Overview

This trial is active, not recruiting.

Condition hurler syndrome
Treatment laronidase
Phase phase 1
Sponsor Masonic Cancer Center, University of Minnesota
Start date May 2012
End date March 2016
Trial size 10 participants
Trial identifier NCT01173016, 1004M80513, 2009LS090, MT2009-20

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Patients with Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant >2 years previously and treated with Laronidase weekly for 2 years after transplant.
laronidase Aldurazyme
Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years

Primary Outcomes

Measure
Toxicities
time frame: Prior to Starting Enzyme Throughout 2 Years on Therapy

Secondary Outcomes

Measure
Change in Orthopedic Measures
time frame: From baseline every 6 months for 2 Years
Change in Markers of Bone Metabolism
time frame: From baseline every 6 months through 2 years
Change in Flexibility and Muscle Strength
time frame: From baseline every 6 months through 2 years
Change in 0xygen Utilization to Monitor "Fitness"
time frame: From baseline every 6 months through 2 years
Change in Neuropsychological Function
time frame: From baseline every 6 months through 2 years
Change in Laboratory Measurements
time frame: From baseline every 3 months through 2 years
Change in Bone Density
time frame: From baseline to 1 and 2 Years

Eligibility Criteria

Male or female participants up to 14 years old.

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

Additional Information

Official title Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome
Principal investigator Paul Orchard, MD
Description 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.
Trial information was received from ClinicalTrials.gov and was last updated in February 2016.
Information provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota.