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| ID | Type | Description | Link |
|---|---|---|---|
| IRB00249753 | Other Identifier | JHU ACH IRB |
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This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TCR alpha beta T cell depletion | Experimental | The leukapheresis product will undergo TCR alpha beta negative selection following a standardized protocol |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Haploidentical Hematopoietic Cell Transplantation | Biological | TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantation |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of successful donor engraftment | The incidence of engraftment at day 100 will be described based on donor chimerism in the whole blood and or fractions sorted for T-cell and myeloid subsets. The donor chimerism will be scored as autologous reconstitution (< 5% donor), mixed chimerism (5-49%=low mixed, 50-95%=high mixed), > 95%=full donor chimerism. | Day 100 after transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival and Event-free survival | Overall survival is defined as the time of enrollment to death from any cause or last follow up. Event-free survival is defined as the time of enrollment to death, primary or secondary graft failure, graft failure necessitating a second HCT procedure, DLI or stem cell boost given for treatment of falling chimerism, or disease recurrence | Up to 2 years post transplant |
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Inclusion Criteria:
Severe sickle cell disease (HbSS, HbSC, HbSB0, HbSB+, HbSD, HbSE) with at least one of the following criteria:
Thalassemia major with at least one of the following criteria:
Bone marrow failure syndromes and autoimmune cytopenias:
Inclusion Criteria:
Patient has a suitable genotypic identical match of 5/10. The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1 and HLA-DQB1.
Patients must have adequate organ function measured by:
Signed written informed consent
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jade Hanson, MSN | Contact | 7277676468 | jade.hanson@jhmi.edu |
| Name | Affiliation | Role |
|---|---|---|
| Deepak Chellapandian, MD | Johns Hopkins All Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins All Children's Hospital | Recruiting | St. Petersburg | Florida | 33701 | United States |
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| Kinetics of neutrophil and platelet engraftment | Neutrophil engraftment defined as absolute neutrophil count ≥500/μL for 3 consecutive measurements on different days and platelet engraftment defined as sustained platelet count >20,000/μL and >50,000//μL with no platelet transfusions in the preceding seven days. | Up to 42 days post transplant |
| Transplant-related mortality | Rate of transplant-related mortality | Up to 100 days post transplant |
| Acute grade II-IV GvHD and Chronic GvHD | Incidence and severity of acute and chronic graft versus host disease | Up to 2 years post transplant |
| Primary and secondary graft failure | Rates of primary and secondary graft failure | Up to 2 years post transplant |
| Transplant-related complications and infections | Frequency of transplant-related complications and rate of infections following transplantation | Up to 2 years post transplant |
| Cellular and Immunological reconstitution by laboratory evaluations | The recovery of different lymphocyte subpopulation (CD3+; CD4+; CD8+; CD3+CD45RA+and CD45RO; TCR alpha beta; TCR gamma delta; CD19+) | Up to 2 years post transplant |
| ID | Term |
|---|---|
| D006453 | Hemoglobinopathies |
| D000741 | Anemia, Aplastic |
| D000080983 | Bone Marrow Failure Disorders |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000740 | Anemia |
| D001855 | Bone Marrow Diseases |
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