Mucopolysaccharidosis I (MPS I) Registry
a study on Mucopolysaccharidosis Mucopolysaccharidosis Type I
Summary
- Location
- at UC Davis UCSD UCSF
- Dates
- study startedcompletion 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