Mucopolysaccharidosis I (MPS I) Registry

ID: NCT00144794
Status: Recruiting
Phase: N/A
Start Date: November 20, 2003
First Submitted: September 02, 2005
Last Updated: February 22, 2018
Results: N/A
Organization: Sanofi
Sponsors & Collaborators: Genzyme, a Sanofi Company
Location: Argentina, Australia, Canada, Chile, Colombia, Czechia, Denmark, France, Hong Kong, Italy, Japan, Korea, Republic of, Malaysia, Philippines, Poland, Portugal, Russian Federation, Singapore, Slovakia, Taiwan, Thailand, United States
Conditions: Mucopolysaccharidosis I (MPS I)
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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

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, [email protected]

- In Europe - +31-35-699-1232, [email protected]

- In Latin America - +617-591-5500, [email protected]

- In North America - +617-591-5500, [email protected]
Condition or disease Intervention/treatment Phase

Mucopolysaccharidosis I (MPS I)

N/A

Tracking Information

First Submitted DateSeptember 02, 2005
Last Update Posted DateFebruary 22, 2018
Start DateNovember 20, 2003
Anticipated Completion DateDecember 31, 2020
Primary Completion DateDecember 31, 2020
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

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

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

Not Available

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitleMucopolysaccharidosis I (MPS I) Registry
Official TitleMucopolysaccharidosis 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, [email protected]

- In Europe - +31-35-699-1232, [email protected]

- In Latin America - +617-591-5500, [email protected]

- In North America - +617-591-5500, [email protected]

Study TypeObservational
Study PhaseN/A
Estimated Enrollment
1500
Allocation
Not Available
Interventional Model
Not Available
Masking
Not Available
Primary Purpose
Not Available
Conditions
Mucopolysaccharidosis I (MPS I)
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodProbability Sample
Study PopulationAll Patients with MPS I
Intervention
Not Available
Study Groups/Cohorts
Not Available
Study Arms
Not Available
Arm Intervention/Treatment

Recruitment Information

Recruitment Status:Recruiting
Enrollment1500
Completion DateDecember 31, 2020
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.
GenderAll
Age N/A to N/A
Accepts Healthy VolunteersNo
Contacts
Listed Location Countries
Argentina
Australia
Canada
Chile
Colombia
Czechia
Denmark
France
Hong Kong
Italy
Japan
Korea, Republic of
Malaysia
Philippines
Poland
Portugal
Russian Federation
Singapore
Slovakia
Taiwan
Thailand
United States
Czech Republic

Administrative Information

NCT Number:NCT00144794
Other Study ID Numbers
MPS I Registry
DIREGC07008
Has Data Monitoring CommitteeNo
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
Genzyme, a Sanofi Company
Collaborators
Not Available
Investigators
Study Director
Medical Monitor
Genzyme, a Sanofi Company