EUCTR2010-023953-12-IT进行中（未招募）不适用
A Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency - ASCEND
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 15
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
- •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
排除标准
- •The patient is female and pregnant or lactating.
- •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
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