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

Phase 1
14 Years
Open (Enrolling)
Hurler Syndrome

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Trial Information

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.

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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:


Outcome Description:

Adverse events that occur after administration with Laronidase.

Outcome Time Frame:

Prior to Starting Enzyme Throughout 2 Years on Therapy

Safety Issue:


Principal Investigator

Paul Orchard, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota


United States: Food and Drug Administration

Study ID:




Start Date:

May 2012

Completion Date:

March 2015

Related Keywords:

  • Hurler Syndrome
  • mucopolysaccharide IH (MPS IH)
  • Mucopolysaccharidosis I



Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota  55455