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

Malnutrition Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06274788
• Other study ID #: OMEG-054-CP4
• Status: Not yet recruiting
• Phase: Phase 4
• Start date: May 1, 2024
• Est. completion date: March 1, 2027

Study information

• Verified date: March 2024
• Source: Fresenius Kabi
• Contact: Schneider-Schranz Cornelia, PhD
• Phone: +491737335940
• Email: cornelia.schneider-schranz[@]fresenius-kabi.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 40
• Est. completion date: March 1, 2027
• Est. primary completion date: March 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 17 Years

Eligibility

Inclusion Criteria:

1. Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).

2. 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.

3. Patient has oral or enteral feeding intolerance or at least one gastrointestinal disorder requiring PN.

Exclusion Criteria:

1. Patient has received Omegaven within four weeks before inclusion in the study

2. 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.

3. Patient has known cirrhosis (liver biopsy is not required under this protocol).

4. Patient has been previously diagnosed with, or has prior evidence of, portal vein thrombosis.

5. Patient has previously received a liver-only or liver-inclusive transplant.

6. Patient has hemodynamic instability due to any major cardiac anomaly.

7. Patient has a major life-threatening disease (e.g., sepsis requiring high-dose vasopressors, acute respiratory distress syndrome, veno-occlusive disease, cancer).

8. 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.

9. Patient has renal failure and requires renal replacement therapy.

10. Patient has a severe hemorrhagic disorder.

11. Patient has severe hyperlipidemia or a severe disorder of lipid metabolism characterized by hypertriglyceridemia (i.e., serum triglyceride level > 1,000 mg/dL).

12. Patient has a record of EFAD before inclusion in the study

13. Patient has been diagnosed with or is suspected to have an inborn error of metabolism.

14. Patient has a known hypersensitivity to fish or egg protein or to any of the active ingredients or excipients of Omegaven.

15. Patient is subject to treatment limitation.

16. Patient is enrolled in any other study with an investigational medicinal product during the course of the current study.

Related Conditions & MeSH terms

• Essential Fatty Acid Deficiency
• Liver Diseases
• Malnutrition
• Parenteral Nutrition Associated Liver Disease (PNALD)
• Pediatric ALL

Intervention

Drug:
• 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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Fresenius Kabi

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of essential fatty acid deficiency (EFAD)	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)	Up to week 56
Secondary	Incidence of clinical EFAD	Clinical EFAD is defined by clinical symptoms (like dry, scaly rash, hair loss, hair depigmentation, poor wound healing, growth restriction, and increased susceptibility to infection) and T:T ratio = 0.083	Up to week 56
Secondary	Time from treatment start to EFAD diagnosis	Time from treatment start to EFAD diagnosis	Up to week 56
Secondary	Incidence of adverse events (AEs)/serious adverse events (SAEs)	The incidence of AEs/SAEs considered by the Investigator to be related to study treatment	Up to week 56
Secondary	Routine laboratory tests: Direct or conjugated bilirubin		Up to week 56
Secondary	Routine laboratory tests: triglycerides		Up to week 56
Secondary	Fatty acid profiles: a-linolenic acid		Up to week 56
Secondary	Fatty acid profiles: linoleic acid		Up to week 56
Secondary	Fatty acid profiles: arachidonic acid		Up to week 56
Secondary	Fatty acid profiles: mead acid		Up to week 56
Secondary	Fatty acid profiles: oleic acid		Up to week 56
Secondary	Fatty acid profiles: docosahexaenoic acid (DHA)		Up to week 56
Secondary	Fatty acid profiles: eicosapentaenoic acid (EPA)		Up to week 56
Secondary	Anthropometric measures: body weight		Up to week 56
Secondary	Anthropometric measures: body height/length		Up to week 56
Secondary	Anthropometric measures: head circumference		Up to week 56