Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

• Status: Completed
• Trial end date: 2016-03-04
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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