Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency — ASCEND  

Sphingomyelin Lipidosis Clinical Trial

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 Acid Sphingomyelinase Deficiency

Status Completed

Clinical Trial 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 participants 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 participant 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 participant 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 SRS is part of the primary objective).

• To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following outcome measures 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.

Clinical Trial Description

The total duration per participant will be at least 3 years and up to 5 years and 3 months. This includes up to approximately two month of screening, 52 weeks of primary analysis period, up to 4 years and 3 months of extension treatment period, an end-of- study visit within 2 weeks of the last treatment, and a safety follow-up 30 to 37 days after the last treatment. ;

Related Conditions & MeSH terms

• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• Sphingomyelin Lipidosis

• NCT number: NCT02004691
• Study type: Interventional
• Source: Sanofi
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: December 18, 2015
• Completion date: October 19, 2023