Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
|ClinicalTrials.gov Identifier: NCT01572636|
Recruitment Status : Active, not recruiting
First Posted : April 6, 2012
Last Update Posted : December 5, 2017
|Condition or disease||Intervention/treatment|
|Mucopolysaccharidosis Type IH MPS I Hurler Syndrome||Drug: Laronidase|
|Study Type :||Observational|
|Actual Enrollment :||20 participants|
|Official Title:||MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).|
|Actual Study Start Date :||March 28, 2012|
|Estimated Primary Completion Date :||July 17, 2018|
|Estimated Study Completion Date :||July 17, 2018|
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
- Overall Survival [ Time Frame: At 1 Year ]Patients alive at 1 year post transplantation.
- Incidence of Engraftment [ Time Frame: 1 Year Post Transplant ]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 ]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 ]Count of patients using ventilator by 1 year.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01572636
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota|
|Minneapolis, Minnesota, United States, 55455|
|Principal Investigator:||Paul Orchard, M.D.||Masonic Cancer Center, University of Minnesota|