Ignite Creation Date:
2024-05-05 @ 5:34 PM
Last Modification Date:
2024-10-26 @ 9:33 AM
Study NCT ID:
NCT00486889
Status:
COMPLETED
Last Update Posted:
2022-08-26
First Post:
2007-06-13
Brief Title:
Growth and Development Study of Alglucosidase Alfa
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa
Status:
COMPLETED
Status Verified Date:
2022-07
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:
Pompe disease also known as glycogen storage disease Type II is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase GAA an enzyme that degrades lysosomal glycogen As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders Pompe disease is characterized by organelle bound lysosomal and extra-lysosomal accumulation of glycogen in many body tissues ultimately leading to multisystemic pathology The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age Participants were to be followed for a 10-year period
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
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 |
|---|---|---|---|
| 2021-005552-11 | EUDRACT_NUMBER | UTN | None |
| LTS12869 | OTHER | None | None |
| U1111-1163-0368 | OTHER | None | None |