Ignite Creation Date:
2024-05-05 @ 4:40 PM
Last Modification Date:
2024-10-26 @ 9:23 AM
Study NCT ID:
NCT00295971
Status:
COMPLETED
Last Update Posted:
2012-11-12
First Post:
2006-02-23
Brief Title:
Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome Leukemia Bone Marrow Failure Syndrome or Severe Immunodeficiency Disease
Sponsor:
University of California San Francisco
Organization:
University of California San Francisco
Study Overview
Official Title:
Stem Cell Enriched T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease Leukemia Marrow Failure Syndromes or Severe Immunodeficiency Diseases
Status:
COMPLETED
Status Verified Date:
2012-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
RATIONALE Giving chemotherapy and total body irradiation before a donor bone marrow transplant or peripheral blood stem cell transplant helps stop the growth of cancer cells It also helps stop the patients immune system from rejecting the donors stem cells When the healthy stem cells from a donor are infused into the patient they may help the patients bone marrow make stem cells red blood cells white blood cells and platelets Sometimes the transplanted cells from a donor can make an immune response against the bodys normal cells Giving antithymocyte globulin and removing the T cells from the donor cells before transplant may stop this from happening
PURPOSE This phase I trial is studying the side effects and best dose of donor T cells and antithymocyte globulin when given together with chemotherapy and total-body irradiation in treating young patients who are undergoing T-cell depleted donor stem cell transplant for myelodysplastic syndrome leukemia bone marrow failure syndrome or severe immunodeficiency disease
PURPOSE This phase I trial is studying the side effects and best dose of donor T cells and antithymocyte globulin when given together with chemotherapy and total-body irradiation in treating young patients who are undergoing T-cell depleted donor stem cell transplant for myelodysplastic syndrome leukemia bone marrow failure syndrome or severe immunodeficiency disease
Detailed Description:
OBJECTIVES
Determine the efficacy and toxicity of stem cell-enriched T-cell-depleted haplocompatible allogeneic hematopoietic stem cell transplantation in children with high-risk myelodysplastic syndromes high-risk leukemia severe acquired or congenital cytopenias or primary immunodeficiency diseases
Determine the toxicity of a fludarabine and thiotepa-containing regimen in combination with lower doses of antithymocyte globulin in these patients
Determine the engraftment rate in patients treated with this regimen
Define T-cell reconstitution in these patients
Determine the toxicity and effects of administering stem cell and T-cell boosts after transplantation on hematopoiesis and immune reconstitution in these patients
OUTLINE This is a dose-escalation study of donor CD3 cells and antithymocyte globulin ATG
Cytoreductive regimen Patients undergo total body irradiation twice daily on days -9 to -7 Patients also receive fludarabine IV on days -6 to -2 thiotepa IV every 12 hours on day -6 and ATG IV on days -5 to -2
Transplantation Patients undergo CD34-enriched T-cell-depleted haplocompatible allogeneic peripheral blood stem cell or bone marrow transplantation on day 0
Donor T-cell infusionPatients with no active graft-vs-host disease and evidence of engraftment but low absolute CD34 lymphocyte count at 12 weeks post transplant may receive donor CD3 cells at 4-week intervals
Donor stem cell boost Patients with engraftment but either cytokine or transfusion dependent at 12 weeks post transplant may receive a boost of donor CD34 cells
Cohorts of 3-6 patients receive escalating doses of donor CD3 cells and ATG until the optimum is determined The optimum dose is defined as the dose at which both engraftment and T-cell recovery occur without dose-limiting toxicity in 5 of 6 patients
After the completion of study treatment patients are followed periodically for 5 years and then every 5 years thereafter
PROJECTED ACCRUAL A total of 21 patients will be accrued for this study
Determine the efficacy and toxicity of stem cell-enriched T-cell-depleted haplocompatible allogeneic hematopoietic stem cell transplantation in children with high-risk myelodysplastic syndromes high-risk leukemia severe acquired or congenital cytopenias or primary immunodeficiency diseases
Determine the toxicity of a fludarabine and thiotepa-containing regimen in combination with lower doses of antithymocyte globulin in these patients
Determine the engraftment rate in patients treated with this regimen
Define T-cell reconstitution in these patients
Determine the toxicity and effects of administering stem cell and T-cell boosts after transplantation on hematopoiesis and immune reconstitution in these patients
OUTLINE This is a dose-escalation study of donor CD3 cells and antithymocyte globulin ATG
Cytoreductive regimen Patients undergo total body irradiation twice daily on days -9 to -7 Patients also receive fludarabine IV on days -6 to -2 thiotepa IV every 12 hours on day -6 and ATG IV on days -5 to -2
Transplantation Patients undergo CD34-enriched T-cell-depleted haplocompatible allogeneic peripheral blood stem cell or bone marrow transplantation on day 0
Donor T-cell infusionPatients with no active graft-vs-host disease and evidence of engraftment but low absolute CD34 lymphocyte count at 12 weeks post transplant may receive donor CD3 cells at 4-week intervals
Donor stem cell boost Patients with engraftment but either cytokine or transfusion dependent at 12 weeks post transplant may receive a boost of donor CD34 cells
Cohorts of 3-6 patients receive escalating doses of donor CD3 cells and ATG until the optimum is determined The optimum dose is defined as the dose at which both engraftment and T-cell recovery occur without dose-limiting toxicity in 5 of 6 patients
After the completion of study treatment patients are followed periodically for 5 years and then every 5 years thereafter
PROJECTED ACCRUAL A total of 21 patients will be accrued for this study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UCSF-H411-17122-07 | None | None | None |
| UCSF-01151 | None | None | None |