Ignite Creation Date:
2024-05-06 @ 7:57 PM
Last Modification Date:
2024-10-26 @ 3:17 PM
Study NCT ID:
NCT06192576
Status:
RECRUITING
Last Update Posted:
2024-06-17
First Post:
2023-12-20
Brief Title:
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency ASMD
Sponsor:
Sanofi
Organization:
Sanofi
Study Overview
Official Title:
A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency
Status:
RECRUITING
Status Verified Date:
2024-06
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:
US multicenter cohort open label observational study with primary data collection Ancillary protocol-specified procedures to address the study objectives eg assessment of ADA may be considered outside the standard of care for acid sphingomyelinase deficiency ASMD but the study methodology remains non-interventional as the additional collection of data from participants will not dictate treatment The total overall study duration will be 5 years The follow-up period will be a minimum of 1 year to a maximum of 3 years The enrollment period will be up to 4 years to allow a minimum of 1 year of follow-up for the last participant enrolled
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 |
|---|---|---|---|
| U1111-1294-8169 | REGISTRY | ICTRP | None |