A Multicenter, Prospective, Controlled, Longitudinal-Cohort Study Assessing the Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 主要终点
- Incidence of:
研究概览
简要总结
This study will demonstrate Short-, mid-, and long-term 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
详细描述
Prospective, controlled, longitudinal-cohort phase IV study to assess safety of Omegaven in Pediatric Patients with PNAC, in order to demonstrate short-, mid-, and long-term safety with respect to EFAD, serious bleeding events, life-threatening pleural and pericardial effusions, and neurocognitive development. Pediatric patients with PNAC receiving Omegaven as part of their routine nutritional management will be compared to those treated with another FDA-approved ILE for pediatric patients. Patients who are switched to a non-FDA-approved ILE or to Omegaven will be analyzed separately. Use of ILE will be as indicated by their treating physician, ideally for ≥ 1 year. Dosing modalities are to be taken from the pertinent prescribing information.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Day 至 —（Child, Adult, Older Adult）
- 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).
- •Patient is PN-dependent and within the past 21 days 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.
- •Patient has feeding intolerance or at least one gastrointestinal disorder requiring PN.
- •Patient is < 6 months corrected age (expected time of delivery to time of screening).
排除标准
- •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 known portal vein thrombosis (imaging studies are not required under this protocol).
- •Patient has previously received a liver-only or liver-inclusive transplant.
- •Patient has a major cardiac anomaly with hemodynamic instability.
- •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 dialysis.
- •Patient has a severe hemorrhagic disorder.
- •Patient has an INR > 2.
- •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 a previous T:T ratio ≥ 0.2 or had a previous diagnosis of EFAD.
- •Patient has a central nervous system anomaly (e.g., anencephaly) or any injury (e.g., grade 3 or 4 intraventricular hemorrhage, moderate to severe hypoxic ischemic encephalopathy) that will affect neurodevelopment.
- •Patient has been diagnosed with or is suspected to have a genetic disorder known to be associated with neurodevelopmental impairment (e.g., trisomy 21, 18, 13).
- •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's medical care has been deemed futile by the medical team.
- •Patient is enrolled in any other study with an investigational medicinal product.
研究组 & 干预措施
Pediatric patients
Pediatric patients with new-onset PNAC
干预措施: Omegaven® (fish oil triglycerides) Injectable Emulsion (Drug)
结局指标
主要结局
Incidence of:
时间窗: Through study completion, a maximum of 6 years
1. Incidence of EFAD 2. Incidence of serious bleeding events 3. Incidence of life-threatening pericardial effusion events 4. Incidence of life-threatening pleural effusion events 5. Severity of any neurodevelopmental delays.
次要结局
未报告次要终点