MPS I Registry: Mucopolysaccharidosis I (MPS I) Registry

This study is currently recruiting patients.

Sponsored By:	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:

Condition

Mucopolysaccharidosis I

Study Type: Observational

Study Design: Natural History, Longitudinal, Defined Population, Retrospective/Prospective Study

Further Study Details:
Study start: October 2003

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

Eligibility Criteria

Ages Eligible for Study: Both

Inclusion Criteria:

All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed diagnosis is defined as:

documented biochemical evidence of a deficiency in alpha-(a)-L-iduronidase enzyme activity and/or 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.

Location Information

Please refer to this study by ClinicalTrials.gov identifier NCT00144794
Central Contact:
MPS I Registry HelpLine 800-745-4447, Ext. x15500 help@MPSIRegistry.com
MPS I Registry HelpLine 617-591-5500 help@MPSIRegistry.com

Registry participation is worldwide and not limited by facility; facilities not yet active may enroll upon identification of a patient.

More Information

Study ID Numbers: MPS I Registry
ClinicalTrials.gov Identifier: NCT00144794
Health Authority: United States: Institutional Review Board