Ignite Creation Date:
2025-12-24 @ 9:54 PM
Ignite Modification Date:
2026-01-02 @ 12:44 AM
Study NCT ID:
NCT04877132
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2022-09-19
First Post:
2021-05-03
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
Sponsor:
Organization:
Study Design
Study Type:
EXPANDED_ACCESS
Expanded Access Type Individual:
None
Expanded Access Type Intermediate:
True
Expanded Access Type Treatment:
None
Patient Registry:
None
Target Duration:
None
Design Allocation:
Design Intervention Model:
Design Intervention Model Description:
Design Masking Description:
Bio Spec Retention:
None
Bio Spec Description:
None
Enrollment Count:
Enrollment Type:
Design Primary Purpose:
Design Masking:
Phases:
Observational Models:
Time Perspective List:
Who Masked List: