Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
| Status: | Completed |
|---|---|
| Conditions: | Endocrine, Hematology, Metabolic |
| Therapuetic Areas: | Endocrinology, Hematology, Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | Any - 21 |
| Updated: | 2/7/2018 |
| Start Date: | December 2009 |
| End Date: | June 2017 |
Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to
stop the patient's immune system from rejecting the donor's stem cells. When healthy stem
cells from a donor are infused into the patient, the donor white blood cells can provide the
missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a
donor can make an immune response against the body's normal cells. Giving a monoclonal
antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before
and after transplant may stop this from happening. This may be an effective treatment for
inherited metabolic disorders.
Purpose: The design of this study is to achieve donor cell engraftment in patients with
standard-risk inherited metabolic diseases with limited peri-transplant morbidity and
mortality. This will be achieved through the administration of the chemotherapy regimen
described. The intention is to follow transplanted patient for years after transplant
monitoring them for complications of their disease and assisting families with a multifaceted
interdisciplinary approach.
stop the patient's immune system from rejecting the donor's stem cells. When healthy stem
cells from a donor are infused into the patient, the donor white blood cells can provide the
missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a
donor can make an immune response against the body's normal cells. Giving a monoclonal
antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before
and after transplant may stop this from happening. This may be an effective treatment for
inherited metabolic disorders.
Purpose: The design of this study is to achieve donor cell engraftment in patients with
standard-risk inherited metabolic diseases with limited peri-transplant morbidity and
mortality. This will be achieved through the administration of the chemotherapy regimen
described. The intention is to follow transplanted patient for years after transplant
monitoring them for complications of their disease and assisting families with a multifaceted
interdisciplinary approach.
Primary Objective:
- To estimate the proportion of patients with donor derived engraftment at day 100 post
transplant as defined by 80% or greater donor cells in the CD3 (T cell) fraction
Secondary Objectives:
- To determine the incidence and severity of graft-versus-host disease (GVHD) by day 100
- To determine the incidence of peri-transplant mortality (death by day 100)
- To monitor donor cell chimerism at various time points following allogeneic
transplantation with this transplant regimen as determined at day 28, 42, 100, 6 months
and yearly for 5 years.
- To estimate the proportion of patients with donor derived engraftment at day 100 post
transplant as defined by 80% or greater donor cells in the CD3 (T cell) fraction
Secondary Objectives:
- To determine the incidence and severity of graft-versus-host disease (GVHD) by day 100
- To determine the incidence of peri-transplant mortality (death by day 100)
- To monitor donor cell chimerism at various time points following allogeneic
transplantation with this transplant regimen as determined at day 28, 42, 100, 6 months
and yearly for 5 years.
Inclusion Criteria:
- Must have diagnosis of one of the following: mucopolysaccharidosis disorder,
glycoprotein metabolic disorder, sphingolipidoses or inherited leukodystrophy,
peroxisomal disorder or other inherited diseases of metabolism
- Must have an acceptable graft source as defined by University of Minnesota criteria
- Adequate organ function
Exclusion Criteria:
- Pregnant - menstruating females must have a negative serum pregnancy test within 14
days of treatment start
- Evidence of human immunodeficiency virus (HIV) infection or known HIV positive
serology
We found this trial at
1
site
425 E River Pkwy # 754
Minneapolis, Minnesota 55455
Minneapolis, Minnesota 55455
612-624-2620
Masonic Cancer Center at University of Minnesota The Masonic Cancer Center was founded in 1991....
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