NCT06274788招募中4 期
Title: A Single-arm, Open-label, Prospective, Multicenter Safety Study to Evaluate the Occurrence of Essential Fatty Acid Deficiency (EFAD) in Pediatric Patients With Parenteral Nutrition-associated Cholestasis (PNAC) Who Require More Than Eight Weeks of Omegaven Treatment
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 17
- 主要终点
- Incidence of essential fatty acid deficiency (EFAD)
研究概览
简要总结
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Day 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
- •Pediatric patient (<18 years) has been diagnosed with PNAC, defined as direct or conjugated bilirubin (DBil) ≥ 2.0 mg/dL with no other known cause of liver dysfunction at the time of enrollment and is expected to require Omegaven treatment for at least eight weeks.
- •Patient has oral or enteral feeding intolerance or at least one gastrointestinal disorder requiring PN.
排除标准
- •Patient has received Omegaven within four weeks before inclusion in the study
- •Patient has any other known cause of chronic liver disease such as hepatitis C, cystic fibrosis, biliary atresia, alpha-1-antitrypsin deficiency, passive hepatic congestion due to heart failure, etc.
- •Patient has known cirrhosis (liver biopsy is not required under this protocol).
- •Patient has been previously diagnosed with, or has prior evidence of, portal vein thrombosis.
- •Patient has previously received a liver-only or liver-inclusive transplant.
- •Patient has hemodynamic instability due to any major cardiac anomaly.
- •Patient has a major life-threatening disease (e.g., sepsis requiring high-dose vasopressors, acute respiratory distress syndrome, veno-occlusive disease, cancer).
- •Patient has multi-organ failure, septic shock, hypotension requiring pressor therapy, persistent pulmonary hypertension requiring inhaled nitric oxides, or requires extracorporeal membrane oxygenation (ECMO) or similar intervention.
- •Patient has renal failure and requires renal replacement therapy.
- •Patient has a severe hemorrhagic disorder.
- •Patient has severe hyperlipidemia or a severe disorder of lipid metabolism characterized by hypertriglyceridemia (i.e., serum triglyceride level > 1,000 mg/dL).
- •Patient has a record of EFAD before inclusion in the study
- •Patient has been diagnosed with or is suspected to have an inborn error of metabolism.
- •Patient has a known hypersensitivity to fish or egg protein or to any of the active ingredients or excipients of Omegaven.
- •Patient is subject to treatment limitation.
- •Patient is enrolled in any other study with an investigational medicinal product during the course of the current study.
研究组 & 干预措施
Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion)
The dose of investigational drug (study treatment), as well as all other components of the overall nutritional regimen is solely at the discretion of the Investigator. It is assumed the Investigator will use sound medical judgement, follow institutional standards of care regarding the nutrition provided to each patient, and review applicable prescribing information indicating the maximum and recommended dose of Omegaven of 1 g/kg/day infused intravenously over 8 to 24 hours as long as the infusion rate does not exceed 1.5 mL/kg/hour.
干预措施: Omegaven® (fish oil triglycerides) Injectable Emulsion (Drug)
结局指标
主要结局
Incidence of essential fatty acid deficiency (EFAD)
时间窗: Up to week 56
EFAD as defined by the triene:tetraene (T:T) ratio; severity of EFAD will be graded and analysed based on T:T ratio (suspected ≥ 0.05, moderate ≥ 0.20, and severe ≥ 0.40)
次要结局
- Time from treatment start to EFAD diagnosis(Up to week 56)
- Routine laboratory tests: Direct or conjugated bilirubin(Up to week 56)
- Fatty acid profiles: linoleic acid(Up to week 56)
- Fatty acid profiles: arachidonic acid(Up to week 56)
- Fatty acid profiles: mead acid(Up to week 56)
- Fatty acid profiles: oleic acid(Up to week 56)
- Incidence of clinical EFAD(Up to week 56)
- Fatty acid profiles: α-linolenic acid(Up to week 56)
- Anthropometric measures: head circumference(Up to week 56)
- Incidence of adverse events (AEs)/serious adverse events (SAEs)(Up to week 56)
- Routine laboratory tests: triglycerides(Up to week 56)
- Fatty acid profiles: docosahexaenoic acid (DHA)(Up to week 56)
- Fatty acid profiles: eicosapentaenoic acid (EPA)(Up to week 56)
- Anthropometric measures: body weight(Up to week 56)
- Anthropometric measures: body height/length(Up to week 56)
研究者
研究点 (17)
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