Overview

This trial is active, not recruiting.

Condition hurler syndrome
Treatment irt laronidase
Phase phase 1
Sponsor Masonic Cancer Center, University of Minnesota
Start date January 2008
End date June 2016
Trial size 25 participants
Trial identifier NCT00638547, 0707M11762, MT2007-10

Summary

This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.

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)
All patients who have received at least one dose of Laronidase.
irt laronidase Aldurazyme
Laronidase belongs to a class of drugs called enzyme replacement therapies or ERT that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingredient in laronidase is a protein that is identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup. Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture

Primary Outcomes

Measure
To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration
time frame: 1 year

Secondary Outcomes

Measure
To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
time frame: 1 year
To determine brain changes with magnetic resonance imaging
time frame: 1 and 2 years
To determine neurocognitive changes present in patients with Hurler syndrome
time frame: 6, 12, and 24 months
To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained
time frame: through 1 year

Eligibility Criteria

Male or female participants from 6 months up to 3 years old.

Inclusion Criteria: - Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines. Exclusion Criteria: - Patients are less than 6 months old, or older than 3 years of age. - There is a history of clinically-severe hypersensitivity to Laronidase. - There is a contraindication for repeated lumbar puncture. - The family is not willing to undergo the necessary procedures and evaluations inherent in the study. - Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.

Additional Information

Official title Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)
Principal investigator Paul Orchard, MD
Description Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap").
Trial information was received from ClinicalTrials.gov and was last updated in January 2016.
Information provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota.