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

Fresenius Kabi

Status and phase: Enrolling; Phase 4

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

Treatments: Drug: SMOFlipid® (lipid injectable emulsion)

Study type: Interventional

Funder types: Industry

Identifiers

NCT06049680

SMOF-044-CP4 (PMR 4240-1)

Details and patient eligibility

About

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.

Enrollment: 100 estimated patients

Sex: All

Ages: 1 month to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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100 participants in 1 patient group

Single arm SMOFlipid® (lipid injectable emulsion)

Other group

Description:

Investigational drug: SMOFlipid® (lipid injectable emulsion).

Treatment:

Drug: SMOFlipid® (lipid injectable emulsion)