Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:03 AM
Study NCT ID:
NCT00003662
Status:
COMPLETED
Last Update Posted:
2011-03-07
First Post:
1999-11-01
Brief Title:
Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases
Sponsor:
Roswell Park Cancer Institute
Organization:
Roswell Park Cancer Institute
Study Overview
Official Title:
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Adults and Children With Bone Marrow Failure Syndromes or Inherited Metabolic or Hematologic Diseases
Status:
COMPLETED
Status Verified Date:
2011-03
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 Umbilical cord blood transplantation may be able to replace cells destroyed by chemotherapy or radiation therapy
PURPOSE Phase II trial to study the effectiveness of umbilical cord blood transplantation in treating patients who have hematologic cancer or other hematologic or metabolic diseases
PURPOSE Phase II trial to study the effectiveness of umbilical cord blood transplantation in treating patients who have hematologic cancer or other hematologic or metabolic diseases
Detailed Description:
OBJECTIVES I Determine the rates of durable engraftment in patients with severe aplastic anemia myelodysplastic syndrome inborn errors of metabolism or inherited hematopoietic disorders refractory to medical management who are undergoing high dose chemoradiotherapy followed by unrelated cord blood UCB transplantation II Determine the incidence and severity of acute and chronic graft-versus-host disease in these patients III Monitor overall and event-free survival of these patients IV Evaluate rate and quality of immunologic reconstitution of these patients V Determine whether nucleated cell or progenitor cell content of the graft is predictive of engraftment
OUTLINE This is a multicenter study Patients are stratified according to low vs high weight Patients with severe aplastic anemia myelodysplastic syndrome or bone marrow failure receive cyclophosphamide IV over 1 hour on days -6 to -3 or melphalan IV over 20 minutes on days -4 to -2 antithymocyte globulin ATG IV over 4 hours or methylprednisolone IV over 1 hour twice a day on days -3 to -1 and total lymphoid irradiation on day -1 On day 0 patients receive umbilical cord blood UCB infusion Patients with inborn errors of metabolism or inherited hematopoietic disorders receive oral busulfan every 6 hours on days -9 to -6 cyclophosphamide IV over 1 hour on days -5 to -2 or melphalan IV over 20 minutes on days -4 to -2 and ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -3 to -1 On day 0 patients receive UCB infusion Patients with Fanconis anemia receive ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -6 to -1 cyclophosphamide IV over 1 hour on days -5 to -2 thoracoabdominal irradiation on day -1 and then the UCB infusion on day 0 Patients also receive cyclosporine and methylprednisolone beginning on day -2 and continuing as necessary as graft-versus-host disease prophylaxis Patients are followed indefinitely for survival and late toxicity
PROJECTED ACCRUAL A total of 4-90 patients will be accrued for this study within 5 years
OUTLINE This is a multicenter study Patients are stratified according to low vs high weight Patients with severe aplastic anemia myelodysplastic syndrome or bone marrow failure receive cyclophosphamide IV over 1 hour on days -6 to -3 or melphalan IV over 20 minutes on days -4 to -2 antithymocyte globulin ATG IV over 4 hours or methylprednisolone IV over 1 hour twice a day on days -3 to -1 and total lymphoid irradiation on day -1 On day 0 patients receive umbilical cord blood UCB infusion Patients with inborn errors of metabolism or inherited hematopoietic disorders receive oral busulfan every 6 hours on days -9 to -6 cyclophosphamide IV over 1 hour on days -5 to -2 or melphalan IV over 20 minutes on days -4 to -2 and ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -3 to -1 On day 0 patients receive UCB infusion Patients with Fanconis anemia receive ATG IV over 4 hours or methylprednisolone IV over 1 hour on days -6 to -1 cyclophosphamide IV over 1 hour on days -5 to -2 thoracoabdominal irradiation on day -1 and then the UCB infusion on day 0 Patients also receive cyclosporine and methylprednisolone beginning on day -2 and continuing as necessary as graft-versus-host disease prophylaxis Patients are followed indefinitely for survival and late toxicity
PROJECTED ACCRUAL A total of 4-90 patients will be accrued for this study within 5 years
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 |
|---|---|---|---|
| NCI-G98-1486 | US NIH GrantContract | None | httpsreporternihgovquickSearchP30CA016056 |
| P30CA016056 | NIH | None | None |
| RPCI-RP-9803 | None | None | None |