Ignite Creation Date:
2025-12-25 @ 1:43 AM
Ignite Modification Date:
2025-12-26 @ 12:00 AM
Study NCT ID:
NCT00144794
Status:
RECRUITING
Last Update Posted:
2025-10-31
First Post:
2005-09-02
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Mucopolysaccharidosis I (MPS I) Registry
Sponsor:
Genzyme, a Sanofi Company
Organization:
Study Overview
Official Title:
Mucopolysaccharidosis I (MPS I) Registry
Status:
RECRUITING
Status Verified Date:
2025-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
* 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 Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| U1111-1294-8266 | REGISTRY | ICTRP | View |