Hunter Syndrome Clinical Trial
Official title:
Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
Verified date | September 2017 |
Source | Masonic Cancer Center, University of Minnesota |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 46 |
Est. completion date | June 2017 |
Est. primary completion date | June 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 21 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United States | Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota |
Lead Sponsor | Collaborator |
---|---|
Masonic Cancer Center, University of Minnesota |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Patients With Donor Derived Engraftment | Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells. | Day 100 Post Transplant | |
Secondary | Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
Day 100 Post Transplant | |
Secondary | Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
Day 100 Post Transplant | |
Secondary | Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
Day 100 Post Transplant | |
Secondary | Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
Day 100 Post Transplant | |
Secondary | Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) | GVHD grading is performed using modified Glucksberg criteria and is as follows: grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4 |
Day 100 Post Transplant | |
Secondary | Number of Patients Who Died Peri-Transplant | Peri-transplant is defined as within 100 days of transplant. | By Day 100 Post Transplant | |
Secondary | Donor Cell Chimerism Following Transplant | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. | Day 28 | |
Secondary | Donor Cell Chimerism Following Transplant | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. | Day 42 | |
Secondary | Donor Cell Chimerism Following Transplant | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. | Day 100 | |
Secondary | Donor Cell Chimerism Following Transplant | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. | 6 months | |
Secondary | Donor Cell Chimerism Following Transplant | Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. | One year |
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