NL-OMON54584招募中2 期
A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency - ASCEND
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Sanofi B.V.
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 64（—）
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 (β-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.
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 below 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 below 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) larger than
- •1.5., The patient has alanine aminotransferase (ALT) or aspartate
- •aminotransferase (AST) above 250 IU/L or total bilirubin above1.5 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.
研究者
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