A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Status:
Active, not recruiting
Trial end date:
2024-02-29
Target enrollment:
Participant gender:
Summary
The primary objective of this study is to obtain data regarding the safety of olipudase alfa
in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term
treatment with olipudase alfa.
The secondary objectives of this study are to obtain data regarding the efficacy of olipudase
alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK)
following long-term administration.