Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B 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
Current Sponsor code: GZ402665
Other descriptive name: RECOMBIN
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patients with documented non-neuronopathic acid sphingomyelinase deficiency • The patient has a diffusing capacity of carbon monoxide (DLco) >20% and =80% of the predicted normal value. • The patient has a spleen volume =6 multiples of normal(MN). A partial splenectomy will be permitted if performed =1 year prior to Screening/Baseline and residual spleen volume is =6 MN. • The patient who is female and of childbearing potential must have a negative serum pregnancy test for ß-HCG. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 14 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: • The patient is breast-feeding • The patient has received an investigational drug within 30 days prior to study enrollment • The patient has a medical condition or any extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. • The patient has had a major organ transplant • ALT or AST >250 IU/L or total bilirubin >1.5 mg/dL. • The patient is unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each rhASM infusion for the duration of the study. • The patient requires medications that may decrease rhASM • The patient is unwilling or unable to avoid the use of medications or herbal supplements that may cause or prolong bleeding, or have potential hepatotoxicity within 10 days prior to and 3 days after liver biopsy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the safety, efficacy, and pharmacokinetics of different doses of rhASM administered intravenously (IV) every 2 weeks for 52 weeks. ;Secondary Objective: ;Primary end point(s): Percentage change in spleen volume;Timepoint(s) of evaluation of this end point: Baseline to end of study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Liver volume Pulmonary imaging and function testing Exercise capacity by cycle ergometry Hematology Physician Global Assessment Efficacy biomarkers;Timepoint(s) of evaluation of this end point: Baseline to end of study | — |
Countries
Brazil, Chile, France, Germany, Italy, Tunisia, United Kingdom, United States
Contacts
Public ContactMedical Information
Genzyme Europe B.V.
Outcome results
None listed