Ignite Creation Date:
2024-05-05 @ 10:17 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001765
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Low Intensity Preparative Regimen Followed by HLA-Matched Mobilized Peripheral Blood Stem Cell Transplantation for Chronic Granulomatous Disease
Status:
COMPLETED
Status Verified Date:
2005-02
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 investigate the safety and effectiveness of a modified stem cell transplant procedure for treating chronic granulomatous disease CGD in patients with active infection CGD is an inherited disorder of neutrophils-a type of infection-fighting white blood cell-that leaves patients vulnerable to life-threatening infections Transplantation of donated stem cells cells produced by the bone marrow that mature into the different blood components-white cells red cells and platelets can cure CGD However this procedure carries a significant risk of death particularly in patients with active infection because it requires completely suppressing the immune system with high-dose chemotherapy and radiation In addition lymphocytes-another type of infection-fighting white blood cell-from the donor may cause what is called graft vs host disease GvHD in which the donor cells recognize the patients cells as foreign and mount an immune response to destroy them To try to reduce these risks patients in this study will be given low-dose chemotherapy and no radiation a regimen that is easier for the body to tolerate and involves a shorter period of complete immune suppression Also the donors lymphocytes will be removed from the rest of the stem cells to be transplanted reducing the risk of GvHD
Patients with CGD between the ages of age 1 and 55 years old who do not have an active infection and who have a family member that is a well matched donor may be eligible for this study Candidates will have a medical history physical examination and blood tests lung and heart function tests X-rays of the chest and sinuses and dental and eye examinations A bone marrow sample may be taken to evaluate disease status This test done under a local anesthetic uses a special needle to draw bone marrow from the hipbone
Stem cells will be collected from both the patient and donor To do this the hormone G-CSF will be injected under the skin for several days to increase stem cell production Then the stem cells will be collected by apheresis In this procedure the blood is drawn through a needle placed in one arm pumped into a machine where the desired cells are separated out and removed and then the rest of the blood is returned through a needle in the other arm
A large plastic tube central venous line is placed into a major vein It can stay in the body and be used the entire treatment period to deliver the donated stem cells give chemotherapy or other medications including antibiotics and blood transfusions if needed and withdraw blood samples Several days before the transplant procedure patients will start low-dose chemotherapy with cyclophosphamide and fludarabine two commonly used anti-cancer drugs They will also be given anti-thymocyte globulin to prevent rejection of the donated cells When this conditioning therapy is completed the stem cells will be infused through the central line Patients will be given cyclosporine by mouth or by vein from 4 days before until 3 months after the stem cell transplant to help prevent rejection
The average hospital stay for stem cell transplant is 30 days After discharge patients will return for follow-up clinic visits weekly or twice weekly for 4 months These visits will include a symptom check physical examination and blood tests Blood transfusions will be given if needed Subsequent visits will be scheduled at 4 6 12 18 24 30 and 36 months after the transplant or more often if required and then yearly
Patients with CGD between the ages of age 1 and 55 years old who do not have an active infection and who have a family member that is a well matched donor may be eligible for this study Candidates will have a medical history physical examination and blood tests lung and heart function tests X-rays of the chest and sinuses and dental and eye examinations A bone marrow sample may be taken to evaluate disease status This test done under a local anesthetic uses a special needle to draw bone marrow from the hipbone
Stem cells will be collected from both the patient and donor To do this the hormone G-CSF will be injected under the skin for several days to increase stem cell production Then the stem cells will be collected by apheresis In this procedure the blood is drawn through a needle placed in one arm pumped into a machine where the desired cells are separated out and removed and then the rest of the blood is returned through a needle in the other arm
A large plastic tube central venous line is placed into a major vein It can stay in the body and be used the entire treatment period to deliver the donated stem cells give chemotherapy or other medications including antibiotics and blood transfusions if needed and withdraw blood samples Several days before the transplant procedure patients will start low-dose chemotherapy with cyclophosphamide and fludarabine two commonly used anti-cancer drugs They will also be given anti-thymocyte globulin to prevent rejection of the donated cells When this conditioning therapy is completed the stem cells will be infused through the central line Patients will be given cyclosporine by mouth or by vein from 4 days before until 3 months after the stem cell transplant to help prevent rejection
The average hospital stay for stem cell transplant is 30 days After discharge patients will return for follow-up clinic visits weekly or twice weekly for 4 months These visits will include a symptom check physical examination and blood tests Blood transfusions will be given if needed Subsequent visits will be scheduled at 4 6 12 18 24 30 and 36 months after the transplant or more often if required and then yearly
Detailed Description:
Chronic Granulomatous Disease CGD is an inherited disorder of neutrophil function Patients are profoundly immunocompromised and are plagued early in life with recurrent and life threatening infections Allogeneic stem cell transplantation is the only cure for CGD
The goal of this phase III study is to investigate the safety and efficacy of a novel approach to allogeneic stem cell transplantation aimed at decreasing transplant related morbidity and mortality We will treat patients with CGD with an allogeneic G-CSF mobilized peripheral blood stem cell transplant from an HLA identical family member The graft will be T-cell depleted which will decrease the incidence of acute graft vs host disease Donor T-cells will be infused post-transplant if engraftment of donor stem cells is unsatisfactory The preparative regimen utilized will provide intense immunosuppression without myeloablation It is designed to allow donor stem cell engraftment while minimizing serious transplant related toxicity
The end points of this study are engraftment degree of donor-host chimerism incidence of acute and chronic GVHD transplant related morbidity and mortality as well as overall survival
The goal of this phase III study is to investigate the safety and efficacy of a novel approach to allogeneic stem cell transplantation aimed at decreasing transplant related morbidity and mortality We will treat patients with CGD with an allogeneic G-CSF mobilized peripheral blood stem cell transplant from an HLA identical family member The graft will be T-cell depleted which will decrease the incidence of acute graft vs host disease Donor T-cells will be infused post-transplant if engraftment of donor stem cells is unsatisfactory The preparative regimen utilized will provide intense immunosuppression without myeloablation It is designed to allow donor stem cell engraftment while minimizing serious transplant related toxicity
The end points of this study are engraftment degree of donor-host chimerism incidence of acute and chronic GVHD transplant related morbidity and mortality as well as overall survival
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 |
|---|---|---|---|
| 98-I-0104 | None | None | None |