Mucopolysaccharidosis I (MPS I) Registry

Description

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

Conditions

Mucopolysaccharidosis I (MPS I)

Study Overview

Study Details

Study overview

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

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I) Registry

Condition
Mucopolysaccharidosis I (MPS I)
Intervention / Treatment

-

Contacts and Locations

Phoenix

Phoenix Children's Hospital- Site Number : 840003, Phoenix, Arizona, United States, 85013

Little Rock

Arkansas Childrens Hospital- Site Number : 840109, Little Rock, Arkansas, United States, 72202

Los Angeles

Southern California Permanente Medical Group- Site Number : 840108, Los Angeles, California, United States, 90027

Los Angeles

USC Health Sciences Center Dept of Genetics- Site Number : 840082, Los Angeles, California, United States, 90033

Oakland

Children's Hospital Oakland- Site Number : 840029, Oakland, California, United States, 94609

Orange

Children's Hospital of Orange County- Site Number : 840074, Orange, California, United States, 92868

Palo Alto

Stanford Unviersity MC Dept of Genetics- Site Number : 840022, Palo Alto, California, United States, 94305

Sacramento

UC Davis MIND Institute- Site Number : 840010, Sacramento, California, United States, 95817

San Bernardino

Loma Linda University Children's Hospital- Site Number : 840117, San Bernardino, California, United States, 92408

San Diego

University of California at San Diego- Site Number : 840007, San Diego, California, United States, 92093

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

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

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Genzyme, a Sanofi Company,

Medical Monitor, STUDY_DIRECTOR, Genzyme, a Sanofi Company

Study Record Dates

2034-01-31