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NCT Number: NCT06049680

Safety Study of SMOFlipid to Evaluate the Risk of Developing EFAD and/or PNAC in Pediatric and Adult Patients

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.

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Key information

Conditions

Malnutrition, Child Child Nutrition Disorders Essential Fatty Acid Deficiency (EFAD) Malnutrition Nutrition Disorders Nutritional and Metabolic Diseases Parenteral Nutrition Associated Cholestasis

Age range

1 month–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Emory University Hospital, Atlanta, Georgia, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Age Limits: and Adults

Inclusion criteria

  • Male or female patients, at least 1 month of age.
  • Patients who require PN for at least 5 days/week.
  • 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.
  • 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

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

Treatment and study plan

SMOFlipid® (lipid injectable emulsion)

Drug

SMOFlipid is a sterile, nonpyrogenic, white, homogenous lipid emulsion for intravenous infusion. The lipid content of SMOFlipid is 0.20 g/mL, and comprises a mixture of soybean oil, MCT, olive oil, and fish oil. SMOFlipid belongs to the pharmacotherapeutic group: "Solutions for parenteral nutrition, fat emulsions" (ATC-code: B05BA02). SMOFlipid is indicated in adult and pediatric patients, including term and preterm neonates, as a source of calories and essential fatty acids for parenteral nutrition when oral or enteral nutrition is not possible, insufficient, or contraindicated.

Primary outcomes

  1. Incidence of PNAC

    Time frame: 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).

    defined as direct bilirubin level > 2 mg/dL, confirmed by a second sample collected 7 days after the first sample

  2. Time to direct bilirubin > 2mg/dL

    Time frame: 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).

    (confirmed by a second sample collected 7 days after the first)

  3. Incidence of EFAD

    Time frame: 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).

    Triene/tetraene ratio in total plasma to assess EFAD (Holman 1960), Severity of EFAD will be graded based on triene/tetraene ratio, as suspected ≥ 0.05, moderate ≥ 0.20 and severe ≥ 0.40 (Cober et al 2012)

  4. Incidence of clinical EFAD

    Time frame: 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).

  5. Fatty acids

    Time frame: 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).

    analyzed in total plasma, including linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid and Mead acid

Secondary outcomes

  1. Laboratory values: triglycerides

    Time frame: 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).

  2. Laboratory values: direct bilirubin

    Time frame: 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).

  3. Laboratory values: total bilirubin

    Time frame: 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).

  4. Laboratory values: alanine aminotransferase (ALT)

    Time frame: 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).

  5. Laboratory values: aspartate aminotransferase (AST)

    Time frame: 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).

  6. Laboratory values: gamma-glutamyl transferase (GGT)

    Time frame: 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).

  7. Laboratory values: alkaline phosphatase (ALP)

    Time frame: 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).

  8. Laboratory values: creatinine

    Time frame: 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).

  9. Laboratory values: urea nitrogen

    Time frame: 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).

  10. Laboratory values: electrolytes

    Time frame: 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).

  11. Laboratory values: glucose

    Time frame: 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).

  12. Laboratory values: total protein

    Time frame: 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).

  13. Laboratory values hematology: white blood cell count (WBC), red blood cell count (RBC), and platelet count

    Time frame: 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).

  14. Laboratory values hematology: Hemoglobin

    Time frame: 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).

  15. Laboratory values hematology Hematocrit

    Time frame: 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).

  16. Laboratory values hematology: International normalized ratio

    Time frame: At baseline before start of treatment to assess eligibility

  17. Vital signs: Blood pressure

    Time frame: 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).

  18. Vital signs: Heart rate

    Time frame: 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).

  19. Vital signs: Body temperature

    Time frame: 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).

  20. Incidence of adverse events

    Time frame: 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).

  21. BMI

    Time frame: 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).

    BMI is the weight in kilograms divided by height in meters squared. Alternatively, BMI can be calculated by dividing the weight in kilograms by the height in centimeters squared, and then multiplying the result by 10,000.

Study contacts

Contact information is provided by the study sponsor or research team.

Jean-Marc Lohse, PhD

CONTACT

[email protected]

+49 6172 686 ext. 4911

Martin Stumpf, PhD

CONTACT

[email protected]

+49 6172 686 ext. 8655

Sponsors and collaborators

Lead sponsor

Fresenius Kabi

Industry

Registry information

Official study 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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Sep 22, 2023
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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