Clinical trial
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
Name
SMOF-028-CP4
Description
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.
Trial arms
Trial start
2019-12-18
Estimated PCD
2020-11-12
Trial end
2022-07-08
Status
Terminated
Phase
Early phase I
Treatment
Smoflipid
The study drugs will be infused via a dedicated line for parenteral nutrition (PN) into a central vein using a central venous catheter or a peripherally inserted central catheter.
The initial rate of infusion should be no more than 0.05 mL/minute for the first 10 to 15 minutes. If no untoward reactions occur, the rate can be changed to permit infusion of 0.5 mL/kg/hour.
The individual dosage of study drug should be infused at a constant rate for 10 to 24 h/d. The administration flow rate is determined by dividing the volume of study drug by the duration of the infusion. Maximum infusion rate for lipid should not exceed 0.125 g/kg/h lipid.
Study drug infusions should be given 5 to 7 days per week. Study treatment will last for a minimum of 90 consecutive days and as long as PN is indicated, up to 365 consecutive days. If the indication for PN continues after Study Day 365, PN will continue per normal institution policy.
Arms:
Smoflipid
Other names:
Smoflipid® Lipid Injectible Emulsion, USP 20%
Intralipid, 20%
The study drugs will be infused via a dedicated line for parenteral nutrition (PN) into a central vein using a central venous catheter or a peripherally inserted central catheter.
The initial rate of infusion should be no more than 0.05 mL/minute for the first 10 to 15 minutes. If no untoward reactions occur, the rate can be changed to permit infusion of 0.5 mL/kg/hour.
The individual dosage of study drug should be infused at a constant rate for 10 to 24 h/d. The administration flow rate is determined by dividing the volume of study drug by the duration of the infusion. Maximum infusion rate for lipid should not exceed 0.125 g/kg/h lipid.
Study drug infusions should be given 5 to 7 days per week. Study treatment will last for a minimum of 90 consecutive days and as long as PN is indicated, up to 365 consecutive days. If the indication for PN continues after Study Day 365, PN will continue per normal institution policy.
Arms:
Intralipid, 20%
Other names:
Intralipid® 20% (20% i.v. fat emulsion)
Size
1
Primary endpoint
Body Weight
from day 1 monthly to day 365
Body Height
from day 1 monthly to day 365
Head Circumference
from day 1 monthly to day 365
Fatty Acid Profile in Total Plasma
from day 1 monthly to day 365
Fatty Acid Profile in Red Blood Cell Membranes
from day 1 monthly to day 365
Triene/Tetraene Ratio
from day 1 weekly to day 365
Number of Patients in Each Treatment Group With Direct Bilirubin Levels > 2 mg/dL
from day 1 monthly to day 365
Time Until Reaching Direct Bilirubin Levels > 2 mg/dL
from day 1 monthly to day 365
Sterols in Plasma Including Phytosterols
from day 1 monthly to day 365
Change From Baseline Triglycerides
from day 1 weekly to day 365
Change From Baseline Urea Nitrogen
from day 1 weekly to day 365
Change From Baseline Alanine Aminotransferase (ALT)
from day 1 weekly to day 365
Change From Baseline Aspartate Aminotransferase (AST)
from day 1 weekly to day 365
Change From Baseline Direct Bilirubin
from day 1 weekly to day 365
Change From Baseline Total Bilirubin
from day 1 weekly to day 365
Change From Baseline Gamma-glutamyl Transferase (GGT)
from day 1 weekly to day 365
Change Form Baseline Alkaline Phosphatase (ALP)
from day 1 weekly to day 365
Change From Baseline Creatinine
from day 1 weekly to day 365
Change From Baseline Electrolytes (Na, K, Mg, Cl,Ca, Phosphate)
from day 1 weekly to day 365
Change From Baseline Trace Elements (Ferritin, Zn, Se, Cu, Mn, Cr)
from day 1 weekly to day 365
Change From Baseline Glucose
from day 1 weekly to day 365
Change From Baseline Total Protein
from day 1 weekly to day 365
Change From Baseline C-reactive Protein (CRP)
from day 1 weekly to day 365
Change From Baseline White Blood Cell (WBC) Count
from day 1 weekly to day 365
Change From Baseline Red Blood Cell (RBC) Count
from day 1 weekly to day 365
Change From Baseline Platelet Count
from day 1 weekly to day 365
Change From Baseline Hemoglobin
from day 1 weekly to day 365
Change From Baseline Hematocrit
from day 1 weekly to day 365
Change From Baseline International Normalized Ratio (INR)
from day 1 weekly to day 365
Change From Baseline Sterols (Beta-sitosterol, Campesterol, Stigmasterol, Brassicasterol, Ergosterol, Cholesterol, Desmosterol, Lanosterol, Beta-sitostanol, Lathosterol, Squalene)
from day 1 monthly to day 365
Vital Signs: Blood Pressure
from day 1 monthly to day 365
Vital Signs: Heart Rate
from day 1 monthly to day 365
Vital Signs: Body Temperature
from day 1 monthly to day 365
Adverse Events
from day 1 weekly to day 365
Genetic Polymorphisms of Fatty Acid Desaturase Genes FADS1 and FADS2
once during treatment phase (day 1 to day 365)
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'SUPPORTIVE_CARE', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 1, 'type': 'ACTUAL'}}
Updated at
2022-12-12
1 organization
2 products
2 indications
Organization
Fresenius KabiProduct
SmoflipidIndication
MalnutritionIndication
PediatricProduct
Intralipid