a study on Sphingomyelin Lipidosis
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.
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
The total duration per patient is 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 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.
The study will be divided into 2 consecutive major periods: 1) a randomized placebo-controlled, double-blinded primary analysis period (PAP) from day -60 to week 52, which will be followed by 2) an extension treatment period (ETP) that will last up to 4 years and 1 month and during which all patients will receive olipudase alfa.
Open to people ages 18 years and up
The patient has alanine aminotransferase (ALT) or aspartate aminotransferase (AST)
250 IU/L or total bilirubin>1.5 mg/dL (except for patients with Gilbert's syndrome).
The patient is breast-feeding.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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