Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Stopped early

Conditions studied: Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome

In brief

This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.

Key facts

Study ID

NCT01572636

Run by

Masonic Cancer Center, University of Minnesota

People needed

20

Starts

2012-03-28

Expected to finish

2018-05-01

Last updated by the study team

2018-09-27

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol

You may not qualify if…

• No prior therapy with laronidase enzyme replacement therapy (ERT)

Where it is running

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

Full record on ClinicalTrials.gov

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