Stem Cell Transplant w/Laronidase for Hurler

University of Minnesota (UMN)

Status and phase: Completed; Phase 2

Conditions: Mucopolysaccharidosis I; Hurler Syndrome

Treatments: Procedure: Stem Cell Transplant; Drug: Laronidase ERT

Study type: Interventional

Funder types: Other

Identifiers

NCT00176891

0403M57728 (Other Identifier)

MT2004-09

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 25 patients

Sex: All

Ages: Under 7 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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25 participants in 1 patient group

Laronidase ERT Treatment

Experimental group

Description:

Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.

Treatment:

Drug: Laronidase ERT

Procedure: Stem Cell Transplant