Mucopolysaccharidosis I (MPS I) Registry

Completed

Conditions studied: Mucopolysaccharidosis I (MPS I)

In brief

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

Key facts

Study ID

NCT00144794

Run by

Genzyme, a Sanofi Company

People needed

1406

Starts

2003-11-20

Expected to finish

2024-12-31

Last updated by the study team

2026-02-27

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify 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 may not qualify if…

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

Where it is running

• Arkansas Children's Hospital- Site Number : 840109 — Little Rock, Arkansas, United States
• Southern California Permanente Medical Group- Site Number : 840108 — Los Angeles, California, United States
• USC Health Sciences Center Dept of Genetics- Site Number : 840082 — Los Angeles, California, United States
• UCSF Benioff Children's Hospital - Oakland- Site Number : 840029 — Oakland, California, United States
• Children's Hospital of Orange County- Site Number : 840074 — Orange, California, United States
• Stanford Unviersity MC Dept of Genetics- Site Number : 840022 — Palo Alto, California, United States
• UC Davis MIND Institute- Site Number : 840010 — Sacramento, California, United States
• University of California at San Diego- Site Number : 840007 — San Diego, California, United States
• University of California at San Francisco- Site Number : 840051 — San Francisco, California, United States
• Children's Hospital Colorado - Aurora- Site Number : 840069 — Aurora, Colorado, United States
• University of Colorado at Denver Genetics Site Number : 840068 — Aurora, Colorado, United States
• Yale University School of Medicine- Site Number : 840047 — New Haven, Connecticut, United States
• Children's National Medical Center - Washington- Site Number : 840067 — Washington D.C., District of Columbia, United States
• University of Florida Dept of Genetics- Site Number : 840083 — Gainesville, Florida, United States
• University Of Miami SOM- Site Number : 840006 — Miami, Florida, United States
• Pediatric Endocrine Associates- Site Number : 840017 — Tampa, Florida, United States
• Emory University School of Medicine- Human Genetics- Site Number : 840060 — Decatur, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011 — Chicago, Illinois, United States
• Rush University Medical Center Genetics- Site Number : 840079 — Chicago, Illinois, United States
• Indianapolis University School of Medicine- Site Number : 840027 — Indianapolis, Indiana, United States
• University of Iowa- Site Number : 840032 — Iowa City, Iowa, United States
• University of Louisville- Genetics- Site Number : 840030 — Louisville, Kentucky, United States
• John Hopkins- Site Number : 840044 — Baltimore, Maryland, United States
• Massachusetts General Hospital-Genetics- Site Number : 840062 — Boston, Massachusetts, United States
• Phoenix Children's Hospital- Site Number : 840003 — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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