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Mucopolysaccharidosis I (MPS I) Registry

This study is currently recruiting participants.
Verified September 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
ClinicalTrials.gov Identifier:
NCT00144794
First Posted: September 5, 2005
Last Update Posted: September 29, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
  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
Time Perspective: Other
Official Title: Mucopolysaccharidosis I (MPS I) Registry

Resource links provided by NLM:


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

Primary Outcome Measures:
  • To evaluate the long-term effectiveness of Aldurazyme [ Time Frame: Approximately 17 Years ]

Estimated Enrollment: 1500
Study Start Date: November 20, 2003
Estimated Study Completion Date: September 30, 2022
Estimated Primary Completion Date: September 30, 2022 (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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00144794


Contacts
Contact: For site information, send an email with site number to Contact-Us@sanofi.com
Contact: MPS I Registry HelpLine 617-591-5500 help@MPSIRegistry.com

  Show 75 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00144794     History of Changes
Other Study ID Numbers: MPS I Registry
DIREGC07008 ( Other Identifier: Sanofi )
First Submitted: September 2, 2005
First Posted: September 5, 2005
Last Update Posted: September 29, 2017
Last Verified: September 2017

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
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