Trial Radar
Sign in

NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

Mucopolysaccharidosis I (MPS I) Registry
Mucopolysaccharidosis I (MPS I) Registry

NCT00144794

CompletedN/A

Sponsor: Genzyme, a Sanofi Company

Conditions: Mucopolysaccharidosis I (MPS I)

Countries: United States, Argentina, Australia, Belgium, Brazil, Canada, Chile, Colombia

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

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility 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.
Locations (141)
  • Phoenix, Arizona, United States
  • Little Rock, Arkansas, United States
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Oakland, California, United States
  • Orange, California, United States
  • Palo Alto, California, United States
  • Sacramento, California, United States
  • San Diego, California, United States
  • San Francisco, California, United States
  • Aurora, Colorado, United States
  • Aurora, Colorado, United States
  • New Haven, Connecticut, United States
  • Washington D.C., District of Columbia, United States
  • Gainesville, Florida, United States
  • Miami, Florida, United States
  • Tampa, Florida, United States
  • Decatur, Georgia, United States
  • Chicago, Illinois, United States
  • Chicago, Illinois, United States
  • Indianapolis, Indiana, United States
  • Iowa City, Iowa, United States
  • Louisville, Kentucky, United States
  • Baltimore, Maryland, United States
  • Boston, Massachusetts, United States
  • Boston, Massachusetts, United States
  • Springfield, Massachusetts, United States
  • Ann Arbor, Michigan, United States
  • Minneapolis, Minnesota, United States
  • St Louis, Missouri, United States
  • + 111 more on CT.gov