Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)
N/A
7 Years
Both
Mucopolysaccharidosis I, Hurler Syndrome
Trial Information
Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)
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.
Inclusion Criteria:
- Patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome)
who are candidates for first hematopoietic stem cell transplant (HSCT) according to a
University of Minnesota myeloablative HSCT protocol.
Exclusion Criteria:
- Not being considered for University of Minnesota myeloablative HSCT protocol.
- Previous administration of laronidase enzyme
- Second or subsequent HSCT.
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Survival at one year and the proportion of patients in need of ventilator support by one year
Outcome Time Frame:
one year
Safety Issue:
No
Principal Investigator
Paul Orchard, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Masonic Cancer Center, University of Minnesota
Authority:
United States: Institutional Review Board
Study ID:
MT2004-09
NCT ID:
NCT00176891
Start Date:
March 2004
Completion Date:
October 2013
Related Keywords:
- Mucopolysaccharidosis I
- Hurler Syndrome
- Laronidase ERT
- Stem cell transplant
- storage disease
- inborn errors of metabolism
- hurler syndrome
- glycosaminoglycans
- enzyme replacement
- Mucopolysaccharidosis I
- Mucopolysaccharidoses
Name | Location |
|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis, Minnesota 55455 |
