Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 20.1 Level: LLT Classification code 10041515 Term: Sphingomyelin lipidosis System Organ Class: 100000004850
Conditions
Sponsors
Genzyme Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The patient completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor The patient and/or the patient's parent(s)/legal guardian(s) is willing and able to provide signed written informed consent. The patient who is female and of childbearing potential must have a negative urine pregnancy test for beta human chorionic gonadotropin (ß HCG). Female patients of childbearing potential and sexually mature 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 up to 15 days following their last dose of study drug Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -The patient has any new condition or worsening of an existing condition which in the opinion of the investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study. -The patient, in the opinion of the investigator, is unable to adhere to the requirements of the study. -The patient is unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period. -The patient is unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (eg, 3 hydroxy 3 methylglutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, antidepressants, kava, echinacea) or may cause or prolong bleeding (eg, anticoagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng) (only patients who previously participated in the DFI13412 study). -The patient requires medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa ;Secondary Objective: The secondary objectives of this study are to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration;Primary end point(s): 1/ AE/TEAEs, including infusion-associated reactions and AESIs. 2/ Complete physical examinations including extended neurologic, weight, height (pediatric patients only) and abbreviated physical exams. 3/ Vital signs, echocardiograms and electrocardiograms with Doppler. 4/ Clinical laboratory tests. 5/ Safety biomarkers. 6/ Liver biopsy (patients previously enrolled in DFI13412). 7/ Liver ultrasound/Doppler (patients previously enrolled in DFI13803) 8/ Immune response assessments ;Timepoint(s) of evaluation of this end point: From 1/ to 5/ and 8/: Time Frame: Baseline to 9 years: 6/: Time Frame: Baseline to 3 years for liver biopsy 7/ Baseline to 5 years for liver ultrasound/doppler | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume - Pulmonary Imaging - Pulmonary function test - Hematology (hemoglobin and platelet count) - Lipid profile - Health outcome questionnaires - Hand X ray for bone age and bone maturation (pediatric patients) - Linear patient growth by height Z -score (pediatric patients);Timepoint(s) of evaluation of this end point: Baseline to 9 years | — |
Countries
Belgium, Brazil, France, Germany, Italy, United Kingdom, United States
Contacts
Public ContactMedical Information
Genzyme Europe B.V.
Outcome results
None listed