Mucopolysaccharidosis I (MPS I) Registry

a study on Mucopolysaccharidosis Mucopolysaccharidosis Type I

Summary

Location
at UC Davis UCSD UCSF
Dates
study started
completion around

Description

Summary

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

Details

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 - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com
  • 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

Keywords

Mucopolysaccharidosis I (MPS I), Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome, Mucopolysaccharidoses, Mucopolysaccharidosis I

Eligibility

You can join if…

  • 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

You CAN'T join if...

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

Locations

  • Children's Hospital Oakland- Site Number : 840029 completed
    Oakland California 94609 United States
  • UC Davis MIND Institute- Site Number : 840010 accepting new patients
    Sacramento California 95817 United States
  • University of California at San Diego- Site Number : 840007 completed
    San Diego California 92093 United States
  • University of California at San Francisco- Site Number : 840051 accepting new patients
    San Francisco California United States
  • Southern California Permanente Medical Group- Site Number : 840108 accepting new patients
    Los Angeles California 90027 United States
  • Children's Hospital of Orange County- Site Number : 840074 accepting new patients
    Orange California 92868 United States
  • USC Health Sciences Center Dept of Genetics- Site Number : 840082 accepting new patients
    Los Angeles California 90033 United States
  • Stanford Unviersity MC Dept of Genetics- Site Number : 840022 accepting new patients
    Palo Alto California 94305 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Genzyme, a Sanofi Company
ID
NCT00144794
Study Type
Observational
Participants
Expecting 1500 study participants
Last Updated