Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome
The primary objective of this pilot study is to determine the feasibility of giving weekly
Laronidase for 2 years in patients with Hurler syndrome after allogeneic transplantation.
Specifically, i) the ability to enroll patients, ii) continued compliance throughout the
study with drug administration and testing, as well as iii) the relevance of various
endpoint determinations will be assessed. The findings of the pilot study will be used to
assess whether a subsequent larger study will be conducted.
Secondary Objectives: The secondary objectives of this study will focus on the toxicity
associated with weekly Laronidase in this patient population, and the evaluation of a
variety of testing and efficacy parameters that would be utilized to measure outcomes and
determine benefit in patients treated on a subsequent larger study.
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.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Adverse events that occur after administration with Laronidase.
Prior to Starting Enzyme Throughout 2 Years on Therapy
Paul Orchard, MD
Masonic Cancer Center, University of Minnesota
United States: Food and Drug Administration
|Masonic Cancer Center, University of Minnesota||Minneapolis, Minnesota 55455|