Mucopolysaccharidosis I (MPS I) Registry
- Conditions
- Mucopolysaccharidosis I (MPS I)
- Registration Number
- NCT00144794
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief 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
- 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 - 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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 1500
- 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
- No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate the long-term effectiveness of Aldurazyme Approximately 17 Years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (180)
Phoenix Children's Hospital- Site Number : 840003
🇺🇸Phoenix, Arizona, United States
Arkansas Childrens 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
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
Loma Linda University Children's Hospital- Site Number : 840117
🇺🇸San Bernardino, California, United States
University of California at San Diego- Site Number : 840007
🇺🇸San Diego, California, United States
Scroll for more (170 remaining)Phoenix Children's Hospital- Site Number : 840003🇺🇸Phoenix, Arizona, United States