Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Completed · Phase 1

Conditions studied: Hurler Syndrome

In brief

This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.

Key facts

Study ID

NCT01173016

Run by

Masonic Cancer Center, University of Minnesota

People needed

11

Starts

2012-05-29

Expected to finish

2016-03-04

Last updated by the study team

2020-03-20

Who can join

Age: any, up to 14. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously
• Age <14 years old
• >10% engrafted based on recent testing (<4 months prior to enrollment)
• Willing to commit to traveling to the University of Minnesota every 6 months
• Written informed consent prior to the performance of any study related procedures

You may not qualify if…

• Previous administration of Laronidase enzyme > 3 months post transplantation
• Anticipated survival less than 2 years
• History of cardiac or pulmonary insufficiency, including an ejection fraction (EF) < 40% or those requiring continuous supplemental oxygen

Where it is running

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

Full record on ClinicalTrials.gov

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