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Mucopolysaccharidosis I (MPS I) Registry

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.
 
ClinicalTrials.gov Identifier: NCT00144794
Recruitment Status : Recruiting
First Posted : September 5, 2005
Last Update Posted : January 13, 2023
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date September 2, 2005
First Posted Date September 5, 2005
Last Update Posted Date January 13, 2023
Actual Study Start Date November 20, 2003
Estimated Primary Completion Date January 31, 2034   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 15, 2013)		 To evaluate the long-term effectiveness of Aldurazyme [ Time Frame: Approximately 17 Years ]
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Mucopolysaccharidosis I (MPS I) Registry
Official Title Mucopolysaccharidosis I (MPS I) Registry
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
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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population All Patients with MPS I
Condition Mucopolysaccharidosis I (MPS I)
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: February 15, 2013)		 1500
Original Enrollment Not Provided
Estimated Study Completion Date January 31, 2034
Estimated Primary Completion Date January 31, 2034   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
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
Listed Location Countries Argentina,   Australia,   Belgium,   Brazil,   Canada,   Chile,   Colombia,   Croatia,   Czechia,   Denmark,   Egypt,   France,   Germany,   Hungary,   India,   Indonesia,   Ireland,   Italy,   Japan,   Korea, Republic of,   Kuwait,   Lebanon,   Malaysia,   Netherlands,   Pakistan,   Philippines,   Poland,   Portugal,   Romania,   Russian Federation,   Saudi Arabia,   Singapore,   Slovakia,   Sweden,   Taiwan,   Thailand,   United Arab Emirates,   United Kingdom,   United States,   Vietnam
Removed Location Countries Czech Republic,   Hong Kong
 
Administrative Information
NCT Number NCT00144794
Other Study ID Numbers DIREGC07008
DIREGC07008 ( Other Identifier: Sanofi )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Current Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Original Responsible Party Not Provided
Current Study Sponsor Genzyme, a Sanofi Company
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date January 12, 2023