Ignite Creation Date:
2025-12-25 @ 12:21 AM
Ignite Modification Date:
2025-12-25 @ 10:24 PM
Study NCT ID:
NCT06949358
Status:
COMPLETED
Last Update Posted:
2025-04-29
First Post:
2025-04-22
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
Sponsor:
Sanofi
Organization:
Study Overview
Official Title:
A Long-Term Follow-up Study to Evaluate Safety and Tolerability of Olipudase Alfa in Patients Who Completed the DFI12712 or the LTS13632 Study in France
Status:
COMPLETED
Status Verified Date:
2025-04
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:
This is an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement is granted in France.
Study and treatment duration:
The period between the patient's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement is available in France.
In case reimbursement will not be obtained, this study will end 5 years after starting.
Visit frequency: every 2 weeks.
Study and treatment duration:
The period between the patient's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement is available in France.
In case reimbursement will not be obtained, this study will end 5 years after starting.
Visit frequency: every 2 weeks.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
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?:
True
Is an FDA AA801 Violation?: