Malnutrition, ChildPhase 4NCT06049680
Safety Study of SMOFlipid to Evaluate the Risk of Developing EFAD and/or PNAC in Pediatric and Adult Patients
A Phase 4 study testing SMOFlipid® (lipid injectable emulsion) in people with Malnutrition, Child and Malnutrition, run by Fresenius Kabi at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 month to 17 years
- Conditions the study is for
- Malnutrition, Child, Malnutrition, Essential Fatty Acid Deficiency (EFAD)
- What is being tested?
- SMOFlipid® (lipid injectable emulsion)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 6 sitesUnited States
- Study dates
- 28 Oct 2024to Jan 2027
- Phase
- Phase 4
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- 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.
Show all 17Show fewer
- 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.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 5 recruiting
- Recruiting
Emory University Hospital
Atlanta, Georgia, 30322
Thomas R. Ziegler, M.D., 404-727-7351, tzieg01@emory.edu
- Terminated
The University of Chicago
Chicago, Illinois, 60638
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
Ethan Mezoff, Dr., 614 722 2286, ethan.mezoff@nationwidechildrens.org
- Not yet recruiting
Duke University Medical Center
Durham, North Carolina, 27710
Arun Ajmera, Dr., 919-684-5068, arun.ajmera@duke.edu
- Recruiting
Riley Hospital for Children
Indianapolis, Indiana, 46202
Charles Vanderpool, MD, 317-944-2078, chavande@iupui.edu
- Recruiting
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Jeffrey Alan Rudolph, MD, 7248144905, jeffrey.rudolph@chp.edu
About this study
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
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
- Being tested
- SMOFlipid® (lipid injectable emulsion)
- Medical subject headings
- Child Nutrition Disorders, Malnutrition
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| Single arm SMOFlipid® (lipid injectable emulsion) | Other | Investigational drug: SMOFlipid® (lipid injectable emulsion). Receives: Drug: SMOFlipid® (lipid injectable emulsion) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of PNAC
defined as direct bilirubin level > 2 mg/dL, confirmed by a second sample collected 7 days after the first sample
Measured: 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)
Measured: 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)
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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)
Measured: 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: gamma-glutamyl transferase (GGT)
Measured: 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: alkaline phosphatase (ALP)
Measured: 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: creatinine
Measured: 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: urea nitrogen
Measured: 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: electrolytes
Measured: 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: glucose
Measured: 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 protein
Measured: 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 hematology: white blood cell count (WBC), red blood cell count (RBC), and platelet count
Measured: 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 hematology: Hemoglobin
Measured: 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 hematology Hematocrit
Measured: 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 hematology: International normalized ratio
Measured: At baseline before start of treatment to assess eligibility
Vital signs: Blood pressure
Measured: 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).
Vital signs: Heart rate
Measured: 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).
Vital signs: Body temperature
Measured: 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 adverse events
Measured: 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).
BMI
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.
Measured: 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).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2023
- Study start28 Oct 2024
- Primary completionNov 2026 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Malnutrition, ChildStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fresenius Kabi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jeffrey Rudolph, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06049680
- Sponsor's protocol ID
- SMOF-044-CP4 (PMR 4240-1)
- Status last verified
- Oct 2026
- First submitted
- 24 Jul 2023
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No