Stem Cell Transplant w/Laronidase for Hurler

NCT ID: NCT00176891

Last Updated: 2019-07-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-03-31
• Study Completion Date: 2012-11-30

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 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.

Conditions

Mucopolysaccharidosis I; Hurler Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Laronidase ERT Treatment

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

Group Type: EXPERIMENTAL

Stem Cell Transplant

Intervention Type: PROCEDURE

enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following

Laronidase ERT

Intervention Type: DRUG

Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.

Interventions

Stem Cell Transplant

enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following

Intervention Type: PROCEDURE

Laronidase ERT

Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.

Intervention Type: DRUG

Other Intervention Names

ERT; Aldurazyme®

Eligibility Criteria

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.

• Maximum Eligible Age: 7 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Paul Orchard, MD

Role: PRINCIPAL_INVESTIGATOR

Masonic Cancer Center, University of Minnesota

Locations

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, United States

Countries

United States

Other Identifiers

0403M57728

Identifier Type: OTHER

Identifier Source: secondary_id

MT2004-09

Identifier Type: -

Identifier Source: org_study_id