A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Genzyme Corporation0 个研究点目标入组 25 人开始时间: 2013年7月22日最近更新: 2020年10月19日

研究概览

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•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

•-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]).

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