Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00011830
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2001-02-28
Brief Title:
Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Pilot Study of G-CSF Induced Stem Cell Mobilization Potential in Patients With Relapsed Severe Aplastic Anemia
Status:
COMPLETED
Status Verified Date:
2006-02
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:
This study will examine 1 whether it is possible to collect enough stem cells cells produced by the bone marrow that mature into white and red blood cells and platelets from patients with aplastic anemia to use for future treatment and 2 whether patients who have been treated successfully and relapse will benefit from autologous stem cell transfusion transfusion of their own stem cells
Patients 12 years of age or older with aplastic anemia who have been successfully treated with immunosuppressive drugs and are now in remission may be eligible for this study Participants will undergo a complete history and physical examination bone marrow biopsy removal of a small sample of bone marrow from the hip bone and blood tests plus procedures to collect stem cells as follows
G-CSF Filgrastim administration - G-CSF will be given by injection under the skin daily for up to 10 days This drug causes stem cells to move from the marrow into the blood where they can be collected more easily
Apheresis - Stem cells will be collected through apheresis usually starting the 5th to 6th day of Filgrastim injections For this procedure whole blood is collected through a needle in an arm vein The blood circulates through a cell separator machine where the white cells and stem cells are removed The red cells platelets and plasma are returned to the body through a second needle in the other arm The procedure takes about 5 hours Up to five procedures done on consecutive days may be required to collect enough cells for transplantation If enough cells are collected they will be purified treated to remove the white blood cells using an experimental device Removing the lymphocytes may reduce the chance of relapse of aplastic anemia following the stem cell transplant The stem cells will be frozen for later use if needed
Follow-up - Participants are followed at NIH at 6-month intervals
Patients 12 years of age or older with aplastic anemia who have been successfully treated with immunosuppressive drugs and are now in remission may be eligible for this study Participants will undergo a complete history and physical examination bone marrow biopsy removal of a small sample of bone marrow from the hip bone and blood tests plus procedures to collect stem cells as follows
G-CSF Filgrastim administration - G-CSF will be given by injection under the skin daily for up to 10 days This drug causes stem cells to move from the marrow into the blood where they can be collected more easily
Apheresis - Stem cells will be collected through apheresis usually starting the 5th to 6th day of Filgrastim injections For this procedure whole blood is collected through a needle in an arm vein The blood circulates through a cell separator machine where the white cells and stem cells are removed The red cells platelets and plasma are returned to the body through a second needle in the other arm The procedure takes about 5 hours Up to five procedures done on consecutive days may be required to collect enough cells for transplantation If enough cells are collected they will be purified treated to remove the white blood cells using an experimental device Removing the lymphocytes may reduce the chance of relapse of aplastic anemia following the stem cell transplant The stem cells will be frozen for later use if needed
Follow-up - Participants are followed at NIH at 6-month intervals
Detailed Description:
Immune mechanisms are responsible for hematopoietic failure in most cases of acquired aplastic anemia AA a disease characterized by hypocellular bone marrow and pancytopenia In aplastic anemia much experimental data points toward an immune-mediated pathophysiology of destruction of hematopoietic progenitor and stem cells Clinically immunosuppressive therapies usually anti-thymocyte globulin ATG and cyclosporine CsA have been shown to be effective in a large proportion of patients with severe AA However at 2 years after initial treatment as many as 35 of patients with initially good response and normal blood counts relapse and require repeated cycles of intense immunosuppression andor chronic immunosuppressive regimens Although relapse in previously immunosuppression-responsive patients has a generally good prognosis there is an increased risk of complications and treatment-related toxicities The outlook of patients who fail to respond to repeated intensive immunosuppression is poor While it is likely that some of the treatment failures occurring with conventional immunosuppressive regimens both at presentation and in relapsed patients may be due to inability to suppress the autoimmune process leading to the bone marrow failure more intense therapies such as cyclophosphamide have a high complication rate due to prolonged and dose-related myelosuppression In this protocol we propose that patients with severe AA who show a good response to the initial round of immunosuppression undergo stem cell mobilization collection and cryopreservation
This pilot study of 20 patients is designed to evaluate 1 the CD34 cell mobilization response to administration of standard doses of granulocyte-colony stimulating factor G-CSF and 2 the potential for collecting stem cells from patients with a history of severe AA who have been given G-CSF Outcome parameters to be monitored are the mobilization response to G-CSF and the safety profile and tolerance of G-CSF and leukapheresis Effectiveness will be gauged by historical comparison of these parameters to normal healthy age-matched volunteers
It is important to point out that there is no therapeutic intent to the majority of this protocol or direct benefit for enrolled patients We do plan however to cryopreserve the remainder of the mobilized cells collected by apheresis for possible autologous transplantation in the event of the patients progression to leukemia or bone marrow failure in the future
This pilot study of 20 patients is designed to evaluate 1 the CD34 cell mobilization response to administration of standard doses of granulocyte-colony stimulating factor G-CSF and 2 the potential for collecting stem cells from patients with a history of severe AA who have been given G-CSF Outcome parameters to be monitored are the mobilization response to G-CSF and the safety profile and tolerance of G-CSF and leukapheresis Effectiveness will be gauged by historical comparison of these parameters to normal healthy age-matched volunteers
It is important to point out that there is no therapeutic intent to the majority of this protocol or direct benefit for enrolled patients We do plan however to cryopreserve the remainder of the mobilized cells collected by apheresis for possible autologous transplantation in the event of the patients progression to leukemia or bone marrow failure in the future
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 |
|---|---|---|---|
| 01-H-0083 | None | None | None |