Date: 2016-07-06
Type of information: Recruitment of the first patient
phase: 2-3
Announcement: recruitment of the first patient
Company: Sanofi Genzyme (USA - MA), a Sanofi company (France)
Product: olipudase alfa
Action
mechanism: enzyme replacement therapy. Olipudase alfa is a recombinant human acid sphingomyelinase. Traditionally called Niemann-Pick Disease types A and B (NPD A and NPD B), acid sphingomyelinase deficiency (ASMD) is one of a group of lysosomal storage diseases that affect the metabolism and that are caused by genetic mutations. ASMD is caused by the deficiency of a specific enzyme, acid sphingomyelinase (ASM). This enzyme is found in special compartments within cells called lysosomes and is required to metabolize a lipid called sphingomyelin. If ASM is absent or not functioning properly, sphingomyelin cannot be metabolized properly and is accumulated within the cell, eventually causing cell death and the malfunction of major organ systems. Niemann-Pick A and Niemann-Pick B are both caused by the same enzymatic deficiency and there is growing evidence that the two forms represent opposite ends of a continuum. The FDA has granted Breakthrough Therapy designation to olipudase alfa.
Disease: acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B
Therapeutic area: Rare diseases - Genetic diseases
Country: France, Germany, Japan, The Netherlands, Spain, Turkey, UK
Trial
details: ASCEND is a phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeat-dose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency. The primary objective of this phase 2/3 study is to evaluate the efficacy of different doses 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 separately, changes in spleen volume as measured by abdominal magnetic resonance imaging (MRI) and infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide. The secondary objectives are to confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks, to characterize the effect of olipudase alfa after 52 weeks of study drug administration on a symptom-based composite score composed of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints), to characterize the effect of olipudase alfa on the splenomegaly-related symptom score after 52 weeks of study drug administration, to characterize the effect of olipudase alfa on liver volume after 52 weeks of study drug administration and to characterize the effect of olipudase alfa on platelet count after 52 weeks of study drug administration. (NCT02004691)
Latest
news: * On July 6, 2016, Sanofi Genzyme, the specialty care global business unit of Sanofi, announced that the first adult patient has enrolled and been dosed in a pivotal Phase 2/3 clinical trial named ASCEND for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B). Thirty-six patients are expected to be enrolled in the study and receive olipudase alfa or a placebo. Upon completion of the 52 week primary analysis period, all patients will receive treatment in an extension period. In June of last year, Sanofi Genzyme announced the beginning of a Phase 1/2 trial in pediatric patients with ASMD, specifically NPD B. * On June 4, 2015, Sanofi and its subsidiary Genzyme announced that Genzyme is preparing for enrollment of a Phase 2/3 adult study in the second half of 2015.
