Clinical Trial: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional



Official Title: A Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With

Brief Summary:

Primary Objective:

The primary objective of this phase 2/3 study is to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult patients with acid sphingomyelinase deficiency (ASMD) by assessing changes in 1) spleen volume as measured by abdominal magnetic resonance imaging (MRI) (and, for the United States [US] only, in association with patient perception related to spleen volume as measured by splenomegaly related score (SRS)); and 2) infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide (DLCO).

Secondary Objectives:

  • To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks.
  • To characterize the effect of olipudase alfa on the patient perception related to spleen volume as measured by the SRS after 52 weeks of study drug administration. (For the US, the effect of olipudase alfa on the splenomegaly related score is part of the primary objective).
  • To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following endpoints assessed sequentially:
  • The effect of olipudase alfa on liver volume;
  • The effect of olipudase alfa on platelet count;
  • The effect of olipudase alfa on fatigue;
  • The effect of olipudase.alfa on pain;
  • The effect of olipudase alfa on dyspnea.