Clinical trial NCT03563222: Safety and Efficacy Study to Compare Smoflipid and Intralipid 20% in Pediatric Patients of 3 Months to 16 Years of Age
Official title: Prospective, Randomized (1:1), Double-Blind, Parallel-Group, Active-Controlled, Multicenter Study to Compare Safety and Efficacy of Smoflipid to Intralipid 20% in Pediatric Patients of 3 Months to 16 Years of Age Requiring Parenteral Nutrition for at Least 90 Days and up to 1 Year
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Malnutrition, Child
Keywords: Parenteral nutrition
Pediatrics
Malnutrition
Nutritional needs
Lead sponsor: Fresenius Kabi
Sponsor class: INDUSTRY
First posted: 2018-06-20
Enrollment: 1
Sex: ALL
Minimum age (years): 3
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

Brief summary: Evaluate the safety and efficacy of Smoflipid compared to standard of care lipid emulsion Intralipid 20% administered via a central vein in pediatric patients 3 months to 16 years of age who require parenteral nutrition for at least 90 days and up to 1 year.


Eligibility criteria: Inclusion Criteria:

1. Male and female patients 3 months to 16 years of age.
2. Patients who require PN for at least 5 days/week.
3. Patients who receive 60% or more of their total energy requirements as PN at enrollment and who are expected to receive 60% or more of their total energy requirements as PN for at least 90 days.
4. Written informed consent from parent(s) or legal representative(s). If possible, patient assent must also be obtained (according to local law).

Exclusion Criteria:

1. Known hypersensitivity to fish, egg, soybean, or peanut proteins, or to any of the active ingredients or excipients of Smoflipid or Intralipid 20%.
2. Hyperlipidemia or disorders of lipid metabolism characterized by hypertriglyceridemia (serum triglyceride concentration \> 250 mg/dL).
3. Inborn errors of amino acid metabolism.
4. Cardiopulmonary instability (including pulmonary edema, cardiac insufficiency, myocardial infarction, acidosis and hemodynamic instability requiring significant vasopressor support).
5. Hemophagocytic syndrome.
6. Liver enzymes (either AST, or ALT, or GGT) exceeding 5 x upper limit of normal range
7. Direct bilirubin ≥ 2.0 mg/dl
8. INR \> 2.
9. Any known hepatic condition outside of Intestinal Failure-Associated Liver Disease (IFALD) that will increase direct bilirubin ≥ 2.0 mg/dl.
10. Clinically significant abnormal levels of any serum electrolyte (sodium, potassium, magnesium, calcium, chloride, phosphate).
11. Active bloodstream infection demonstrated by positive blood culture at screening.
12. Severe renal failure including patients on renal replacement therapy.
13. Abnormal blood pH, oxygen saturation, or carbon dioxide.
14. Pregnancy or lactation.
15. Participation in another clinical study.
16. Unlikely to survive longer than 90 days.

Primary outcomes:
measure: Body Weight
timeFrame: from day 1 monthly to day 365
measure: Body Height
timeFrame: from day 1 monthly to day 365
measure: Head Circumference
timeFrame: from day 1 monthly to day 365
measure: Fatty Acid Profile in Total Plasma
timeFrame: from day 1 monthly to day 365
measure: Fatty Acid Profile in Red Blood Cell Membranes
timeFrame: from day 1 monthly to day 365
measure: Triene/Tetraene Ratio
timeFrame: from day 1 weekly to day 365
measure: Number of Patients in Each Treatment Group With Direct Bilirubin Levels > 2 mg/dL
timeFrame: from day 1 monthly to day 365
measure: Time Until Reaching Direct Bilirubin Levels > 2 mg/dL
timeFrame: from day 1 monthly to day 365
measure: Sterols in Plasma Including Phytosterols
timeFrame: from day 1 monthly to day 365
measure: Change From Baseline Triglycerides
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Urea Nitrogen
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Alanine Aminotransferase (ALT)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Aspartate Aminotransferase (AST)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Direct Bilirubin
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Total Bilirubin
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Gamma-glutamyl Transferase (GGT)
timeFrame: from day 1 weekly to day 365
measure: Change Form Baseline Alkaline Phosphatase (ALP)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Creatinine
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Electrolytes (Na, K, Mg, Cl,Ca, Phosphate)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Trace Elements (Ferritin, Zn, Se, Cu, Mn, Cr)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Glucose
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Total Protein
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline C-reactive Protein (CRP)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline White Blood Cell (WBC) Count
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Red Blood Cell (RBC) Count
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Platelet Count
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Hemoglobin
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Hematocrit
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline International Normalized Ratio (INR)
timeFrame: from day 1 weekly to day 365
measure: Change From Baseline Sterols (Beta-sitosterol, Campesterol, Stigmasterol, Brassicasterol, Ergosterol, Cholesterol, Desmosterol, Lanosterol, Beta-sitostanol, Lathosterol, Squalene)
timeFrame: from day 1 monthly to day 365
measure: Vital Signs: Blood Pressure
timeFrame: from day 1 monthly to day 365
measure: Vital Signs: Heart Rate
timeFrame: from day 1 monthly to day 365
measure: Vital Signs: Body Temperature
timeFrame: from day 1 monthly to day 365
measure: Adverse Events
timeFrame: from day 1 weekly to day 365
measure: Genetic Polymorphisms of Fatty Acid Desaturase Genes FADS1 and FADS2
timeFrame: once during treatment phase (day 1 to day 365)
Secondary outcomes:
Other outcomes:
Locations:
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
Study officials:
affiliation: University of Pittsburgh
name: Jeffrey Rudolph, MD
role: PRINCIPAL_INVESTIGATOR
References: