Evaluating the safety of SMOFlipid in preventing malnutrition-related complications

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

This study is testing if SMOFlipid, a special fat mixture, can help both kids and adults who need long-term feeding through a vein avoid serious nutrition-related problems.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 1 Month to 17 Years
• Sex: All
• Sponsor: Fresenius Kabi (Industry-sponsored)
• Locations: 5 sites (Atlanta, Georgia and 4 other locations)
• Trial ID: NCT06049680 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety of SMOFlipid, a lipid injectable emulsion, in both pediatric and adult patients who require long-term parenteral nutrition. Participants must be at least 1 month old and expected to receive parenteral nutrition for at least 56 days. The study aims to assess the risk of developing essential fatty acid deficiency (EFAD) and parenteral nutrition-associated cholestasis (PNAC) during treatment. By monitoring these patients, the study seeks to provide insights into the effectiveness of SMOFlipid in preventing these complications.

Who should consider this trial

Good fit: Ideal candidates include male and female patients aged 1 month and older who need parenteral nutrition for at least 56 days.

Not a fit: Patients with known hypersensitivity to the components of SMOFlipid or those with certain lipid metabolism disorders may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could help improve nutritional outcomes and reduce complications in patients requiring long-term parenteral nutrition.

How similar studies have performed: While this approach is focused on a specific lipid emulsion, similar studies have shown promise in improving outcomes for patients on parenteral nutrition.

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.

Where this trial is running

Atlanta, Georgia and 4 other locations

• Emory University Hospital — Atlanta, Georgia, United States (Recruiting)
• The University of Chicago — Chicago, Illinois, United States (Not_yet_recruiting)
• Riley Hospital for Children — Indianapolis, Indiana, United States (Recruiting)
• Nationwide Children's Hospital — Columbus, Ohio, United States (Not_yet_recruiting)
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States (Recruiting)

Study contacts

• Principal investigator: Jeffrey Rudolph, MD — University of Pittsburgh
• Study coordinator: Martin Stumpf, PhD

Conditions Malnutrition, Child; Malnutrition; Essential Fatty Acid Deficiency; Parenteral Nutrition Associated Cholestasis