Stem Cell Transplant w/Laronidase for Hurler

Completed · Phase 2

Conditions studied: Mucopolysaccharidosis I, Hurler Syndrome

In brief

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.

Key facts

Study ID

NCT00176891

Run by

Masonic Cancer Center, University of Minnesota

People needed

25

Starts

2004-03-01

Expected to finish

2012-11-01

Last updated by the study team

2019-07-11

Who can join

Age: any, up to 7. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Not being considered for University of Minnesota myeloablative HSCT protocol.
• Previous administration of laronidase enzyme
• Second or subsequent HSCT.

Where it is running

• Masonic Cancer Center, University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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