Parenteral Nutrition Associated Liver Disease (PNALD)NCT06274788
Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
An observational study of Parenteral Nutrition Associated Liver Disease (PNALD) and Essential Fatty Acid Deficiency, run by Fresenius Kabi at 10 sites in United States.
Trial summary
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population Read more about this study
- Who is it for?
- Ages 1 day to 17 years
- Conditions the study is for
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition
- What is being tested?
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Type of treatment
- Injection or infusion
- How many people
- 40 participants
- Where
- 10 sitesUnited States
- Study dates
- 15 Dec 2024to 1 Apr 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 day to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Male and female pediatric patients with PNAC and expected requirement of Omegaven for at least 8 weeks will be enrolled at multiple sites; PNAC is defined as being PN-dependent with a DBil level ≥ 2.0 mg/dL and no other known cause of liver dysfunction.
Who may be able to join
- Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
- Pediatric patient (<18 years) has been diagnosed with PNAC, defined as direct or conjugated bilirubin (DBil) ≥ 2.0 mg/dL with no other known cause of liver dysfunction at the time of enrollment and is expected to require Omegaven treatment for at least eight weeks.
- Patient has oral or enteral feeding intolerance or at least one gastrointestinal disorder requiring PN.
Who cannot join
- Patient has received Omegaven within four weeks before inclusion in the study
- Patient has any other known cause of chronic liver disease such as hepatitis C, cystic fibrosis, biliary atresia, alpha-1-antitrypsin deficiency, passive hepatic congestion due to heart failure, etc.
- Patient has known cirrhosis (liver biopsy is not required under this protocol).
- Patient has been previously diagnosed with, or has prior evidence of, portal vein thrombosis.
- Patient has previously received a liver-only or liver-inclusive transplant.
- Patient has hemodynamic instability due to any major cardiac anomaly.
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- Patient has a major life-threatening disease (e.g., sepsis requiring high-dose vasopressors, acute respiratory distress syndrome, veno-occlusive disease, cancer).
- Patient has multi-organ failure, septic shock, hypotension requiring pressor therapy, persistent pulmonary hypertension requiring inhaled nitric oxides, or requires extracorporeal membrane oxygenation (ECMO) or similar intervention.
- Patient has renal failure and requires renal replacement therapy.
- Patient has a severe hemorrhagic disorder.
- Patient has severe hyperlipidemia or a severe disorder of lipid metabolism characterized by hypertriglyceridemia (i.e., serum triglyceride level > 1,000 mg/dL).
- Patient has a record of EFAD before inclusion in the study
- Patient has been diagnosed with or is suspected to have an inborn error of metabolism.
- Patient has a known hypersensitivity to fish or egg protein or to any of the active ingredients or excipients of Omegaven.
- Patient is subject to treatment limitation.
- Patient is enrolled in any other study with an investigational medicinal product during the course of the current study.
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
United States10 sites, 10 recruiting
- Recruiting
Children's Hospital Corporation d/b/a Boston Children's Hospital
Boston, Massachusetts, 02115
- Recruiting
The University of Chicago
Chicago, Illinois, 60637
- Recruiting
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
- Recruiting
Memorial Health Service
Fountain Valley, California, 92708
- Recruiting
Baylor College of Medicine Houston
Houston, Texas, 77030
- Recruiting
University of California Los Angeles
Los Angeles, California, 10911
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
- Recruiting
Board of Regents of the University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
- Recruiting
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229-3901
- Recruiting
Seattle Children's Hospital d/b/a Seattle Children's Research Institute
Seattle, Washington, 98105
About this study
Official title: Title: A Single-arm, Open-label, Prospective, Multicenter Safety Study to Evaluate the Occurrence of Essential Fatty Acid Deficiency (EFAD) in Pediatric Patients With Parenteral Nutrition-associated Cholestasis (PNAC) Who Require More Than Eight Weeks of Omegaven Treatment
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
- Conditions
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition, Pediatric ALL
- Being tested
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Medical subject headings
- Malnutrition, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Omegaven® (fish oil triglycerides) Injectable EmulsionDrug
Pediatric patients Pediatric patients with new-onset PNAC
Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion Dose, frequency and duration is a decision of the Investigator
| Study group | Role | What they receive |
|---|---|---|
| Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion) | The dose of investigational drug (study treatment), as well as all other components of the overall nutritional regimen is solely at the discretion of the Investigator. It is assumed the Investigator will use sound medical judgement, follow institutional standards of care regarding the nutrition provided to each patient, and review applicable prescribing information indicating the maximum and recommended dose of Omegaven of 1 g/kg/day infused intravenously over 8 to 24 hours as long as the infusion rate does not exceed 1.5 mL/kg/hour. Receives: Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of essential fatty acid deficiency (EFAD)
EFAD as defined by the triene:tetraene (T:T) ratio; severity of EFAD will be graded and analysed based on T:T ratio (suspected ≥ 0.05, moderate ≥ 0.20, and severe ≥ 0.40)
Measured: Up to week 56
Secondary outcomes (15)
Incidence of clinical EFAD
Clinical EFAD is defined by clinical symptoms (like dry, scaly rash, hair loss, hair depigmentation, poor wound healing, growth restriction, and increased susceptibility to infection) and T:T ratio ≥ 0.083
Measured: Up to week 56
Time from treatment start to EFAD diagnosis
Time from treatment start to EFAD diagnosis
Measured: Up to week 56
Incidence of adverse events (AEs)/serious adverse events (SAEs)
The incidence of AEs/SAEs considered by the Investigator to be related to study treatment
Measured: Up to week 56
Routine laboratory tests: Direct or conjugated bilirubin
Measured: Up to week 56
Routine laboratory tests: triglycerides
Measured: Up to week 56
Fatty acid profiles: α-linolenic acid
Measured: Up to week 56
Fatty acid profiles: linoleic acid
Measured: Up to week 56
Fatty acid profiles: arachidonic acid
Measured: Up to week 56
Fatty acid profiles: mead acid
Measured: Up to week 56
Fatty acid profiles: oleic acid
Measured: Up to week 56
Fatty acid profiles: docosahexaenoic acid (DHA)
Measured: Up to week 56
Fatty acid profiles: eicosapentaenoic acid (EPA)
Measured: Up to week 56
Anthropometric measures: body weight
Measured: Up to week 56
Anthropometric measures: body height/length
Measured: Up to week 56
Anthropometric measures: head circumference
Measured: Up to week 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2024
- Study start15 Dec 2024
- Primary completion1 Apr 2028 (estimated)
- Study completion1 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parenteral Nutrition Associated Liver Disease (PNALD)Studies 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fresenius Kabi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06274788
- Sponsor's protocol ID
- OMEG-054-CP4
- Status last verified
- Aug 2026
- First submitted
- 26 Jan 2024
- Last updated
- 14 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Title: A Single-arm, Open-label, Prospective, Multicenter Safety Study to Evaluate the Occurrence of Essential Fatty Acid Deficiency (EFAD) in Pediatric Patients With Parenteral Nutrition-associated Cholestasis (PNAC) Who Require More Than Eight Weeks of Omegaven Treatment
- ClinicalTrials.gov ID
- NCT06274788
- Acronym / sponsor ID
- OMEG-054-CP4
- Conditions
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition, Pediatric ALL
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Fresenius KabiIndustry (pharma, biotech, devices)
- Study start
- 15 Dec 2024 (actual)
- Primary completion
- 1 Apr 2028 (estimated)
- Study completion
- 1 Apr 2028 (estimated)
- First posted
- 23 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion) | – | Drug: Omegaven® (fish oil triglycerides) Injectable EmulsionThe dose of investigational drug (study treatment), as well as all other components of the overall nutritional regimen is solely at the discretion of the Investigator. It is assumed the Investigator will use sound medical judgement, follow institutional standards of care regarding the nutrition provided to each patient, and review applicable prescribing information indicating the maximum and recommended dose of Omegaven of 1 g/kg/day infused intravenously over 8 to 24 hours as long as the infusion rate does not exceed 1.5 mL/kg/hour. |
Who may qualify
- Ages
- 1 day to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Male and female pediatric patients with PNAC and expected requirement of Omegaven for at least 8 weeks will be enrolled at multiple sites; PNAC is defined as being PN-dependent with a DBil level ≥ 2.0 mg/dL and no other known cause of liver dysfunction.
Key inclusion criteria (3 listed)
- Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
- Pediatric patient (<18 years) has been diagnosed with PNAC, defined as direct or conjugated bilirubin (DBil) ≥ 2.0 mg/dL with no other known cause of liver dysfunction at the time of enrollment and is expected to require Omegaven treatment for at least eight weeks.
- Patient has oral or enteral feeding intolerance or at least one gastrointestinal disorder requiring PN.
Key exclusion criteria (16 listed)
- Patient has received Omegaven within four weeks before inclusion in the study
- Patient has any other known cause of chronic liver disease such as hepatitis C, cystic fibrosis, biliary atresia, alpha-1-antitrypsin deficiency, passive hepatic congestion due to heart failure, etc.
- Patient has known cirrhosis (liver biopsy is not required under this protocol).
- Patient has been previously diagnosed with, or has prior evidence of, portal vein thrombosis.
- Patient has previously received a liver-only or liver-inclusive transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Omegaven® (fish oil triglycerides) Injectable EmulsionPediatric patients Pediatric patients with new-onset PNAC Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion Dose, frequency and duration is a decision of the Investigator | Drug | Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion) |
Primary outcome
Incidence of essential fatty acid deficiency (EFAD)
Time frame: Up to week 56
EFAD as defined by the triene:tetraene (T:T) ratio; severity of EFAD will be graded and analysed based on T:T ratio (suspected ≥ 0.05, moderate ≥ 0.20, and severe ≥ 0.40)
15 secondary outcomes
Incidence of clinical EFAD
Time frame: Up to week 56
Time from treatment start to EFAD diagnosis
Time frame: Up to week 56
Incidence of adverse events (AEs)/serious adverse events (SAEs)
Time frame: Up to week 56
Routine laboratory tests: Direct or conjugated bilirubin
Time frame: Up to week 56
Routine laboratory tests: triglycerides
Time frame: Up to week 56
Fatty acid profiles: α-linolenic acid
Time frame: Up to week 56
Fatty acid profiles: linoleic acid
Time frame: Up to week 56
Fatty acid profiles: arachidonic acid
Time frame: Up to week 56
Fatty acid profiles: mead acid
Time frame: Up to week 56
Fatty acid profiles: oleic acid
Time frame: Up to week 56
Fatty acid profiles: docosahexaenoic acid (DHA)
Time frame: Up to week 56
Fatty acid profiles: eicosapentaenoic acid (EPA)
Time frame: Up to week 56
Anthropometric measures: body weight
Time frame: Up to week 56
Anthropometric measures: body height/length
Time frame: Up to week 56
Anthropometric measures: head circumference
Time frame: Up to week 56
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2026.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Parenteral Nutrition Associated Liver Disease (PNALD) and Essential Fatty Acid Deficiency, run by Fresenius Kabi at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Niess Ulf, PhD (M +49 173 5439924, ulf.niess@fresenius-kabi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of essential fatty acid deficiency (EFAD) (time frame: Up to week 56). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2026.