EUCTR2015-000371-26-IT进行中（未招募）1 期
A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency - ASCEND
GENZYME CORPORATIO0 个研究点目标入组 35 人开始时间: 2018年2月6日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 35
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •The patient is willing and able to provide signed written informed consent. The patient is male or female aged 18 years or older. The patient has documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes; and a clinical diagnosis consistent with Niemann-Pick disease type B (NPD B). The patient has diffuse capacity of the lung for carbon monoxide =70% of the predicted normal value. The patient has a spleen volume =6 multiples of normal (MN) measured by MRI; patients who have had partial splenectomy will be allowed if the procedure was performed =1 year before screening/baseline and the residual spleen volume is =6 MN. Female patients of childbearing potential must have a negative serum pregnancy test for beta-human chorionic gonadotropin (ß-HCG). 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.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range: 1
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 34
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •The patient has received an investigational drug within 30 days before study enrollment. The patient has a medical condition, including significant intercurrent illness; significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or <40% left ventricular ejection fraction by echocardiogram); active hepatitis B or hepatitis C, or infection with human immunodeficiency virus (HIV); cirrhosis (as determined by clinical evaluation or liver biopsy); malignancy diagnosed within the past 5 years (other than basal cell carcinoma), or any other extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities. The patient has a platelet count <60,000/µL (based on the average of 2 samples obtained at least 12 hours but no longer than 24 hours apart). The patient has an international normalized ratio (INR) >1.5. The patient has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >250 IU/L or total bilirubin >1.5 mg/dL (except for patients with Gilbert's syndrome). The patient has had a major organ transplant (eg, bone marrow or liver). The patient is scheduled during the study for in-patient hospitalization including elective surgery and excluding the liver biopsies required per protocol. 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 before and 3 days after each study drug infusion. Testing for blood alcohol levels will not be required. The patient is unwilling or unable to avoid 10 days before and 3 days after the protocol scheduled liver biopsies that are required at screening/baseline and at week 52, the use of medications or herbal supplements that are potentially hepatotoxic (e.g., 3-hydroxy-3-methyl glutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, anti-depressants, kava, echinacea) and/or may cause or prolong bleeding (e.g., anti-coagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng). The patient requires medications that may decrease olipudase alfa activity (e.g., fluoxetine, chlorpromazine, tricyclic antidepressants [e.g., imipramine, or desipramine]). The patient requires use of invasive ventilatory support. The patient requires use of noninvasive ventilator support while awake for longer than 12 hours daily. The patient is breast-feeding.
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