NCT01173016

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 14 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes are assessed semi-annually and compared to historic controls. Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme. The findings of this Pilot Study will be used to assess whether a subsequent larger study can be conducted.

Conditions

• Hurler Syndrome

Interventions

Type	Name	Description
DRUG	Laronidase	Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years

Timeline

• Start date: 2012-05-29
• Primary completion: 2016-03-04
• Completion: 2016-03-04
• First posted: 2010-07-30
• Last updated: 2020-03-20
• Results posted: 2020-03-20

Locations

1 site across 1 country: United States