Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients
Status:
Completed
Trial end date:
2014-01-01
Target enrollment:
Participant gender:
Summary
To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM
in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose
administration.