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| ID | Type | Description | Link |
|---|---|---|---|
| MT2011-21C | Other Identifier | Blood and Marrow Transplant Program |
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Research Cancelled
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This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.
Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to reduce pulmonary complications.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Laronidase use in Hurler Syndrome | Laronidase receiving prior and post transplant |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Laronidase | Drug | Administered 0.58 mg/kg/dose intravenously (IV) once a week beginning 12 weeks before planned hematopoietic stem cell transplant (HSCT) and resume same dosing regimen for 8 weeks after HSCT. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival | Patients alive at 1 year post transplantation. | At 1 Year |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Engraftment | The incidence of donor engraftment will be estimated by taking the simple proportion of patients achieving donor engraftment over the number of evaluable patients. Donor engraftment will be defined as achieving an absolute neutrophil count ≥ 5x10^8/kg for three consecutive days before day 42 and maintenance of >10% donor chimerism through one year post transplant or death. |
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Inclusion Criteria:
Exclusion Criteria:
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Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol
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| Name | Affiliation | Role |
|---|---|---|
| Paul Orchard, M.D. | Masonic Cancer Center, University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
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| ID | Term |
|---|---|
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| D007068 | Iduronidase |
| ID | Term |
|---|---|
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
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| 1 Year Post Transplant |
| Incidence of Grade III-IV Acute Graft Versus Host Disease | Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk. | Day 100 |
| Proportion of patients in need of ventilator support | Count of patients using ventilator by 1 year. | 1 Year |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |