New York, New York 10029


Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 52 weeks. Secondary Objective: To characterize the pharmacokinetic profile and evaluate the pharmacodynamics and exploratory efficacy of olipudase alfa administered intravenously in pediatric patients every 2 weeks for 52 weeks.

Study summary:

The maximum study duration per patient is approximately 18 months (screening period: up to 60 days; treatment period: 64 weeks; post-treatment period: up to 37 days, not applicable if patient enrolls in a long term extension treatment trial).


Inclusion criteria: - The patient and/or patient's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed. - The patient is aged 0 to <18 years of age on the date of informed assent/consent. - The patient has documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes. - The patient has a spleen volume ≥5 multiples of normal (MN) measured by magnetic resonance imaging (MRI); patients who have had partial splenectomy will be allowed if the procedure was performed ≥1 year before screening and the residual spleen volume is ≥5 MN. - The patient's height is -1 Z-score or lower. - A negative serum pregnancy test in female patients of childbearing potential. - Female patients of childbearing potential and male patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception. Exclusion criteria: - The patient has received an investigational drug within 30 days before study enrollment. - The patient has any of the following medical conditions: - An active, serious, intercurrent illness. - Active hepatitis B or hepatitis C infection. - Infection with human immunodeficiency virus (HIV). - Cirrhosis (determined by clinical evaluation). - Significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or <40% left ventricular ejection fraction by echocardiogram). - Malignancy diagnosed within the previous 5 years (except basal cell carcinoma). - Any other extenuating circumstance that can significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. - The patient has acute or rapidly progressive neurological abnormalities. - The patient is homozygous for SMPD1 gene mutations R496L, L302P, and fs330 or any combination of these 3 mutations. - The patient has a delay of gross motor skills. - The patient has had a major organ transplant (eg, bone marrow, liver). - The patient requires use of invasive ventilatory support. - The patient requires use of noninvasive ventilatory support while awake and for >12 hours a day. - The patient, in the investigator's opinion, is unable to adhere to the requirements of the study. - The patient has a platelet count <60 × 10^3/µL (based on the average of 2 screening samples obtained up to 24 hours apart). - The patient has alanine aminotransferase or aspartate aminotransferase >250 IU/L or total bilirubin >1.5 mg/dL. - The patient has an international normalized ratio (INR) >1.5 - The patient is unwilling or unable to abstain from ingesting alcohol the day before through 3 days after each infusion of olipudase alfa during the treatment period. Measuring alcohol concentration in blood is not required. - The patient is scheduled during the study for in-patient hospitalization including elective surgery. - The patient requires medication(s) that may can decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, or desipramine]). - 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.



Primary Contact:

Study Director
Clinical Sciences & Operations

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Backup Contact:


Location Contact:

New York, New York 10029
United States

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Site Status: Recruiting

Data Source:

Date Processed: November 17, 2017

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