Mucopolysaccharidosis I (MPS I) Registry

Study Description

Brief Summary

Detailed Description

Condition or disease	Intervention/treatment	Phase
Mucopolysaccharidosis I (MPS I)		N/A

Tracking Information

First Submitted Date	September 02, 2005
Last Update Posted Date	February 22, 2018
Start Date	November 20, 2003
Anticipated Completion Date	December 31, 2020
Primary Completion Date	December 31, 2020
Results First Submitted Date	N/A
Received Results Disposit Date	N/A

Current Primary Outcome Measures

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

Original Primary Outcome Measures

Current Secondary Outcome Measures

Original Secondary Outcome Measures

Study Design

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, [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 Type	Observational
Study Phase	N/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 Method	Probability Sample
Study Population	All Patients with MPS I
Intervention	Not Available
Study Groups/Cohorts	Not Available
Study Arms	Not Available

Arm	Intervention/Treatment

Recruitment Information

Recruitment Status:	Recruiting
Enrollment	1500
Completion Date	December 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.
Gender	All
Age	N/A to N/A
Accepts Healthy Volunteers	No
Contacts	For site information, send an email with site number to [email protected] MPS I Registry HelpLine 617-591-5500 [email protected]
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 Committee	No
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