CholestasisPhase 1/2NCT02721277
SMOFlipid to Lessen the Severity of Neonatal Cholestasis
A Phase 1/2 study testing SMOFlipid in people with Cholestasis and Jaundice, Obstructive, run by University of Florida at 1 site in United States.
Trial summary
Parenteral nutrition (PN) provides intravenous nutritional supplementation for infants unable to absorb adequate enteral nutrients secondary to insufficient intestinal length or function. Read more about this study
- Who is it for?
- Ages 23 weeks to 1 yearHealthy volunteers can take part
- Conditions the study is for
- Cholestasis, Jaundice, Obstructive
- What is being tested?
- SMOFlipid
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- May 2016to 2 Feb 2017
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 23 weeks to 1 year
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Greater than 14 days old and less than 1 year of age.
- Greater than 1 kg.
- Mild cholestasis, defined as a conjugated bilirubin between 0.5-1.9 mg/dL
- Currently standard therapy with soy-based Intralipid
- Evidence of growth of weight, head circumference or length below our standards for post-menstrual age for at least 1 week.
- Be expected to require intravenous nutrition for at least an additional 21 days
Who cannot join
- Have a congenitally lethal condition.
- Have clinically severe bleeding or clinical liver failure not able to be managed with routine measures.
- Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis.
- Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Compassionate Use of an Intravenous Fat Emulsion Comprised of Soy Oil, Medium Chain Triglycerides, Olive Oil, and Fish Oil for Neonates With Parenteral Nutrition Induced Liver Injury
Parenteral nutrition (PN) provides intravenous nutritional supplementation for infants unable to absorb adequate enteral nutrients secondary to insufficient intestinal length or function. In early PN-associated cholestasis, the dose of traditional soy based lipid is limited to 1 g/kg/day which often limits the growth capacity of parenteral nutrition-dependent infants. Inadequate growth is directly related to poor neurological outcomes, failure to facilitate mechanical ventilation, and less growth of the neonate's already damaged intestine. Ultimately, these outcomes can lead to severe disability and death. To mitigate these deleterious effects and optimize growth, parenteral nutrition-dependent infants with cholestasis who are not adequately growing on 1 g/kg/day of soy-based lipid emulsion must have a greater intake of lipids to meet their needs for weight, length, and head circumference growth.
SMOFlipid contains a mixture of 4 different lipid sources: soybean oil which provides essential fatty acids, olive oil which is high in monounsaturated fatty acids that are less susceptible to lipid peroxidation than polyunsaturated fatty acids, medium-chain triglycerides which show a faster metabolic clearance than long-chain triglycerides, and fish oil which provides the supply of omega-3 fatty acids. The utility of Omegaven and soy-based lipid emulsion is limited as these are restricted to 1 g/kg/day in cholestatic infants. SMOFlipid is safe to be provided at the usual goal infusion amount of 3 g/kg/day. Because this product includes both omega-6 and omega-3 lipids, it provides the benefits of the omega-3s for the liver and provides more than enough omega-6s to meet essential fatty acid requirements. Its use in situations in which growth is inadequate in babies who must be restricted to 1 g/kg/day can be expected to improve their growth and likely markedly increase their chances of both a good neurological outcome and survival.
The aim of this research study is to determine if the unique formulation of SMOFLipid will cause less hepatic inflammation compared to soy only intralipids.
Read the detailed description
The neonates who are being treated at the University of Florida Health Neonates Intensive Care Unit and are anticipated to need more than 21 days of intravenous nutrition will be considered as potential subjects. These subjects will receive the unique formulation of SMOFLipid
The following data will be collected from the subject's EMR by members of the research team:
- Lab values that evaluate liver function
- Growth parameters like head circumference, length, weight
- Medical/surgical history
- Time to resolution of bilirubin, time to liver transplant, time to death
- Length of hospital stay
- Rates of blood infections
Subjects will remain on SMOFlipid until weaned from PN.
- Conditions
- Cholestasis, Jaundice, Obstructive
- Being tested
- SMOFlipid
- Keywords
- Parenteral nutrition, Total, Infant, newborn, Cholestasis, Enterocolitis, Necrotizing
- Medical subject headings
- Cholestasis, Jaundice, Obstructive, Hyperphagia, Enterocolitis, Necrotizing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SMOFlipidDrug
Also called Parenteral nutrition (PN)
SMOFlipid contains a mixture of 4 different lipid sources: soybean oil which provides essential fatty acids, olive oil which is high in monounsaturated fatty acids that are less susceptible to lipid peroxidation than polyunsaturated fatty acids, medium-chain triglycerides which show a faster metabolic clearance than long-chain triglycerides, and fish oil which provides the supply of omega-3 fatty acids.
| Study group | Role | What they receive |
|---|---|---|
| SMOFlipid | Experimental | Subjects with cholestasis will receive 3 G/kg/day of intravenous SMOFlipid daily until parenteral nutrition (PN) is discontinued. In addition, the following monitoring for effects of SMOFlipid will be performed: total days of parenteral nutrition, maximum conjugated bilirubin, time to resolution of bilirubin, time to liver transplant, time to death, positive blood cultures, rates of increase in weight, length, and head circumference, time dependent changes in liver function tests, including triglycerides, and length of hospital stay. Receives: Drug: SMOFlipid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Inflammation of the Liver Between the Groups
Inflammation of the liver will be evaluated by comparing direct bilirubin values between the two groups.
Measured: 6 months
Secondary outcomes (18)
Measurement of Head Circumference for Growth Increase
Growth increase will be measured by head circumference of participants.
Measured: 6 months
Measurement of Weight for Growth Increase
Growth increase will be measured by weight of participants.
Measured: 6 months
Measurement of Length for Growth Increase
Growth increase will be measured by length of participants.
Measured: 6 months
Number of Subjects Requiring Surgery
Measured: 6 months
Number of Concomitant Medications Received
Measured: 6 months
Length of IV Nutritional Therapy
Measured: 6 months
Number of Subjects Receiving Formula Diet
Enteral administration of formula will be noted
Measured: 6 months
Number of Subjects Receiving Breast Milk Diet
Enteral administration of breast milk will be noted
Measured: 6 months
Number of Participants With Adverse Events Related to Treatment
Laboratory values will be used to determine adverse events.
Measured: 6 months
Carbon Dioxide Total
Laboratory value that determines acid-base balance
Measured: 6 months
Total Protein
Laboratory value that evaluates liver function
Measured: 6 months
Albumin
Laboratory value that evaluates liver function
Measured: 6 months
Aspartate Aminotransferase
Laboratory value that evaluates liver function
Measured: 6 months
Alanine Aminotransferase
Laboratory value that evaluates liver function
Measured: 6 months
Total Bilirubin
Laboratory value that evaluates liver function
Measured: 6 months
Serum Glucose
Laboratory values that evaluates glucose in the blood
Measured: 6 months
Alkaline Phosphatase
Laboratory value that evaluates liver function
Measured: 6 months
Triglyceride
Laboratory value that evaluates liver function and metabolism of fat
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2016 (estimated)
- Study startMay 2016
- Primary completionDec 2016
- Study completion2 Feb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for CholestasisStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Josef Neu, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02721277
- Sponsor's protocol ID
- IRB201600068
- Status last verified
- Mar 2017
- First submitted
- 23 Feb 2016
- Last updated
- 17 Apr 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Use of an Intravenous Fat Emulsion Comprised of Soy Oil, Medium Chain Triglycerides, Olive Oil, and Fish Oil for Neonates With Parenteral Nutrition Induced Liver Injury
- ClinicalTrials.gov ID
- NCT02721277
- Acronym / sponsor ID
- IRB201600068
- Conditions
- Cholestasis, Jaundice, Obstructive
- Keywords
- Parenteral nutrition, Total, Infant, newborn, Cholestasis, Enterocolitis, Necrotizing
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Study start
- May 2016 (actual)
- Primary completion
- Dec 2016 (actual)
- Study completion
- 2 Feb 2017 (actual)
- First posted
- 29 Mar 2016 (estimated)
- Results
- Posted 17 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Parenteral nutrition (PN) provides intravenous nutritional supplementation for infants unable to absorb adequate enteral nutrients secondary to insufficient intestinal length or function. In early PN-associated cholestasis, the dose of traditional soy based lipid is limited to 1 g/kg/day which often limits the growth capacity of parenteral nutrition-dependent infants. Inadequate growth is directly related to poor neurological outcomes, failure to facilitate mechanical ventilation, and less growth of the neonate's already damaged intestine. Ultimately, these outcomes can lead to severe disability and death. To mitigate these deleterious effects and optimize growth, parenteral nutrition-dependent infants with cholestasis who are not adequately growing on 1 g/kg/day of soy-based lipid emulsion must have a greater intake of lipids to meet their needs for weight, length, and head circumference growth.
SMOFlipid contains a mixture of 4 different lipid sources: soybean oil which provides essential fatty acids, olive oil which is high in monounsaturated fatty acids that are less susceptible to lipid peroxidation than polyunsaturated fatty acids, medium-chain triglycerides which show a faster metabolic clearance than long-chain triglycerides, and fish oil which provides the supply of omega-3 fatty acids. The utility of Omegaven and soy-based lipid emulsion is limited as these are restricted to 1 g/kg/day in cholestatic infants. SMOFlipid is safe to be provided at the usual goal infusion amount of 3 g/kg/day. Because this product includes both omega-6 and omega-3 lipids, it provides the benefits of the omega-3s for the liver and provides more than enough omega-6s to meet essential fatty acid requirements. Its use in situations in which growth is inadequate in babies who must be restricted to 1 g/kg/day can be expected to improve their growth and likely markedly increase their chances of both a good neurological outcome and survival.
The aim of this research study is to determine if the unique formulation of SMOFLipid will cause less hepatic inflammation compared to soy only intralipids.
Detailed description
The neonates who are being treated at the University of Florida Health Neonates Intensive Care Unit and are anticipated to need more than 21 days of intravenous nutrition will be considered as potential subjects. These subjects will receive the unique formulation of SMOFLipid
The following data will be collected from the subject's EMR by members of the research team:
- Lab values that evaluate liver function
- Growth parameters like head circumference, length, weight
- Medical/surgical history
- Time to resolution of bilirubin, time to liver transplant, time to death
- Length of hospital stay
- Rates of blood infections
Subjects will remain on SMOFlipid until weaned from PN.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SMOFlipid | Experimental | Drug: SMOFlipidSubjects with cholestasis will receive 3 G/kg/day of intravenous SMOFlipid daily until parenteral nutrition (PN) is discontinued. In addition, the following monitoring for effects of SMOFlipid will be performed: total days of parenteral nutrition, maximum conjugated bilirubin, time to resolution of bilirubin, time to liver transplant, time to death, positive blood cultures, rates of increase in weight, length, and head circumference, time dependent changes in liver function tests, including triglycerides, and length of hospital stay. |
Who may qualify
- Ages
- 23 weeks to 1 year
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Greater than 14 days old and less than 1 year of age.
- Greater than 1 kg.
- Mild cholestasis, defined as a conjugated bilirubin between 0.5-1.9 mg/dL
- Currently standard therapy with soy-based Intralipid
- Evidence of growth of weight, head circumference or length below our standards for post-menstrual age for at least 1 week.
Key exclusion criteria (4 listed)
- Have a congenitally lethal condition.
- Have clinically severe bleeding or clinical liver failure not able to be managed with routine measures.
- Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis.
- Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves
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 |
|---|---|---|
| SMOFlipidAlso: Parenteral nutrition (PN)SMOFlipid contains a mixture of 4 different lipid sources: soybean oil which provides essential fatty acids, olive oil which is high in monounsaturated fatty acids that are less susceptible to lipid peroxidation than polyunsaturated fatty acids, medium-chain triglycerides which show a faster metabolic clearance than long-chain triglycerides, and fish oil which provides the supply of omega-3 fatty acids. | Drug | SMOFlipid |
Primary outcome
Inflammation of the Liver Between the Groups
Time frame: 6 months
Inflammation of the liver will be evaluated by comparing direct bilirubin values between the two groups.
18 secondary outcomes
Measurement of Head Circumference for Growth Increase
Time frame: 6 months
Measurement of Weight for Growth Increase
Time frame: 6 months
Measurement of Length for Growth Increase
Time frame: 6 months
Number of Subjects Requiring Surgery
Time frame: 6 months
Number of Concomitant Medications Received
Time frame: 6 months
Length of IV Nutritional Therapy
Time frame: 6 months
Number of Subjects Receiving Formula Diet
Time frame: 6 months
Number of Subjects Receiving Breast Milk Diet
Time frame: 6 months
Number of Participants With Adverse Events Related to Treatment
Time frame: 6 months
Carbon Dioxide Total
Time frame: 6 months
Total Protein
Time frame: 6 months
Albumin
Time frame: 6 months
Aspartate Aminotransferase
Time frame: 6 months
Alanine Aminotransferase
Time frame: 6 months
Total Bilirubin
Time frame: 6 months
Serum Glucose
Time frame: 6 months
Alkaline Phosphatase
Time frame: 6 months
Triglyceride
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
Josef Neu, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SMOFlipid in people with Cholestasis and Jaundice, Obstructive, run by University of Florida at 1 site 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 6 inclusion and 4 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: stopped early, as of the sponsor's last update on 17 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Apr 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Inflammation of the Liver Between the Groups (time frame: 6 months). 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 17 Apr 2017.