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

NCT06049680 | Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: SMOF-044-CP4 (PMR 4240-1)
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 5

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.

Conditions

Malnutrition, Child; Malnutrition; Essential Fatty Acid Deficiency (EFAD); Parenteral Nutrition Associated Cholestasis

Outcome Measures

Primary Outcomes (5)

• Incidence of PNAC

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

  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).

• Time to direct bilirubin > 2mg/dL

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

  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).

• Incidence of EFAD

  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)

  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).

• Incidence of clinical EFAD

  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).

• Fatty acids

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

  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 (21)

• Laboratory values: triglycerides

  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).

• Laboratory values: direct bilirubin

  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).

• Laboratory values: total bilirubin

  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).

• Laboratory values: alanine aminotransferase (ALT)

  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).

• Laboratory values: aspartate aminotransferase (AST)

  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).

Study Arms (1)

Single arm SMOFlipid® (lipid injectable emulsion)

OTHER

Investigational drug: SMOFlipid® (lipid injectable emulsion).

Drug: SMOFlipid® (lipid injectable emulsion)

Interventions

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.

Single arm SMOFlipid® (lipid injectable emulsion)

Eligibility Criteria

• Age: 1 Month - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Fresenius Kabi: lead

Study Sites (5)

Emory University Hospital

Atlanta, Georgia, 30322, United States

RECRUITING

The University of Chicago

Chicago, Illinois, 60638, United States

NOT YET RECRUITING

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

NOT YET RECRUITING

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

MeSH Terms

Conditions

Child Nutrition Disorders; Malnutrition

Interventions

SMOFlipid

Condition Hierarchy (Ancestors)

Nutrition Disorders; Nutritional and Metabolic Diseases

Study Officials

• Jeffrey Rudolph, MD

  University of Pittsburgh

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Martin Stumpf, PhD

CONTACT

+49 6172 686; martin.stumpf@fresenius-kabi.com

Jean-Marc Lohse, PhD

CONTACT

+49 6172 686; Jean-Marc.Lohse@fresenius-kabi.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2023
• First Posted: September 22, 2023
• Study Start: October 28, 2024
• Primary Completion: March 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: December 4, 2025

Record last verified: 2025-11

Locations

US (5)