Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment

Официальное название

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

Обзор

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

Вмешательства

• Препарат Omegaven® (fish oil triglycerides) Injectable Emulsion
  Pediatric patients Pediatric patients with new-onset PNAC Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion Dose, frequency and duration is a decision of the Investigator

Первичные конечные точки

• Incidence of essential fatty acid deficiency (EFAD) [Срок оценки: Up to week 56]

Вторичные конечные точки (12)

• Incidence of clinical EFAD [Срок оценки: Up to week 56]
• Time from treatment start to EFAD diagnosis [Срок оценки: Up to week 56]
• Incidence of adverse events (AEs)/serious adverse events (SAEs) [Срок оценки: Up to week 56]
• Routine laboratory tests: Direct or conjugated bilirubin [Срок оценки: Up to week 56]
• Routine laboratory tests: triglycerides [Срок оценки: Up to week 56]
• Fatty acid profiles: α-linolenic acid [Срок оценки: Up to week 56]
• Fatty acid profiles: linoleic acid [Срок оценки: Up to week 56]
• Fatty acid profiles: arachidonic acid [Срок оценки: Up to week 56]
• Fatty acid profiles: mead acid [Срок оценки: Up to week 56]
• Fatty acid profiles: oleic acid [Срок оценки: Up to week 56]
• Fatty acid profiles: docosahexaenoic acid (DHA) [Срок оценки: Up to week 56]
• Fatty acid profiles: eicosapentaenoic acid (EPA) [Срок оценки: Up to week 56]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

США · 10 центров

• Memorial Health Service — Fountain Valley
• University of California Los Angeles — Los Angeles
• The University of Chicago — Chicago
• Children's Hospital Corporation d/b/a Boston Children's Hospital — Boston
• Children's Hospital Medical Center — Cincinnati
• Board of Regents of the University of Oklahoma Health Sciences Center — Oklahoma City
• Vanderbilt University Medical Center — Nashville
• Baylor College of Medicine Houston — Houston
• … и ещё 2 центра

Идентификаторы

NCT: NCT06274788 · OMEG-054-CP4

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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