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Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.
Patients wiith selected malignant or non-malignant conditions meeting eligibility criteria will be enrolled on this study. Patients will receive one of either full intensity, reduced intensity, or reduced toxicity conditioning appropriate based on disease, disease status, organ function and performance status and will undergo α/β T-cell and CD 19+ B cell depleted alloSCT.
Patients will be following for engraftment, chimerism, immune reconstitution, GVHD and QOL.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| alpha beta cell depletion | Experimental | Matched allogeneic donor stem cells will be processed utilizing α/β CD3+/CD19+ cell depletion with the Prodigy system. Standard pre-conditioning and post-transplant motioning will be given. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| alpha beta depletion | Drug | donor cells will be collected and subsequently undergo α/β CD3+/CD19+ cell depletion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| incidence of adverse events related to administration of α/β CD3+/CD19+ cell depleted stem cells | patients will be monitored for any adverse events related to administration of α/β CD3+/CD19+ cell depleted stem cells | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| incidence of hematpoitic engraftment following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion | patients will have routine chimerism performed to monitoring engraftment of donor cells | 1 year |
| incidence of GVHD following Allogeneic stem cell transplantation (AlloSCT) utilizing α/β CD3+/CD19+ cell depletion |
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Inclusion Criteria:
5. Bone marrow failure syndromes: Kostmann syndrome refractory or intolerant to granulocyte colony-33stimulating factor; Diamond-Blackfan anemia refractory or intolerant to corticosteroids and/or cyclosporine'; amegakaryocytic thrombocytopenia 6. Sickle Cell Disease (Homozygous Hemoglobin S Disease, or Hemoglobin S β 0/+ thalassemia, or Hemoglobin SC Disease) 7. age 0-30 years 8. adequate organ function
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Mitchell S Cairo, MD | Contact | 9145942150 | mitchell_cairo@nymc.edu | |
| Lauren Harrison, RN | Contact | 6172857844 | lauren_harrison@nymc.edu |
| Name | Affiliation | Role |
|---|---|---|
| Mitchell S Cairo | New York Medical College | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| New York Medical College | Recruiting | Valhalla | New York | 10595 | United States |
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patients will be monitored post transplant for signs of acute and chronic GVHD |
| 1 year |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| D008228 | Lymphoma, Non-Hodgkin |
| D006689 | Hodgkin Disease |
| C537592 | Neutropenia, Severe Congenital, Autosomal Recessive 3 |
| D029503 | Anemia, Diamond-Blackfan |
| D000755 | Anemia, Sickle Cell |
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D008223 | Lymphoma |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D029502 | Anemia, Hypoplastic, Congenital |
| D012010 | Red-Cell Aplasia, Pure |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D006453 | Hemoglobinopathies |
| D013789 | Thalassemia |
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