EUCTR2014-003198-40-DE进行中（未招募）1 期
A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Recombinant Human Acid Sphingomyelinase in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase DeficiencyRevised title further to the protocol amendment 1A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of olipudase afa in pediatric patients aged <18 years with acid sphingomyelinase deficiency
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
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 and/or patient’s parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed.
- •The patient is aged 0 to <18 years of age on the date of informed assent/consent.
- •The patient has documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes.
- •The patient has a spleen volume =5 multiples of normal (MN) measured by magnetic resonance imaging (MRI); patients who have had partial splenectomy will be allowed if the procedure was performed =1 year before screening and the residual spleen volume is =5 MN.
- •The patient’s height is -1 Z-score or lower.
- •A negative serum pregnancy test in female patients of childbearing potential.
- •Female patients of childbearing potential and sexually active 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? yes
- •Number of subjects for this age range: 20
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •The patient has received an investigational drug within 30 days before study enrollment
- •The patient has any of the following medical conditions:
- •- An active, serious, intercurrent illness.
- •- Active hepatitis B or hepatitis C infection.
- •- Infection with human immunodeficiency virus (HIV).
- •- Cirrhosis (determined by clinical evaluation).
- •- Significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or <40% left ventricular ejection fraction by echocardiogram).
- •- Malignancy diagnosed within the previous 5 years (except basal cell carcinoma).
- •- Any other extenuating circumstance that can significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
- •The patient has acute or rapidly progressive neurological abnormalities.
- •The patients is homozygous for SMPD1 gene mutations R496L, L302P, and fs330 or any combination of these 3 mutations.
- •The patient has a delay of gross motor skills.
- •The patient has had a major organ transplant (eg, bone marrow, liver).
- •The patient requires use of invasive ventilatory support.
- •The patient requires use of noninvasive ventilatory support while awake and for >12 hours a day.
- •The patient, in the investigator’s opinion, is unable to adhere to the requirements of the study.
- •The patient has a platelet count <60,000 /µL (based on the average of 2 screening samples obtained greater than 24 hours apart).
- •The patient has alanine aminotransferase or aspartate aminotransferase >250 IU/L or total bilirubin>1.5 mg/dL.
- •The patient has an international normalized ratio (INR) >1.5
- •The patient is unwilling or unable to abstain from ingesting alcohol the day before through 3 days after each infusion of rhASM during the treatment period. Measuring alcohol concentration in blood is not required.
- •The patient is scheduled during the study for in-patient hospitalization including elective surgery.
- •The patient requires medication(s) that may decrease rhASM activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, or desipramine]).
- •The patient is breast-feeding.
研究者
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