Ignite Creation Date:
2024-05-05 @ 10:23 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00006054
Status:
TERMINATED
Last Update Posted:
2009-10-15
First Post:
2000-07-05
Brief Title:
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
Sponsor:
Fairview University Medical Center
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
TERMINATED
Status Verified Date:
2003-10
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:
OBJECTIVES I Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies
II Determine relevant outcomes of this treatment in these patients including quality of survival extent of morbidity and mortality from complications of the treatment eg graft versus host disease regimen related toxicities B- cell lymphoproliferative disease and completeness of functional immunoreconstitution
II Determine relevant outcomes of this treatment in these patients including quality of survival extent of morbidity and mortality from complications of the treatment eg graft versus host disease regimen related toxicities B- cell lymphoproliferative disease and completeness of functional immunoreconstitution
Detailed Description:
PROTOCOL OUTLINE Patients with severe combined immunodeficiency SCID using a matched sibling donor receive allogeneic bone marrow or umbilical cord blood transplantation on day 0 Patients receive graft versus host disease GVHD prophylaxis with methotrexate IV on days 1 3 6 and 11 and cyclosporine IV on days -3 to 50
Patients with SCID using donors other than histocompatible siblings Wiskott Aldrich syndrome using a histocompatible sibling donor Wiskott Aldrich syndrome and under 5 years of age using donors other than histocompatible siblings X-linked CD40 ligand deficiency using a histocompatible sibling donor X-linked CD40 ligand deficiency and under 5 years of age using donors other than histocompatible siblings other primary immunodeficiencies without manifestations of hemophagocytosis using a histocompatible sibling donor or other primary immunodeficiencies without manifestations of hemophagocytosis and under 5 years of age using donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on days -9 to -6 cyclophosphamide IV on days -5 to -2 and antithymocyte globulin ATG twice daily on days -4 to -1 Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0 Patients receive graft versus host disease GVHD prophylaxis with methotrexate IV on days 1 3 6 and 11 and cyclosporine IV on days -3 to 50
Patients with hemophagocytic lymphohistiocytosis Chediak Higashi syndrome X-linked lymphoproliferative syndrome severe progressive Langerhans cell histiocytosis or other primary immunodeficiencies with complications of hemophagocytosis receive busulfan IV over 2 hours every 6 hours on days -9 to -6 cyclophosphamide IV over 2 hours on days -5 to -2 etoposide IV over 22 hours on days -5 to -3 and ATG IV twice daily on days -2 -1 1 and 2 Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0 Patients receive graft versus host disease GVHD prophylaxis with methotrexate IV on days 1 3 6 and 11 and cyclosporine IV on days -3 to 50
Patients with Wiskott Aldrich syndrome or other primary immunodeficiencies without manifestations of hemophagocytosis who are over 5 years of age and using donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on days -6 and -5 cyclophosphamide IV over 2 hours on days -4 and -3 total body irradiation on day -2 and ATG IV over 2 hours twice daily on days -2 -1 2 and 3 Allogeneic bone marrow or umbilical cord blood transplantation takes place on days 0 and 1 Patients receive GVHD prophylaxis with methylprednisolone IV every 12 hours on days 2-21 oral prednisone every 12 hours on days 22-100 and then tapered off over days 101 to 128 and cyclosporine IV over 2 hours every 8-12 hours on days -3 to 100
All patients are followed as determined by their primary physician
Patients with SCID using donors other than histocompatible siblings Wiskott Aldrich syndrome using a histocompatible sibling donor Wiskott Aldrich syndrome and under 5 years of age using donors other than histocompatible siblings X-linked CD40 ligand deficiency using a histocompatible sibling donor X-linked CD40 ligand deficiency and under 5 years of age using donors other than histocompatible siblings other primary immunodeficiencies without manifestations of hemophagocytosis using a histocompatible sibling donor or other primary immunodeficiencies without manifestations of hemophagocytosis and under 5 years of age using donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on days -9 to -6 cyclophosphamide IV on days -5 to -2 and antithymocyte globulin ATG twice daily on days -4 to -1 Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0 Patients receive graft versus host disease GVHD prophylaxis with methotrexate IV on days 1 3 6 and 11 and cyclosporine IV on days -3 to 50
Patients with hemophagocytic lymphohistiocytosis Chediak Higashi syndrome X-linked lymphoproliferative syndrome severe progressive Langerhans cell histiocytosis or other primary immunodeficiencies with complications of hemophagocytosis receive busulfan IV over 2 hours every 6 hours on days -9 to -6 cyclophosphamide IV over 2 hours on days -5 to -2 etoposide IV over 22 hours on days -5 to -3 and ATG IV twice daily on days -2 -1 1 and 2 Allogeneic bone marrow or umbilical cord blood transplantation takes place on day 0 Patients receive graft versus host disease GVHD prophylaxis with methotrexate IV on days 1 3 6 and 11 and cyclosporine IV on days -3 to 50
Patients with Wiskott Aldrich syndrome or other primary immunodeficiencies without manifestations of hemophagocytosis who are over 5 years of age and using donors other than histocompatible siblings receive busulfan IV over 2 hours every 6 hours on days -6 and -5 cyclophosphamide IV over 2 hours on days -4 and -3 total body irradiation on day -2 and ATG IV over 2 hours twice daily on days -2 -1 2 and 3 Allogeneic bone marrow or umbilical cord blood transplantation takes place on days 0 and 1 Patients receive GVHD prophylaxis with methylprednisolone IV every 12 hours on days 2-21 oral prednisone every 12 hours on days 22-100 and then tapered off over days 101 to 128 and cyclosporine IV over 2 hours every 8-12 hours on days -3 to 100
All patients are followed as determined by their primary physician
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UMN-MT-9526 | None | None | None |
| UMN-MT-1995-26 | None | None | None |