Parenteral Nutrition Associated Liver DiseaseNCT04807478
Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
An observational study of Parenteral Nutrition Associated Liver Disease and Essential Fatty Acid Deficiency, run by Fresenius Kabi.
Trial summary
This study will demonstrate Short-, mid-, and long-term 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 and older
- Conditions the study is for
- Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency, Neurocognitive Deficit
- What is being tested?
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Type of treatment
- Injection or infusion
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2022to 1 May 2029
- Phase
- Observational
- Are there results?
- None posted in the registry
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 and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male and female pediatric patients < 6 months old (corrected age) with new-onset PNAC (diagnosed within the past 21 days) 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).
- Patient is PN-dependent and within the past 21 days 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.
- Patient has feeding intolerance or at least one gastrointestinal disorder requiring PN.
- Patient is < 6 months corrected age (expected time of delivery to time of screening).
Who cannot join
- 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 known portal vein thrombosis (imaging studies are not required under this protocol).
- Patient has previously received a liver-only or liver-inclusive transplant.
- Patient has a major cardiac anomaly with hemodynamic instability.
- Patient has a major life-threatening disease (e.g., sepsis requiring high-dose vasopressors, acute respiratory distress syndrome, veno-occlusive disease, cancer).
Show all 18Show fewer
- 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 dialysis.
- Patient has a severe hemorrhagic disorder.
- Patient has an INR > 2.0.
- 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 a previous T:T ratio ≥ 0.2 or had a previous diagnosis of EFAD.
- Patient has a central nervous system anomaly (e.g., anencephaly) or any injury (e.g., grade 3 or 4 intraventricular hemorrhage, moderate to severe hypoxic ischemic encephalopathy) that will affect neurodevelopment.
- Patient has been diagnosed with or is suspected to have a genetic disorder known to be associated with neurodevelopmental impairment (e.g., trisomy 21, 18, 13).
- 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's medical care has been deemed futile by the medical team.
- Patient is enrolled in any other study with an investigational medicinal product.
About this study
Official title: A Multicenter, Prospective, Controlled, Longitudinal-Cohort Study Assessing the Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
This study will demonstrate Short-, mid-, and long-term 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 the detailed description
Prospective, controlled, longitudinal-cohort phase IV study to assess safety of Omegaven in Pediatric Patients with PNAC, in order to demonstrate short-, mid-, and long-term safety with respect to EFAD, serious bleeding events, life-threatening pleural and pericardial effusions, and neurocognitive development. Pediatric patients with PNAC receiving Omegaven as part of their routine nutritional management will be compared to those treated with another FDA-approved ILE for pediatric patients. Patients who are switched to a non-FDA-approved ILE or to Omegaven will be analyzed separately. Use of ILE will be as indicated by their treating physician, ideally for ≥ 1 year. Dosing modalities are to be taken from the pertinent prescribing information.
- Conditions
- Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency, Neurocognitive Deficit, 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
Dose, frequency and duration is a decision of the Investigator
| Study group | Role | What they receive |
|---|---|---|
| Pediatric patients | Pediatric patients with new-onset PNAC 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:
- Incidence of EFAD
- Incidence of serious bleeding events
- Incidence of life-threatening pericardial effusion events
- Incidence of life-threatening pleural effusion events
- Severity of any neurodevelopmental delays.
Measured: Through study completion, a maximum of 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2021
- Study start1 Apr 2022 (estimated)
- Primary completion1 May 2028 (estimated)
- Study completion1 May 2029 (estimated)
Why it stopped: New study protocol under development
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 DiseaseStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fresenius Kabi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04807478
- Sponsor's protocol ID
- OMEG-038-CP4
- Status last verified
- Aug 2022
- First submitted
- 10 Mar 2021
- Last updated
- 4 Aug 2022
- 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 4 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Prospective, Controlled, Longitudinal-Cohort Study Assessing the Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
- ClinicalTrials.gov ID
- NCT04807478
- Acronym / sponsor ID
- OMEG-038-CP4
- Conditions
- Parenteral Nutrition Associated Liver Disease, Essential Fatty Acid Deficiency, Neurocognitive Deficit, Malnutrition, Pediatric ALL
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: New study protocol under development
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Fresenius KabiIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2022 (estimated)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 1 May 2029 (estimated)
- First posted
- 19 Mar 2021 (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 Short-, mid-, and long-term 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
Detailed description
Prospective, controlled, longitudinal-cohort phase IV study to assess safety of Omegaven in Pediatric Patients with PNAC, in order to demonstrate short-, mid-, and long-term safety with respect to EFAD, serious bleeding events, life-threatening pleural and pericardial effusions, and neurocognitive development. Pediatric patients with PNAC receiving Omegaven as part of their routine nutritional management will be compared to those treated with another FDA-approved ILE for pediatric patients. Patients who are switched to a non-FDA-approved ILE or to Omegaven will be analyzed separately. Use of ILE will be as indicated by their treating physician, ideally for ≥ 1 year. Dosing modalities are to be taken from the pertinent prescribing information.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric patients | – | Drug: Omegaven® (fish oil triglycerides) Injectable EmulsionPediatric patients with new-onset PNAC |
Who may qualify
- Ages
- 1 day and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male and female pediatric patients < 6 months old (corrected age) with new-onset PNAC (diagnosed within the past 21 days) 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 (4 listed)
- Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
- Patient is PN-dependent and within the past 21 days 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.
- Patient has feeding intolerance or at least one gastrointestinal disorder requiring PN.
- Patient is < 6 months corrected age (expected time of delivery to time of screening).
Key exclusion criteria (18 listed)
- 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 known portal vein thrombosis (imaging studies are not required under this protocol).
- Patient has previously received a liver-only or liver-inclusive transplant.
- Patient has a major cardiac anomaly with hemodynamic instability.
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 EmulsionDose, frequency and duration is a decision of the Investigator | Drug | Pediatric patients |
Primary outcome
Incidence of:
Time frame: Through study completion, a maximum of 6 years
1. Incidence of EFAD 2. Incidence of serious bleeding events 3. Incidence of life-threatening pericardial effusion events 4. Incidence of life-threatening pleural effusion events 5. Severity of any neurodevelopmental delays.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2022.
Related trials for Parenteral Nutrition Associated Liver Disease
RecruitingObservational study
Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Parenteral Nutrition Associated Liver Disease and Essential Fatty Acid Deficiency, run by Fresenius Kabi.
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 4 inclusion and 18 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: withdrawn, as of the sponsor's last update on 4 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of: (time frame: Through study completion, a maximum of 6 years). 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 4 Aug 2022.