Trial record 5 of 38 for:    Open Studies | "Mucopolysaccharidoses"

Mucopolysaccharidosis I (MPS I) Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00144794
First received: September 2, 2005
Last updated: August 26, 2014
Last verified: August 2014
  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 Sanofi:

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00144794     History of Changes
Other Study ID Numbers: MPS I Registry, DIREGC07008
Study First Received: September 2, 2005
Last Updated: August 26, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Sanofi:
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis I
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 September 18, 2014