Ignite Creation Date:
2024-05-05 @ 11:20 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004378
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Brief Title:
Stem Cell Transplantation SCT for Genetic Diseases
Sponsor:
National Center for Research Resources NCRR
Organization:
National Center for Research Resources NCRR
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2002-04
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 Ascertain whether stem cell transplantation SCT is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism
II Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment
II Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment
Detailed Description:
PROTOCOL OUTLINE Patients receive either cyclophosphamide and high dose total body irradiation TBI or busulfan and cyclophosphamide
Cyclophosphamide IV is given on days -5 and -4 and TBI on days -2 -1 and 0 Busulfan is given orally every 6 hours on days -9 through -6 and cyclophosphamide IV on days -5 through -2 Patients rest on day -1
Patients receive bone marrow infusion on day 0 For GVHD prophylaxis patients receive methotrexate on day 1 then on days 3 6 and 11 Cyclosporine IV begins on day -2 over 12 hours followed by continuous infusion for 21 days Then oral doses of cyclosporine are given every 12 hours to patients who tolerate oral feeding Cyclosporine is continued 6 months posttransplant then tapered 10 per week and stopped
Patients who receive genotypically HLA nonidentical stem cells undergo additional GVHD prophylaxis with methylprednisolone IV or PO or its equivalent every 12 hours on days 3 to day 100 Dose is then tapered as tolerated over 1 month
Patients who receive cord blood stem cells receive methylprednisolone instead of methotrexate for GHVD prophylaxis Methylprednisolone is given 3 times daily beginning on day 5 and continuing until day 17 Then methylprednisolone is tapered 10 per week as clinically tolerated
To accelerate engraftment patients receive filgrastim IM daily beginning on day 1 and continuing until ANC equals 5000
Cyclophosphamide IV is given on days -5 and -4 and TBI on days -2 -1 and 0 Busulfan is given orally every 6 hours on days -9 through -6 and cyclophosphamide IV on days -5 through -2 Patients rest on day -1
Patients receive bone marrow infusion on day 0 For GVHD prophylaxis patients receive methotrexate on day 1 then on days 3 6 and 11 Cyclosporine IV begins on day -2 over 12 hours followed by continuous infusion for 21 days Then oral doses of cyclosporine are given every 12 hours to patients who tolerate oral feeding Cyclosporine is continued 6 months posttransplant then tapered 10 per week and stopped
Patients who receive genotypically HLA nonidentical stem cells undergo additional GVHD prophylaxis with methylprednisolone IV or PO or its equivalent every 12 hours on days 3 to day 100 Dose is then tapered as tolerated over 1 month
Patients who receive cord blood stem cells receive methylprednisolone instead of methotrexate for GHVD prophylaxis Methylprednisolone is given 3 times daily beginning on day 5 and continuing until day 17 Then methylprednisolone is tapered 10 per week as clinically tolerated
To accelerate engraftment patients receive filgrastim IM daily beginning on day 1 and continuing until ANC equals 5000
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 |
|---|---|---|---|
| UCLA-92010034 | None | None | None |