Ignite Creation Date:
2024-05-05 @ 11:25 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00031005
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2002-02-20
Brief Title:
Stem Cell Transplantation to Treat Leukocyte Adhesion Deficiency
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Treatment of Leukocyte Adhesion Deficiency With Allogeneic Stem Cell Transplantation
Status:
COMPLETED
Status Verified Date:
2003-02
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 leukocyte adhesion deficiency LAD LAD is an inherited blood disorder of leukocytes infection-fighting white blood cells that leaves patients vulnerable to life-threatening infections Transplantation of donated stem cells cells produced by the bone marrow that mature into blood cells can improve the immune system of patients with LAD 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 T-cells a type of 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 the donors T-cells will be removed from the rest of the stem cells to be transplanted
Patients with LAD who weigh at least 12 kg 264 LB 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 Pregnant or breast feeding women may not participate Candidates will have a medical history physical examination and blood tests lung and heart function tests X-rays or computed tomography CT scans of the body and dental and eye examinations They will fill out questionnaires that measure emotional well being quality of life and intelligence the ability to learn and understand
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 move stem cells from the bone marrow to the bloodstream The stem cells will be collected by apheresis where blood is drawn through a needle placed in one arm and pumped into a machine separating and removing the required cells The rest of the blood is then returned through a needle in the other arm
Before the transplant a central venous line large plastic tube is placed into a major vein This tube can stay in the body and be used during the entire treatment period to deliver the donated stem cells give medications transfuse blood if needed and withdraw blood samples Several days before the transplant procedure patients will begin a conditioning regimen of low-dose chemotherapy with cyclophosphamide fludarabine and Campath 1H When the conditioning therapy is completed the stem cells will be infused To help prevent rejection of donor cells cyclosporine will be given by mouth or by vein starting 1 month after the transplant procedure
The average hospital stay for stem cell transplantation is 21 days After discharge patients will return for follow-up clinic visits weekly or twice weekly for 2 to 3 months These visits will include a symptom check physical examination and blood tests Subsequent visits will be scheduled at 4 6 9 and 12 months after the transplant or more often if required and then yearly
Patients with LAD who weigh at least 12 kg 264 LB 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 Pregnant or breast feeding women may not participate Candidates will have a medical history physical examination and blood tests lung and heart function tests X-rays or computed tomography CT scans of the body and dental and eye examinations They will fill out questionnaires that measure emotional well being quality of life and intelligence the ability to learn and understand
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 move stem cells from the bone marrow to the bloodstream The stem cells will be collected by apheresis where blood is drawn through a needle placed in one arm and pumped into a machine separating and removing the required cells The rest of the blood is then returned through a needle in the other arm
Before the transplant a central venous line large plastic tube is placed into a major vein This tube can stay in the body and be used during the entire treatment period to deliver the donated stem cells give medications transfuse blood if needed and withdraw blood samples Several days before the transplant procedure patients will begin a conditioning regimen of low-dose chemotherapy with cyclophosphamide fludarabine and Campath 1H When the conditioning therapy is completed the stem cells will be infused To help prevent rejection of donor cells cyclosporine will be given by mouth or by vein starting 1 month after the transplant procedure
The average hospital stay for stem cell transplantation is 21 days After discharge patients will return for follow-up clinic visits weekly or twice weekly for 2 to 3 months These visits will include a symptom check physical examination and blood tests Subsequent visits will be scheduled at 4 6 9 and 12 months after the transplant or more often if required and then yearly
Detailed Description:
Leukocyte Adhesion Deficiency LAD is an inherited disorder of leukocyte function Patients are profoundly immunocompromised and plagued early in life with recurrent and often life threatening infections Allogeneic stem cell transplantation significantly improves immune function in patients LAD however severe toxicities are associated with conventional approaches to treatment The primary objective of this phase II study is to investigate efficacy of a novel approach to allogeneic stem cell transplantation that is designed to promote partial or complete donor stem cell engraftment hematopoietic chimerism with reduced transplant morbidity and mortality In an attempt to reduce toxicity from pre-transplant bone marrow conditioning a highly immunosuppressive low intensity bone marrow conditioning regimen will be used Patients will be transplanted with peripheral blood stem cells from an HLA identical family member T-lymphocytes will be removed from the stem cell graft in an attempt to decrease the risk of graft vs host disease Donor T-cells will be infused at various time points following the transplant to augment donor hematopoietic chimerism and aid in immune reconstitution The primary end points of this study are the establishment of donor hematopoietic chimerism acute and chronic graft versus host disease and transplant related mortality
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 |
|---|---|---|---|
| 02-I-0126 | None | None | None |