Mucopolysaccharidosis I (MPS I) Registry - Full Text View

Sponsors and Collaborators:	Genzyme BioMarin/Genzyme LLC
Information provided by:	Genzyme
ClinicalTrials.gov Identifier:	NCT00144794

Purpose

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.

The objectives of the Registry are:

To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase)
To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I
To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Condition
Mucopolysaccharidosis I

Genetics Home Reference related topics: mucopolysaccharidosis type I

U.S. FDA Resources

Study Type:

Observational

Further study details as provided by Genzyme:

Study Start Date:

October 2003

Detailed Description:

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

In Asia-Pacific - Margie Jimenez, +852 2810 1613
In Europe - +31-35-699-1232, europe@MPSIregistry.com
In Latin America - +617-591-5500, help@MPSIregistry.com
In North America - +617-591-5500, help@MPSIregistry.com

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha (a)-L-iduronidase enzyme activity and/or B. mutation(s) in the gene coding for a-L-iduronidase, or measurable clinical signs and symptoms of MPS I
For all patients there should be a completed patient authorization form

Exclusion Criteria:

No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00144794

Contacts

Contact: MPS I Registry HelpLine	800-745-4447 ext x15500	help@MPSIRegistry.com
Contact: MPS I Registry HelpLine	617-591-5500	help@MPSIRegistry.com

Locations

United States, Massachusetts
Registry participation is worldwide and not limited to this facility; facilities not yet active may enroll upon identification of a patient.	Recruiting
Cambridge, Massachusetts, United States, 02142

Sponsors and Collaborators

Genzyme

BioMarin/Genzyme LLC

Investigators

Study Director:

Gerald F Cox, M.D. , PhD

Genzyme

More Information

Study ID Numbers:	MPS I Registry
Study First Received:	September 2, 2005
Last Updated:	July 29, 2008
ClinicalTrials.gov Identifier:	NCT00144794
Health Authority:	United States: Institutional Review Board

Keywords provided by Genzyme:

Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome

Study placed in the following topic categories:

Alpha-L-iduronidase deficiency
Metabolism, Inborn Errors
Mucopolysaccharidoses
Metabolic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases

Connective Tissue Diseases
Mucopolysaccharidosis
Metabolic disorder
Hurler syndrome
Mucopolysaccharidosis I

Additional relevant MeSH terms:

Mucinoses
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 16, 2009