Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
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.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).|
- Overall Survival [ Time Frame: At 1 Year ] [ Designated as safety issue: No ]Patients alive at 1 year post transplantation.
- Incidence of Engraftment [ Time Frame: 1 Year Post Transplant ] [ Designated as safety issue: No ]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.
- Incidence of Grade III-IV Acute Graft Versus Host Disease [ Time Frame: Day 100 ] [ Designated as safety issue: Yes ]Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk.
- Proportion of patients in need of ventilator support [ Time Frame: 1 Year ] [ Designated as safety issue: Yes ]Count of patients using ventilator by 1 year.
|Study Start Date:||April 2012|
|Estimated Study Completion Date:||April 2022|
|Estimated Primary Completion Date:||April 2022 (Final data collection date for primary outcome measure)|
Laronidase use in Hurler Syndrome
Laronidase receiving prior and post transplant
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.
Other Name: Aldurazyme
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01572636
|Contact: Paul Orchard, M.D.||firstname.lastname@example.org|
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Paul Orchard, M.D. 612-626-2313 email@example.com|
|Principal Investigator: Paul Orchard, M.D.|
|Principal Investigator:||Paul Orchard, M.D.||Masonic Cancer Center, University of Minnesota|