Ignite Creation Date:
2024-05-06 @ 3:33 PM
Last Modification Date:
2024-10-26 @ 1:52 PM
Study NCT ID:
NCT04676373
Status:
COMPLETED
Last Update Posted:
2024-03-08
First Post:
2020-12-15
Brief Title:
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Single Arm Prospective Open-label Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment
Status:
COMPLETED
Status Verified Date:
2024-08
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:
APOLLO-LOPD
Brief Summary:
Primary Objective
To evaluate the effect of one-year Alglucosidase alfa treatment on motor function Six-minute walk test 6MWT and lung function predicted Forced vital capacity FVC among Chinese Late Onset Pompe Disease patients above 5 years old
To evaluate the safety of Myozyme 20mgkg IV biweekly in Chinese LOPD patients above 3 years old
Secondary Objective
To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test MMT Maximal inspiratory and expiratory pressure MIP and MEP Quick Motor Function Test scores and health-related quality of life SF-12 among LOPD patients over 5 years old
To evaluate the effect of one-year Alglucosidase alfa treatment on motor function Six-minute walk test 6MWT and lung function predicted Forced vital capacity FVC among Chinese Late Onset Pompe Disease patients above 5 years old
To evaluate the safety of Myozyme 20mgkg IV biweekly in Chinese LOPD patients above 3 years old
Secondary Objective
To evaluate the effect of one-year treatment with Alglucosidase alfa on improvement of manual muscle test MMT Maximal inspiratory and expiratory pressure MIP and MEP Quick Motor Function Test scores and health-related quality of life SF-12 among LOPD patients over 5 years old
Detailed Description:
Study duration per participants is approximatively 56 weeks including a 52-week treatment period
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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 |
|---|---|---|---|
| LPS15677 | OTHER | Sanofi Identifier | None |
| U1111-1238-1267 | OTHER | None | None |