Ignite Creation Date:
2024-05-05 @ 12:04 PM
Last Modification Date:
2024-10-26 @ 9:19 AM
Study NCT ID:
NCT00231400
Status:
RECRUITING
Last Update Posted:
2024-02-01
First Post:
2005-09-30
Brief Title:
Pompe Disease Registry Protocol
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Pompe Disease Registry
Status:
RECRUITING
Status Verified Date:
2024-01
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 Pompe Registry is a global multicenter international longitudinal observational and voluntary program for patients with Pompe disease designed to track the diseases natural history and outcomes in patients both treated and not Data from the Registry are also used to fulfill various global regulatory commitments to support product developmentreimbursement and for other research and non-research related purposes
The objectives of the Registry are
To enhance understanding of the variability progression identification and natural history of Pompe disease with the ultimate goal of better guiding and assessing therapeutic intervention
To assist the Pompe medical community with the development of recommendations for monitoring patients and to provide reports on patient outcomes to optimize patient care
To characterize the Pompe disease population
To evaluate the long-term effectiveness of alglucosidase alfa
The objectives of the Registry are
To enhance understanding of the variability progression identification and natural history of Pompe disease with the ultimate goal of better guiding and assessing therapeutic intervention
To assist the Pompe medical community with the development of recommendations for monitoring patients and to provide reports on patient outcomes to optimize patient care
To characterize the Pompe disease population
To evaluate the long-term effectiveness of alglucosidase alfa
Detailed Description:
Study Design Time Perspective Retrospective and Prospective
Study Oversight
Has Oversight DMC:
None
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?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| DIREGC07005 | OTHER | Sanofi Identifier | None |