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

Primary Purpose

Malnutrition, Child, Malnutrition

Status
Not yet recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
SMOFlipid® (lipid injectable emulsion)
Sponsored by
Fresenius Kabi
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Malnutrition, Child

Eligibility Criteria

1 Month - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital of Pittsburgh of UPMC

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Single arm SMOFlipid® (lipid injectable emulsion)

Arm Description

Investigational drug: SMOFlipid® (lipid injectable emulsion).

Outcomes

Primary Outcome Measures

Incidence of PNAC
defined as direct bilirubin level > 2 mg/dL, confirmed by a second sample collected 7 days after the first sample
Time to direct bilirubin > 2mg/dL
(confirmed by a second sample collected 7 days after the first)
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)
Incidence of clinical EFAD
Fatty acids
analyzed in total plasma, including linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid and Mead acid

Secondary Outcome Measures

Laboratory values: triglycerides
Laboratory values: direct bilirubin
Laboratory values: total bilirubin
Laboratory values: alanine aminotransferase (ALT)
Laboratory values: aspartate aminotransferase (AST)
Laboratory values: gamma-glutamyl transferase (GGT)
Laboratory values: alkaline phosphatase (ALP)
Laboratory values: creatinine
Laboratory values: urea nitrogen
Laboratory values: electrolytes
Laboratory values: glucose
Laboratory values: total protein
Laboratory values hematology: white blood cell count (WBC), red blood cell count (RBC), and platelet count
Laboratory values hematology: Hemoglobin
Laboratory values hematology Hematocrit
Laboratory values hematology: International normalized ratio
Vital signs: Blood pressure
Vital signs: Heart rate
Vital signs: Body temperature
Incidence of adverse events
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.

Full Information

First Posted
July 24, 2023
Last Updated
September 19, 2023
Sponsor
Fresenius Kabi
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1. Study Identification

Unique Protocol Identification Number
NCT06049680
Brief Title
Safety Study of SMOFlipid to Evaluate the Risk of Developing EFAD and/or PNAC in Pediatric and Adult Patients
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
September 2023 (Anticipated)
Primary Completion Date
March 2026 (Anticipated)
Study Completion Date
September 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Fresenius Kabi

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Malnutrition, Child, Malnutrition

7. Study Design

Primary Purpose
Other
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Single arm SMOFlipid® (lipid injectable emulsion)
Arm Type
Other
Arm Description
Investigational drug: SMOFlipid® (lipid injectable emulsion).
Intervention Type
Drug
Intervention Name(s)
SMOFlipid® (lipid injectable emulsion)
Intervention Description
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 Outcome Measure Information:
Title
Incidence of PNAC
Description
defined as direct bilirubin level > 2 mg/dL, confirmed by a second sample collected 7 days after the first sample
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).
Title
Time to direct bilirubin > 2mg/dL
Description
(confirmed by a second sample collected 7 days after the first)
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).
Title
Incidence of EFAD
Description
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)
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).
Title
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).
Title
Fatty acids
Description
analyzed in total plasma, including linoleic acid, α-linolenic acid, arachidonic acid, docosahexaenoic acid, eicosapentaenoic acid and Mead acid
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).
Secondary Outcome Measure Information:
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
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).
Title
Laboratory values hematology: International normalized ratio
Time Frame
At baseline before start of treatment to assess eligibility
Title
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).
Title
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).
Title
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).
Title
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).
Title
BMI
Description
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.
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. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Cornelia Schneider-Schranz, PhD
Phone
+49 6172 608
Ext
96024
Email
Cornelia.Schneider-Schranz@fresenius-kabi.com
First Name & Middle Initial & Last Name or Official Title & Degree
Jean-Marc Lohse, PhD
Phone
+49 6172 686
Ext
4911
Email
Jean-Marc.Lohse@fresenius-kabi.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jeffrey Rudolph, MD
Organizational Affiliation
University of Pittsburgh
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jeffrey Alan Rudolph, MD
Phone
724-814-4905
Email
jeffrey.rudolph@chp.edu

12. IPD Sharing Statement

Learn more about this trial

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

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