Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion (Part A)

Phase 4

Completed

Conditions: Essential Fatty Acid Deficiency (EFAD)

Interventions: Drug: Clinolipid
Drug: Intralipid

Registration Number: NCT04555044

Lead Sponsor: Baxter Healthcare Corporation

Brief Summary: This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care (Intralipid) from 7 up to 90 days to develop Essential Fatty Acid Deficiency (EFAD). Additionally, this study design will evaluate the safety and efficacy of using Clinolipid or Intralipid in a pediatric population.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 101

Inclusion Criteria

Patients and/or their legal representative must be able to understand the study and voluntarily sign the informed consent form (ICF) per 21 CFR Part 50.55(e)
Patients and/or their legal representative accept adherence to protocol requirements
Patients who are expected to require parenteral nutrition (PN)for at least 7 days
Premature infants (born at 24 to <37 weeks of gestation with a birth weight ≥750g) require at least 80% of targeted energy requirements by PN at study entry (up to 1 month CA); full term infants and children require at least 70% of targeted energy requirements by PN at study entry

Exclusion Criteria

Patients who are not expected to survive hospitalization or with a severe critical unresponsive illness at time of initiation with foreseeable intercurrent events that could jeopardize the patient's participation in the study, as judged by the Investigator (e.g., unresponsive shock, sepsis, renal failure requiring dialysis, severe unresponsive metabolic acidosis, and/or severe unresponsive metabolic disorders);
Patients with a known hypersensitivity to lipid emulsion, egg or soybean proteins, or any of the active substances, excipients, or components of the container or who have a history of an adverse event due to ILE;
Patients with liver disease including cholestasis;
Patients with severe hyperlipidemia or severe disorders of lipid metabolism characterized by hypertriglyceridemia (triglyceride >400 mg/dL);
Patients who are unable to tolerate the necessary laboratory monitoring;
Patients who are enrolled in another clinical trial involving an investigational agent;
Patients with a known history of either severe hemorrhagic or severe hemolytic disease as judged by the investigator;
Premature infants born <24 weeks of gestation and patients ≥18 years;
Premature infants with a birth weight <750 g;
Patient requires or is expected to require propofol for sedation;
Patient has received a diagnosis of Coronavirus Disease of 2019 (COVID-19) (diagnosis <2 months prior and/or symptoms have not resolved.
Newborn patient born to a mother who was diagnosed as COVID-19 positive at delivery or within 2 months prior to delivery
Female patients who are pregnant. Note: All female patients ≥12 years of age must have a negative urine human chorionic gonadotropin (hCG) pregnancy test at screening. For female patients <12 years of age, a urine hCG test at screening will be performed at the discretion of the investigator based on childbearing potential.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Clinolipid	Clinolipid	Dosing based on American Academy of Pediatrics (AAP), American Academy Society for Parenteral and Enteral Nutrition (ASPEN), European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN)/ European Society for Parenteral and Enteral Nutrition (ESPEN)/ European Society of Paediatric Research (ESPR)/ Chinese Society for Parenteral and Enteral Nutrition (CSPEN) guidelines. Study treatment will be administered intravenously in the hospital setting from 7 up to 90 days using either a syringe pump or an infusion pump as appropriate for the daily volume of infusate. The flow rate will be prescribed by the Investigator to provide the daily dosage over 20 to 24 hours.
Intralipid	Intralipid	Dosing based on AAP, ASPEN, ESPGHAN/ESPEN/ESPR/CSPEN guidelines. Study treatment will be administered intravenously in the hospital setting from 7 up to 90 days using either a syringe pump or an infusion pump as appropriate for the daily volume of infusate. The flow rate will be prescribed by the Investigator to provide the daily dosage over 20 to 24 hours.

Primary Outcome Measures

Name	Time	Method
Number of Participants to Develop Essential Fatty Acid Deficiency (EFAD) Defined by Holman Index > 0.4	Up to Day 90	Holman Index is the plasma Triene:Tetraene ratio, specifically 5,8,11-eicosatrienoic acid \[mead acid\] to 5,8,11,14 eicosatetraenoic acid \[arachidonic acid, \[ARA\] ratio

Secondary Outcome Measures

Name	Time	Method
Stigmasterol Blood Level	Up to Day 90	Phytosterol species
Calories Nutritional Intake	Up to Day 90
Gamma-Glutamyl Transferase (GGT)	Up to Day 90	Plasma liver function test
Direct Bilirubin	Up to Day 90	Plasma liver function test
Campesterol Blood Level	Up to Day 90	Phytosterol species
Total Bilirubin	Up to Day 90	Plasma liver function test
Sitosterol Blood Level	Up to day 90	Phytosterol species.
Squalene Blood Level	Up to Day 90
Protein Nutritional Intake	Up to Day 90
Alkaline Phosphatase (ALP)	Up to Day 90	Plasma liver function test
Number of Adverse Events of Special Interest	Up to Day 120 (30 Days After Subject's Last Study Treatment if hospital discharge has not occurred)
Aspartate Aminotransferase (AST)	Up to Day 90	Plasma liver function test
Alanine Aminotransferase (ALT)	Up to Day 90	Plasma liver function test
Lipid Nutritional Intake	Up to Day 90
Number of Participants to Develop Parenteral Nutrition-Associated Liver Disease (PNALD)	Up to Day 90	Defined by direct bilirubin ≥2 mg/dL in patients receiving with intravenous lipid emulsion (ILE).
Cholesterol Blood Level	Up to Day 90
Carbohydrates Nutritional Intake	Up to Day 90
Change in Length or Height (and Head Circumference for Infants <1 Year of Age) From Baseline	Up to Day 90	Change in length/height from baseline (mm/week in all) = \[Length (mm) on Day X - Length (mm) at baseline\] / \[X/7\] Change in head circumference from baseline (mm/week in infants \<1 year) = \[Head circumference (mm) on Day X - Head circumference (mm) at baseline\] / \[X/7\]
Body Weight	Upto Day 90	Change in weight from baseline to end of treatment (EOT) for those \> 1 year of age (g/day)
Number of Participants With Neonatal Morbidities	Up to Day 90	Neonatal Morbidities are presented for premature infants born \< 37 weeks of gestation up to 1 month corrected age. Patients may have more than 1 neonatal morbidity.