Trials Sponsors Conditions Drugs Pricing

Conditions

Hurler Syndrome

Keywords

Hurler Syndromemucopolysaccharidosis type IIduronidase deficiency

Outcome Measures

Primary Outcomes (1)

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

Secondary Outcomes (4)

To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I
1 year
To determine brain changes with magnetic resonance imaging
1 and 2 years
To determine neurocognitive changes present in patients with Hurler syndrome
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
through 1 year

Study Arms (1)

Intent-to-Treat

EXPERIMENTAL

All patients who have received at least one dose of Laronidase.

Drug: IRT Laronidase

Interventions

IRT LaronidaseDrug

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

Also known as: Aldurazyme

Intent-to-Treat

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age6 Months - 3 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.

You may not qualify if:

Sponsors & Collaborators

Masonic Cancer Center, University of Minnesota
Otherlead

Study Sites (1)

University of Minnesota, Fairview

Minneapolis, Minnesota, 55455, United States

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

Paul Orchard, MD
University of Minnesota Medical Center
PRINCIPAL INVESTIGATOR

Enrollment

26 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

March 11, 2008

First Posted

March 19, 2008

Study Start

January 2, 2008

Actual (reported)

Primary Completion

Intrathecal Enzyme Replacement for Hurler Syndrome

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (4)

Study Arms (1)

Intent-to-Treat

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Intervention Hierarchy (Ancestors)

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (4)

Study Arms (1)

Intent-to-Treat

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

Intervention Hierarchy (Ancestors)

Source record

Locations