | 100 days | infusions | infusions |
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| Secondary | Engraftment and Sustained Donor Chimerism | Initial neutrophil engraftment prior to day +28 was determined by monitoring for neutrophil count recovery post-transplant and performing blood tests to confirm presence of donor cells. Sustained donor chimerism at 1 year post transplant was determined again by performing blood tests to confirm presence of donor cells. | Posted | Count of Participants | Participants | 28 days and 1 year |
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| Secondary | Number of Participants With Acute GVHD | Patients were monitored for symptoms of acute graft versus host disease including rash, diarrhea, and increased bilirubin using the Modified Glucksberg Criteria. | 14 patients received 15 transplants. | Posted | Count of Participants | Participants | 100 days |
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| Secondary | Number of Participants With Chronic GVHD | Patients were monitored for symptoms of chronic graft versus host disease using the NIH Consensus Criteria. | Five patients were evaluable for chronic graft versus host disease at 1 year time point. | Posted | Count of Participants | Participants | 1 year |
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| Secondary | GVHD-free Survival | GVHD-free survival was determined based on the presence or not of acute or chronic GVHD at 1 year. | Posted | Count of Participants | Participants | 1 year |
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Adverse Events were collected for the first week of the study. Serious Adverse Events and All-Cause Mortality were collected up to 1 year.
Only events that were determined to be attributable to study intervention noted after the first week were recorded and reported.
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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 | TCRαβ+/CD19+ Depleted HSCT | TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold. | 6 | 15 | 2 | 15 | 7 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
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| Sepsis | Infections and infestations | CTCAE (5.0) | Systematic Assessment |
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| Pericardial effusion | Cardiac disorders | CTCAE (5.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | CTCAE (5.0) | Systematic Assessment |
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| Anorexia | Metabolism and nutrition disorders | CTCAE (5.0) | Systematic Assessment |
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| Worsening pain | General disorders | CTCAE (5.0) | Systematic Assessment |
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| Hypokalemia | Investigations | CTCAE (5.0) | Systematic Assessment |
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| Hypertension | Vascular disorders | CTCAE (5.0) | Systematic Assessment |
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| Hypotension | Vascular disorders | CTCAE (5.0) | Systematic Assessment |
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| Bacteremia (strep mitis) | Infections and infestations | CTCAE (5.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Rebecca Marsh, MD | Cincinnati Children's Hospital Medical Center | (513) 803-1139 | Rebecca.Marsh@cchmc.org |
| Jun 13, 2023 |
| Prot_SAP_000.pdf |
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| African American |
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