Essential Fatty Acid Deficiency (EFAD)
Conditions
Keywords
Essential Fatty Acids (EFA), Parenteral Nutrition, Clinolipid/Clinoleic, Parenteral Nutrition Associated Cholestasis (PNAC), Parenteral Nutrition Associated Liver Disease (PNALD), Intestinal Failure Associated Liver Disease (IFALD), Phytosterols, Intralipid, Infants/Preterm Infants, FADS1 and FADS2, Short Bowel Syndrome, Cancer Nutrition, Olive Oil Emulsion, Soybean Oil Emulsion
Brief summary
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care for up to 90 days to develop Essential Fatty Acid Deficiency (EFAD). Additionally, this study design will evaluate the safety and efficacy of using Clinolipid in a pediatric population.
Interventions
DRUGClinolipid
DRUGIntralipid
Standard-of-Care Soybean Oil-Based Lipid Emulsion
Sponsors
Baxter Healthcare Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Patients and/or their legal representative must be able to understand the study and voluntarily sign the ICF 2. Patients age \<18 years 3. Patients who are able to adhere to protocol requirements 4. Patients who are expected to require PN for at least 7 days 5. Premature infants (\<36 weeks of gestation) require at least 80% PN to meet nutrition requirements at study entry; full term infants and children require at least 70% PN to meet nutrition requirements at study entry
Exclusion criteria
1. Patients who are not expected to survive hospitalization or with a severe illness with foreseeable intercurrent events that could jeopardize the patient's participation in the study 2. Patients with a known hypersensitivity to lipid emulsion, egg or soybean proteins, or any of the active substances, excipients, or components of the container 3. Patients with a diagnosis of shock, renal failure requiring dialysis, or severe metabolic acidosis (eg, pH \<7.10, serum bicarbonate level ≤15 mEq/L , and/or an Anion Gap \>16 mEq/L) 4. Patients with hemodynamic instability as judged by the Investigator 5. Patients with uncorrected metabolic disorders (eg, diabetes) or liver disease including cholestasis 6. Patients with severe hyperlipidemia or severe disorders of lipid metabolism characterized by hypertriglyceridemia (triglyceride \>400 mg/dL) 7. Patients who are unable to tolerate the necessary laboratory monitoring 8. Patients who have a new and active infection (as assessed by the investigator) at time of initiation of study treatment 9. Patients who are enrolled in another clinical trial involving an investigational agent 10. Patients who were treated with IV lipids within 48 hours of randomization into the study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Essential Fatty Acid Deficiency (EFAD) | Up to 90 Days | Holman Index Calculation |
Secondary
| Measure | Time frame |
|---|---|
| Genetic Polymorphisms in Fatty Acid Desaturase Genes FADS1 and FADS2 | Baseline |
| Fatty Acid Profile | Up to 90 Days |
| Weight | Up to 90 Days |
| Phytosterol, Cholesterol, and Squalene Levels | Up to 90 Days |
| Parenteral Nutrition-Associated Cholestasis (PNAC) | Up to 90 Days |
| Prescribed and Actual (Total Calories From PN and Oral) Nutritional Intake | Up to 90 Days |
| Vital Signs | Up to 90 Days |
| Adverse Events and Serious Adverse Events | Up to 30 Days After Subject's Last Study Treatment |
| Clinical Laboratory Tests | Up to 90 Days |
| Change From Baseline of Hepatic Integrity (ALP, AST, ALT, GGT, Total and Direct Bilirubin) | Up to 90 Days |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Clinolipid (Lipid Injectable Emulsion, USP) 20% Dosing schedule based on subject's age, weight, and medical condition and as per ESPEN-ESPHAN guidelines.
Clinolipid | 1 |
| Intralipid 20% (Lipid Injectable Emulsion, USP) Dosing schedule based on subject's age, weight, and medical condition and as per ESPEN-ESPHAN guidelines.
Intralipid: Standard-of-Care Soybean Oil-Based Lipid Emulsion | 0 |
| Total | 1 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Based on feedback from FDA | 1 | 0 |
Baseline characteristics
| Characteristic | Clinolipid (Lipid Injectable Emulsion, USP) 20% | Intralipid 20% (Lipid Injectable Emulsion, USP) | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 1 Participants | 0 Participants | 1 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 1 | 0 / 0 |
| other Total, other adverse events | 0 / 1 | 0 / 0 |
| serious Total, serious adverse events | 0 / 1 | 0 / 0 |
Outcome results
Primary
Essential Fatty Acid Deficiency (EFAD)
Holman Index Calculation
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Adverse Events and Serious Adverse Events
Time frame: Up to 30 Days After Subject's Last Study Treatment
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Change From Baseline of Hepatic Integrity (ALP, AST, ALT, GGT, Total and Direct Bilirubin)
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Clinical Laboratory Tests
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Fatty Acid Profile
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Genetic Polymorphisms in Fatty Acid Desaturase Genes FADS1 and FADS2
Time frame: Baseline
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Parenteral Nutrition-Associated Cholestasis (PNAC)
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Phytosterol, Cholesterol, and Squalene Levels
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Prescribed and Actual (Total Calories From PN and Oral) Nutritional Intake
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Vital Signs
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.
Secondary
Weight
Time frame: Up to 90 Days
Population: Due to early termination of the study, no formal analysis was conducted.