Clinical Trial: Stem Cell Transplant w/Laronidase for Hurler

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)

Brief Summary: The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden that is associated with decreased complications following transplant.

Detailed Summary:

Subjects will receive laronidase once a week intravenously for 10-12 weeks prior to transplant and for approximately 8 weeks after transplant. Laronidase will be given by intravenous infusion (IV) through a catheter and from there to your child's body's cells and organs to break down the glycosaminoglycans (GAG) buildup.

Prior to starting ERT, subjects will have a complete physical examination, which includes a complete assessment of your child's airway and lungs. In addition to standard treatment evaluations and tests, which are done prior to hematopoietic stem cell transplant (HSCT), subjects will have the following tests: an additional teaspoon of blood for a baseline test for serum antibodies against laronidase, before and after the fourth dose of laronidase, the investigators will collect 2 teaspoons of blood for an alpha-L-iduronidase enzyme level; to watch for side effects to laronidase and the development of antibodies to laronidase, approximately 2 teaspoons of blood will be collected every 3 weeks while the subject is receiving laronidase ERT.


Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Survival at one year and the proportion of patients in need of ventilator support by one year [ Time Frame: one year ]

Original Primary Outcome: Survival at one year and the proportion of patients in need of ventilator support by one year

Current Secondary Outcome:

  • Donor engraftment [ Time Frame: Day 100 ]
  • Patients with Grade III-IV acute GVHD [ Time Frame: Day 100 ]
  • Reduction in glycosaminoglycans (GAG) [ Time Frame: Prior to, During and After ERT ]
  • Toxicity (adverse events) associated with infusions of enzyme [ Time Frame: 1 year ]
  • Development of anti-iduronidase antibodies in serum [ Time Frame: 1 Year ]
  • Patients with Improvement in Obstructive Apnea (Breathing) by Polysomnography [ Time Frame: Baseline, 12 weeks after ERT, after transplant ]


Original Secondary Outcome:

  • Donor engraftment over one year post transplant
  • Grade III-IV acute GvHD
  • Disease specific measurements:
  • (reduction in GAG, improvement in polysomnography)
  • Toxicity associated with infusions of enzyme
  • Development of anti-iduronidase antibodies in serum


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: September 12, 2005
Date Started: March 2004
Date Completion:
Last Updated: July 8, 2014
Last Verified: April 2014