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Mucopolysaccharidosis I (MPS I) Registry

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ClinicalTrials.gov Identifier: NCT00144794
Recruitment Status : Recruiting
First Posted : September 5, 2005
Last Update Posted : December 12, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief 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

Condition or disease
Mucopolysaccharidosis I (MPS I)

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

Study Design

Study Type : Observational
Estimated Enrollment : 1500 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Mucopolysaccharidosis I (MPS I) Registry
Study Start Date : November 20, 2003
Estimated Primary Completion Date : September 30, 2022
Estimated Study Completion Date : September 30, 2022


Groups and Cohorts


Outcome Measures

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

Eligibility Criteria

Information from the National Library of Medicine

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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 79 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 Posted: September 5, 2005    Key Record Dates
Last Update Posted: December 12, 2017
Last Verified: December 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