Prospective, Randomized (1:1), Double-Blind, Parallel-Group, Active-Controlled, Multicenter Study to Compare Safety and Efficacy of Smoflipid to Intralipid 20% in Pediatric Patients of 3 Months to 16 Years of Age Requiring Parenteral Nutrition for at Least 90 Days and up to 1 Year
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Body Weight
研究概览
简要总结
Evaluate the safety and efficacy of Smoflipid compared to standard of care lipid emulsion Intralipid 20% administered via a central vein in pediatric patients 3 months to 16 years of age who require parenteral nutrition for at least 90 days and up to 1 year.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 3 Months 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients 3 months to 16 years of age.
- •Patients who require PN for at least 5 days/week.
- •Patients who receive 60% or more of their total energy requirements as PN at enrollment and who are expected to receive 60% or more of their total energy requirements as PN for at least 90 days.
- •Written informed consent from parent(s) or legal representative(s). If possible, patient assent must also be obtained (according to local law).
排除标准
- •Known hypersensitivity to fish, egg, soybean, or peanut proteins, or to any of the active ingredients or excipients of Smoflipid or Intralipid 20%.
- •Hyperlipidemia or disorders of lipid metabolism characterized by hypertriglyceridemia (serum triglyceride concentration > 250 mg/dL).
- •Inborn errors of amino acid metabolism.
- •Cardiopulmonary instability (including pulmonary edema, cardiac insufficiency, myocardial infarction, acidosis and hemodynamic instability requiring significant vasopressor support).
- •Hemophagocytic syndrome.
- •Liver enzymes (either AST, or ALT, or GGT) exceeding 5 x upper limit of normal range
- •Direct bilirubin ≥ 2.0 mg/dl
- •Any known hepatic condition outside of Intestinal Failure-Associated Liver Disease (IFALD) that will increase direct bilirubin ≥ 2.0 mg/dl.
- •Clinically significant abnormal levels of any serum electrolyte (sodium, potassium, magnesium, calcium, chloride, phosphate).
- •Active bloodstream infection demonstrated by positive blood culture at screening.
- •Severe renal failure including patients on renal replacement therapy.
- •Abnormal blood pH, oxygen saturation, or carbon dioxide.
- •Pregnancy or lactation.
- •Participation in another clinical study.
- •Unlikely to survive longer than 90 days.
研究组 & 干预措施
Smoflipid
Smoflipid is a sterile, nonpyrogenic, white, homogenous lipid emulsion for intravenous infusion. The lipid content of Smoflipid is 0.20 g/mL, and comprises a mixture of soybean oil, medium chain triglycerides, olive oil, and fish oil. Smoflipid is indicated as a source of calories and essential fatty acids for parenteral nutrition when oral or enteral nutrition is not possible, insufficient, or contraindicated.
The mean essential fatty acid content of Smoflipid is 35 mg/mL linoleic acid (omega-6) and 4.5 mg/mL α-linolenic acid (omega-3).
干预措施: Smoflipid (Drug)
Intralipid, 20%
Intralipid 20% is a sterile, non-pyrogenic fat emulsion intended as a source of calories and essential fatty acids. Intralipid 20% is indicated as a source of calories and essential fatty acids for patients requiring parenteral nutrition for extended periods of time and as a source of essential fatty acids for prevention of essential fatty acid deficiency. The major component fatty acids are linoleic acid, oleic acid, palmitic acid, α-linolenic acid and stearic acid.
干预措施: Intralipid, 20% (Drug)
结局指标
主要结局
Body Weight
时间窗: from day 1 monthly to day 365
Body weight of patients (patients \< 36 months of age)
Sterols in Plasma Including Phytosterols
时间窗: from day 1 monthly to day 365
Change From Baseline Urea Nitrogen
时间窗: from day 1 weekly to day 365
Change From Baseline Total Bilirubin
时间窗: from day 1 weekly to day 365
Change From Baseline Triglycerides
时间窗: from day 1 weekly to day 365
Fatty Acid Profile in Total Plasma
时间窗: from day 1 monthly to day 365
Fatty acid profile including linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid and Mead acid, analyzed in total plasma
Triene/Tetraene Ratio
时间窗: from day 1 weekly to day 365
Triene/tetraene ratio (Holman Index) in total plasma to assess essential fatty acid deficiency (EFAD)
Number of Patients in Each Treatment Group With Direct Bilirubin Levels > 2 mg/dL
时间窗: from day 1 monthly to day 365
Time Until Reaching Direct Bilirubin Levels > 2 mg/dL
时间窗: from day 1 monthly to day 365
Change From Baseline Aspartate Aminotransferase (AST)
时间窗: from day 1 weekly to day 365
Change From Baseline Direct Bilirubin
时间窗: from day 1 weekly to day 365
Change From Baseline White Blood Cell (WBC) Count
时间窗: from day 1 weekly to day 365
Change From Baseline Platelet Count
时间窗: from day 1 weekly to day 365
Change From Baseline Hemoglobin
时间窗: from day 1 weekly to day 365
Change From Baseline Sterols (Beta-sitosterol, Campesterol, Stigmasterol, Brassicasterol, Ergosterol, Cholesterol, Desmosterol, Lanosterol, Beta-sitostanol, Lathosterol, Squalene)
时间窗: from day 1 monthly to day 365
Change From Baseline Gamma-glutamyl Transferase (GGT)
时间窗: from day 1 weekly to day 365
Change From Baseline Glucose
时间窗: from day 1 weekly to day 365
Change From Baseline Total Protein
时间窗: from day 1 weekly to day 365
Change From Baseline Hematocrit
时间窗: from day 1 weekly to day 365
Change From Baseline International Normalized Ratio (INR)
时间窗: from day 1 weekly to day 365
Adverse Events
时间窗: from day 1 weekly to day 365
Genetic Polymorphisms of Fatty Acid Desaturase Genes FADS1 and FADS2
时间窗: once during treatment phase (day 1 to day 365)
The relation between genetic polymorphisms in the fatty acid desaturase genes Fatty acid desaturase 1 (FADS1) and Fatty acid desaturase 2 (FADS2) and plasma concentrations of linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid, and Mead acid, as well as relation to and EFAD (triene/tetraene ratio)
Change From Baseline Alanine Aminotransferase (ALT)
时间窗: from day 1 weekly to day 365
Body Height
时间窗: from day 1 monthly to day 365
Height oder length of body (patients \<36 months of age)
Head Circumference
时间窗: from day 1 monthly to day 365
Circumference of head in patients \> 36 months old
Change Form Baseline Alkaline Phosphatase (ALP)
时间窗: from day 1 weekly to day 365
Change From Baseline Creatinine
时间窗: from day 1 weekly to day 365
Change From Baseline Electrolytes (Na, K, Mg, Cl,Ca, Phosphate)
时间窗: from day 1 weekly to day 365
Vital Signs: Blood Pressure
时间窗: from day 1 monthly to day 365
Systolic and diastolic blood pressure
Vital Signs: Body Temperature
时间窗: from day 1 monthly to day 365
Fatty Acid Profile in Red Blood Cell Membranes
时间窗: from day 1 monthly to day 365
Fatty acid profile including linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid and Mead acid, analyzed in red blood cell membranes
Change From Baseline Trace Elements (Ferritin, Zn, Se, Cu, Mn, Cr)
时间窗: from day 1 weekly to day 365
Change From Baseline C-reactive Protein (CRP)
时间窗: from day 1 weekly to day 365
Change From Baseline Red Blood Cell (RBC) Count
时间窗: from day 1 weekly to day 365
Vital Signs: Heart Rate
时间窗: from day 1 monthly to day 365
次要结局
未报告次要终点