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


Phase 2
N/A
7 Years
Open (Enrolling)
Both
Mucopolysaccharidosis I, Hurler Syndrome

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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 MinnesotaMinneapolis, Minnesota  55455