Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 17.1 Level: LLT Classification code 10041515 Term: Sphingomyelin lipidosis System Organ Class: 100000004850
Conditions
Interventions
Product Name: rhASM
Product Code: GZ402665
Pharmaceutical Form: Powder for concentrate for solution for infusion
CAS Number: 927883-84-9
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 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 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 rhASM 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 decrease rhASM activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, or desipramine]). The patient is breast-feeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of recombinant human acid sphingomyelinase (rhASM) 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 rhASM administered intravenously in pediatric patients every 2 weeks for 52 weeks.; Primary end point(s): Number of adverse events Clinically significant changes in laboratory parameters Clinically significant changes in physical examinations ;Timepoint(s) of evaluation of this end point: From screening through week 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Maximum concentration (Cmax) Area under the curve until the last measurable concentration (AUClast) Area under the curve extrapolated to infinity (AUC) Half-life (t1/2) Clearance (CL) Volume of distribution (Vss) Change in sphingomyelin levels Change in sphingomyelin metabolite levels ; Timepoint(s) of evaluation of this end point: With the first infusion at 0.3, 1.0 and 3.0 mg/kg and at week 52 Maximum concentration (Cmax) Area under the curve until the last measurable concentration (AUClast) Area under the curve extrapolated to infinity (AUC) Half-life (t1/2) Clearance (CL) Volume of distribution (Vss) From day 1 through week 52 Change in sphingomyelin levels Change in sphingomyelin metabolite levels | — |
Countries
Brazil, Chile, France, Germany, Italy, United Kingdom, United States
Contacts
Public ContactContact Point
Sanofi S.p.A.
Outcome results
None listed