Clinical Trial: Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Overall Survival [ Time Frame: At 1 Year ]

Patients alive at 1 year post transplantation.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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.


Original Secondary Outcome: Same as current

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: April 4, 2012
Date Started: April 2012
Date Completion: April 2022
Last Updated: August 15, 2016
Last Verified: August 2016