Intrathecal Enzyme Replacement for Hurler Syndrome

Hurler Syndrome Clinical Trial

Official title:

Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00638547
• Other study ID #: MT2007-10
• Secondary ID: 0707M11762
• Status: Completed
• Phase: Phase 1
• Start date: January 2, 2008
• Est. completion date: November 18, 2018

Study information

• Verified date: November 2018
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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").

Recruitment information / eligibility

• Status: Completed
• Enrollment: 26
• Est. completion date: November 18, 2018
• Est. primary completion date: February 18, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 3 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hurler Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Syndrome

Intervention

Drug:
• IRT Laronidase
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

Locations

Country	Name	City	State
United States	University of Minnesota, Fairview	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

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