Clinical trial NCT06049680: Safety Study of SMOFlipid to Evaluate the Risk of Developing EFAD and/or PNAC in Pediatric and Adult Patients
Official title: A Single-arm Open-label Safety Study of SMOFlipid to Evaluate the Risk of Developing Essential Fatty Acid Deficiency (EFAD) and/or Parenteral Nutrition-associated Cholestasis (PNAC) in Pediatric Patients 1 Month to 17 Years of Age and in Adult Patients, Who Are Anticipated to Need 8 Weeks or Longer of Parenteral Nutrition Treatment
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Malnutrition, Child
Malnutrition
Essential Fatty Acid Deficiency (EFAD)
Parenteral Nutrition Associated Cholestasis
Lead sponsor: Fresenius Kabi
Sponsor class: INDUSTRY
First posted: 2023-09-22
Enrollment: 100
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: Evaluate the risk of developing EFAD and/or PNAC in adult and pediatric patients 1 month of age and older, who are anticipated to need 8 weeks or longer of parenteral nutrition treatment with SMOFlipid.


Eligibility criteria: Age Limits: and Adults

Inclusion Criteria:

1. Male or female patients, at least 1 month of age.
2. Patients who require PN for at least 5 days/week.
3. Patients who receive 80% or more of their total energy requirements as PN at enrollment and who are expected to receive 80% or more of their total energy requirements as PN for at least 56 days.
4. Written informed consent. In case of pediatric patients, informed consent must be obtained from parent(s) or legal representatives. If possible, the assent of the pediatric patient must also be obtained (according to local law).

Exclusion Criteria:

1. Use of any other lipid injectable emulsion than SMOFlipid within 6 months prior to study participation
2. Known hypersensitivity to fish, egg, soybean, or peanut proteins, or to any of the active ingredients or excipients of SMOFlipid.
3. Hyperlipidemia or disorders of lipid metabolism characterized by hypertriglyceridemia (serum triglyceride concentration \>250 mg/dL in infants or \>400 mg/dL in older pediatric and adult patients).
4. Inborn errors of amino acid metabolism.
5. Cardiopulmonary instability (including pulmonary edema, cardiac insufficiency, myocardial infarction, acidosis and hemodynamic instability requiring significant vasopressor support).
6. Hemophagocytic syndrome.
7. Liver enzymes (either AST, or ALT, or GGT) exceeding 2 x upper limit of normal range
8. Direct bilirubin exceeding 2 x upper limit of normal range
9. INR exceeding 2 x upper limit of normal range and patient not receiving oral anticoagulants.
10. Any known hepatic condition outside of IFALD that will increase direct bilirubin ≥2.0 mg/dL.
11. Clinically significant abnormal levels of any serum electrolyte (sodium, potassium, magnesium, calcium, chloride, phosphate).
12. Active bloodstream infection demonstrated by positive blood culture at screening.
13. Severe renal failure (eGFR \<15 ml/min per 1.73 m2) including patients on renal replacement therapy.
14. Abnormal blood pH, oxygen saturation, or carbon dioxide.
15. Pregnancy or lactation.
16. Participation in another interventional clinical study.
17. Unlikely to survive longer than 56 days.

Primary outcomes:
measure: Incidence of PNAC
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Time to direct bilirubin > 2mg/dL
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Incidence of EFAD
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Incidence of clinical EFAD
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Fatty acids
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
Secondary outcomes:
measure: Laboratory values: triglycerides
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: direct bilirubin
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: total bilirubin
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: alanine aminotransferase (ALT)
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: aspartate aminotransferase (AST)
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: gamma-glutamyl transferase (GGT)
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: alkaline phosphatase (ALP)
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: creatinine
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: urea nitrogen
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: electrolytes
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: glucose
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values: total protein
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values hematology: white blood cell count (WBC), red blood cell count (RBC), and platelet count
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values hematology: Hemoglobin
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values hematology Hematocrit
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Laboratory values hematology: International normalized ratio
timeFrame: At baseline before start of treatment to assess eligibility
measure: Vital signs: Blood pressure
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Vital signs: Heart rate
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Vital signs: Body temperature
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: Incidence of adverse events
timeFrame: Start of Treatment until After End of Last Study PN (+6 months for pediatric patients). Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
measure: BMI
timeFrame: Start of Treatment until After End of Last Study PN Duration of Treatment: Study treatment will last for a minimum of 8 weeks (56 consecutive days) and as long as PN is indicated, up to 1 year (365 consecutive days).
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University Hospital
state: Georgia
city: Chicago
country: United States
facility: The University of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
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: