Mucopolysaccharidosis I (MPS I) Registry

This study is currently recruiting participants.
Verified August 2013 by Genzyme, a Sanofi Company
Sponsor:
Information provided by (Responsible Party):
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00144794
First received: September 2, 2005
Last updated: August 5, 2013
Last verified: August 2013
  Purpose

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

Condition
Mucopolysaccharidosis I (MPS I)

Study Type: Observational
Study Design: Observational Model: Cohort
Official Title: Mucopolysaccharidosis I (MPS I) Registry

Resource links provided by NLM:


Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • To evaluate the long-term effectiveness of Aldurazyme [ Time Frame: Approximately 17 Years ] [ Designated as safety issue: No ]

Estimated Enrollment: 1500
Study Start Date: October 2003
Estimated Study Completion Date: January 2020
Estimated Primary Completion Date: January 2020 (Final data collection date for primary outcome measure)
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
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

All Patients with MPS I

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00144794

Contacts
Contact: MPS I Registry HelpLine 800-745-4447 ext x15500 help@MPSIRegistry.com
Contact: MPS I Registry HelpLine 617-591-5500 help@MPSIRegistry.com

Locations
United States, Massachusetts
Registry participation is worldwide and not limited to this facility; facilities not yet active may enroll upon identification of a patient. Recruiting
Cambridge, Massachusetts, United States, 02142
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00144794     History of Changes
Other Study ID Numbers: MPS I Registry
Study First Received: September 2, 2005
Last Updated: August 5, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Genzyme, a Sanofi Company:
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome

Additional relevant MeSH terms:
Mucopolysaccharidosis I
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on April 20, 2014