Clinical trial NCT04555044: Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion (Part A)
Official title: A Randomized, Double-Blind, Controlled, Clinical Trial to Evaluate the Risk of Developing Essential Fatty Acid Deficiency in Pediatric Patients, Including Neonates, Receiving Either Clinolipid (Lipid Injectable Emulsion, USP) 20% or Standard-of-Care Soybean Oil-Based Lipid Emulsion (Part A)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Essential Fatty Acid Deficiency (EFAD)
Keywords: Essential Fatty Acids (EFA)
Parenteral Nutrition
Clinolipid/Clinoleic
Parenteral Nutrition Associated Cholestasis (PNAC)
Parenteral Nutrition Associated Liver Disease (PNALD)
Intestinal Failure Associated Liver Disease (IFALD)
Phytosterols
Intralipid
Infants/Preterm Infants
FADS1 and FADS2
Short Bowel Syndrome
Cancer Nutrition
Olive Oil Emulsion
Soybean Oil Emulsion
Lead sponsor: Baxter Healthcare Corporation
Sponsor class: INDUSTRY
First posted: 2020-09-18
Enrollment: 101
Sex: ALL
Minimum age (years): 0
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. 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)
2. Patients and/or their legal representative accept adherence to protocol requirements
3. Patients who are expected to require parenteral nutrition (PN)for at least 7 days
4. 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:

1. 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);
2. 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;
3. Patients with liver disease including cholestasis;
4. Patients with severe hyperlipidemia or severe disorders of lipid metabolism characterized by hypertriglyceridemia (triglyceride \>400 mg/dL);
5. Patients who are unable to tolerate the necessary laboratory monitoring;
6. Patients who are enrolled in another clinical trial involving an investigational agent;
7. Patients with a known history of either severe hemorrhagic or severe hemolytic disease as judged by the investigator;
8. Premature infants born \<24 weeks of gestation and patients ≥18 years;
9. Premature infants with a birth weight \<750 g;
10. Patient requires or is expected to require propofol for sedation;
11. Patient has received a diagnosis of Coronavirus Disease of 2019 (COVID-19) (diagnosis \<2 months prior and/or symptoms have not resolved.
12. Newborn patient born to a mother who was diagnosed as COVID-19 positive at delivery or within 2 months prior to delivery
13. 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.

Primary outcomes:
measure: Number of Participants to Develop Essential Fatty Acid Deficiency (EFAD) Defined by Holman Index > 0.4
timeFrame: Up to Day 90
Secondary outcomes:
measure: Number of Participants to Develop Parenteral Nutrition-Associated Liver Disease (PNALD)
timeFrame: Up to Day 90
measure: Alkaline Phosphatase (ALP)
timeFrame: Up to Day 90
measure: Aspartate Aminotransferase (AST)
timeFrame: Up to Day 90
measure: Alanine Aminotransferase (ALT)
timeFrame: Up to Day 90
measure: Gamma-Glutamyl Transferase (GGT)
timeFrame: Up to Day 90
measure: Total Bilirubin
timeFrame: Up to Day 90
measure: Direct Bilirubin
timeFrame: Up to Day 90
measure: Stigmasterol Blood Level
timeFrame: Up to Day 90
measure: Campesterol Blood Level
timeFrame: Up to Day 90
measure: Sitosterol Blood Level
timeFrame: Up to day 90
measure: Cholesterol Blood Level
timeFrame: Up to Day 90
measure: Squalene Blood Level
timeFrame: Up to Day 90
measure: Calories Nutritional Intake
timeFrame: Up to Day 90
measure: Protein Nutritional Intake
timeFrame: Up to Day 90
measure: Lipid Nutritional Intake
timeFrame: Up to Day 90
measure: Carbohydrates Nutritional Intake
timeFrame: Up to Day 90
measure: Change in Length or Height (and Head Circumference for Infants <1 Year of Age) From Baseline
timeFrame: Up to Day 90
measure: Body Weight
timeFrame: Up to day 90
measure: Body Weight
timeFrame: Upto Day 90
measure: Number of Adverse Events of Special Interest
timeFrame: Up to Day 120 (30 Days After Subject's Last Study Treatment if hospital discharge has not occurred)
measure: Number of Participants With Neonatal Morbidities
timeFrame: Up to Day 90
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Baxter Investigational Site
state: Alabama
city: New Haven
country: United States
facility: Baxter Investigational Site
state: Connecticut
city: Orlando
country: United States
facility: Baxter Investigational Site
state: Florida
city: Boston
country: United States
facility: Baxter Investigational Site
state: Massachusetts
city: Jackson
country: United States
facility: Baxter Investigational Site
state: Mississippi
city: Greenville
country: United States
facility: Baxter Investigational Site
state: North Carolina
city: Memphis
country: United States
facility: Baxter Investigational Site
state: Tennessee
city: San Antonio
country: United States
facility: Baxter Investigational Site
state: Texas
city: Provo
country: United States
facility: Baxter Investigational Site
state: Utah
Study officials:
affiliation: Baxter Healthcare Corporation
name: Baxter Healthcare Corporation
role: STUDY_DIRECTOR
References: