Short Bowel SyndromePhase 2NCT01739517

Efficacy and Safety of Omega-3 Lipid Therapy in Pediatric Patients With Parenteral Nutrition-Associated Liver Disease

A Phase 2 study testing Omegaven Therapy in people with Short Bowel Syndrome and Cholestasis, run by Amarnath, Rathna, M.D. at 1 site in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

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Trial summary

This pilot study seeks to demonstrate the efficacy of an intravenous lipid preparation high in omega-3 fatty acids (Omegaven) in the treatment of cholestasis in parenteral nutrition dependent patients with short gut syndrome. Read more about this study

Who is it for?: Ages Up to 1 year
Conditions the study is for: Short Bowel Syndrome, Cholestasis
What is being tested?: Omegaven Therapy
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 20 participants
Where: 1 siteUnited States
Study dates: Mar 2009to Dec 2013
Phase: Phase 2
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 Up to 1 year
All sexes
No healthy volunteers
Child

Who may be able to join

Patient will be dependent upon parenteral nutrition (PN)
Patient will have short gut syndrome (loss of >50% of small bowel)
Patient's guardian/caregiver provides informed consent for patient to receive therapy
Pediatric patient ≤ 1 year of age
Expected PN duration is greater than 30 days
Direct bilirubin >2.0 mg/dL measured on two occasions no more than one week apart

Who cannot join

Liver dysfunction secondary to cause other than PN verified by standard of care diagnostic procedures and lab work to rule out alternative causes of neonatal cholestasis.
Any patient in whom Omegaven therapy would be contraindicated, such as an allergy to any seafood product, egg protein, and/or previously established allergy to Omegaven
impaired lipid metabolism
severe hemorrhagic disorder
unstable diabetes mellitus
collapse and shock, stroke/embolism, recent cardiac infarction, or undefined coma status

Trial locations and contacts

1 site in 1 country.

United States1 site, 1 recruiting

Palmetto Health Children's Hospital
Columbia, South Carolina, 29203
Terra R Varner, PharmD, 803-434-3034, terra.varner@palmettohealth.org
Terra R Varner, PharmD
Recruiting

Other trials in United States

About this study

Conditions: Short Bowel Syndrome, Cholestasis
Being tested: Omegaven Therapy
Keywords: Short bowel syndrome, Cholestasis, Omegaven, Parenteral nutrition-associated Liver Disease
Medical subject headings: Short Bowel Syndrome, Cholestasis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Omegaven TherapyDrug
After baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.

Study groups in NCT01739517
Study group	Role	What they receive
Omegaven Therapy	Experimental	After baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. Receives: Drug: Omegaven Therapy

What is being measured

The primary outcome is the main question the study is designed to answer.

Improvement of liver dysfunction as measured by time to achieve 50 % decrease in direct bilirubin
Direct bilirubin will be collected at baseline, then weekly for 30 days, and then biweekly, thereafter, up to an expected average of 108 weeks
Measured: weekly then biweekly data collection

Secondary outcomes (3)

a) Maintenance of nutritional status
Nutritional status will be monitored by reviewing complete metabolic panel, magnesium, weight, vitals, phosphorus, prealbumin, lipid panel, and essential free fatty acid profile. The essential fatty acid profile will be checked at baseline and then monthly for at least 6 months until the patient is determined to be receiving at least 2.7% of caloric intake from linoleic acid. If the patient's essential fatty acid profile indicates that the patient is absorbing adequate amounts of essential fatty acid, the essential fatty acid profile will be discontinued .
Measured: Labwork will be collected at baseline, then weekly for the first month. Thereafter, a lipid panel will be collected every 2 months, complete metabolic panel every 2 weeks, and essential fatty acid profile monthly, up to an expected average of 108 weeks
Occurrence of potential adverse side effects
Adverse events may include but are not limited to prolonged prothrombin time, hypertriglyceridemia, and anaphylaxis in relation to the patient's therapy.
Measured: biweekly labwork up to an expected average of 108 weeks
c) Resolution of liver dysfunction
Resolution of liver dysfunction will be defined by achievement of normal direct bilirubin, aspartate aminotransferase and alanine transaminase.
Measured: weekly complete metabolic panel for the first month and then biweekly thereafter, up to an expected average of 108 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Mar 2009Dec 2013

First posted3 Dec 2012 (estimated)
Study startMar 2009
Primary completionDec 2013 (estimated)
Study completionDec 2013 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (11)Papers the sponsor has linked to this study.
Other trials for Short Bowel SyndromeStudies 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: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 20 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01739517
Sponsor's protocol ID: IND104555
Status last verified: Nov 2012
First submitted: 28 Nov 2012
Last updated: 3 Dec 2012
Results posted: No
Data monitoring committee: Yes

Publications

Christensen RD, Henry E, Wiedmeier SE, Burnett J, Lambert DK. Identifying patients, on the first day of life, at high-risk of developing parenteral nutrition-associated liver disease. J Perinatol. 2007 May;27(5):284-90. doi: 10.1038/sj.jp.7211686. Epub 2007 Mar 8.
PubMed 17344923
Diamond IR, Sterescu A, Pencharz PB, Wales PW. The rationale for the use of parenteral omega-3 lipids in children with short bowel syndrome and liver disease. Pediatr Surg Int. 2008 Jul;24(7):773-8. doi: 10.1007/s00383-008-2174-0. Epub 2008 May 27.
PubMed 18504595
Kelly DA. Intestinal failure-associated liver disease: what do we know today? Gastroenterology. 2006 Feb;130(2 Suppl 1):S70-7. doi: 10.1053/j.gastro.2005.10.066.
PubMed 16473076
Gura KM, Duggan CP, Collier SB, Jennings RW, Folkman J, Bistrian BR, Puder M. Reversal of parenteral nutrition-associated liver disease in two infants with short bowel syndrome using parenteral fish oil: implications for future management. Pediatrics. 2006 Jul;118(1):e197-201. doi: 10.1542/peds.2005-2662.
PubMed 16818533
Moss RL, Das JB, Ansari G, Raffensperger JG. Hepatobiliary dysfunction during total parenteral nutrition is caused by infusate, not the route of administration. J Pediatr Surg. 1993 Mar;28(3):391-6; discussion 396-7. doi: 10.1016/0022-3468(93)90238-g.
PubMed 8468653
Chen WJ, Yeh SL, Huang PC. Effects of fat emulsions with different fatty acid composition on plasma and hepatic lipids in rats receiving total parenteral nutrition. Clin Nutr. 1996 Feb;15(1):24-8. doi: 10.1016/s0261-5614(96)80257-3.
PubMed 16843991
de Meijer VE, Le HD, Meisel JA, Gura KM, Puder M. Parenteral fish oil as monotherapy prevents essential fatty acid deficiency in parenteral nutrition-dependent patients. J Pediatr Gastroenterol Nutr. 2010 Feb;50(2):212-8. doi: 10.1097/MPG.0b013e3181bbf51e.
PubMed 20038849
Colomb V, Jobert-Giraud A, Lacaille F, Goulet O, Fournet JC, Ricour C. Role of lipid emulsions in cholestasis associated with long-term parenteral nutrition in children. JPEN J Parenter Enteral Nutr. 2000 Nov-Dec;24(6):345-50. doi: 10.1177/0148607100024006345.
PubMed 11071594
Yeh SL, Chen WJ, Huang PC. Effects of fish oil and safflower oil emulsions on diet-induced hepatic steatosis in rats receiving total parenteral nutrition. Clin Nutr. 1996 Apr;15(2):80-3. doi: 10.1016/s0261-5614(96)80024-0.
PubMed 16844003
Gura KM, Lee S, Valim C, Zhou J, Kim S, Modi BP, Arsenault DA, Strijbosch RA, Lopes S, Duggan C, Puder M. Safety and efficacy of a fish-oil-based fat emulsion in the treatment of parenteral nutrition-associated liver disease. Pediatrics. 2008 Mar;121(3):e678-86. doi: 10.1542/peds.2007-2248.
PubMed 18310188
Puder M, Valim C, Meisel JA, Le HD, de Meijer VE, Robinson EM, Zhou J, Duggan C, Gura KM. Parenteral fish oil improves outcomes in patients with parenteral nutrition-associated liver injury. Ann Surg. 2009 Sep;250(3):395-402. doi: 10.1097/SLA.0b013e3181b36657.
PubMed 19661785

Want to know more?

Each site lists its own contact. Pick the site nearest to you.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Short Bowel Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 3 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Efficacy and Safety of Omega-3 Lipid Therapy in Pediatric Patients With Parenteral Nutrition-Associated Liver Disease
ClinicalTrials.gov ID: NCT01739517
Acronym / sponsor ID: IND104555
Conditions: Short Bowel Syndrome, Cholestasis
Keywords: Short bowel syndrome, Cholestasis, Omegaven, Parenteral nutrition-associated Liver Disease
Phase: Phase 2
Study type: Interventional
Recruitment status: Unknown status
Enrolment: 20 participants (estimated)
Lead sponsor: Amarnath, Rathna, M.D.
Study start: Mar 2009
Primary completion: Dec 2013 (estimated)
Study completion: Dec 2013 (estimated)
First posted: 3 Dec 2012 (estimated)
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.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Omegaven Therapy	Experimental	Drug: Omegaven TherapyAfter baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.

Who may qualify

Ages: Up to 1 year
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (6 listed)

Patient will be dependent upon parenteral nutrition (PN)
Patient will have short gut syndrome (loss of >50% of small bowel)
Patient's guardian/caregiver provides informed consent for patient to receive therapy
Pediatric patient ≤ 1 year of age
Expected PN duration is greater than 30 days

Key exclusion criteria (6 listed)

Liver dysfunction secondary to cause other than PN verified by standard of care diagnostic procedures and lab work to rule out alternative causes of neonatal cholestasis.
Any patient in whom Omegaven therapy would be contraindicated, such as an allergy to any seafood product, egg protein, and/or previously established allergy to Omegaven
impaired lipid metabolism
severe hemorrhagic disorder
unstable diabetes mellitus

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 TherapyAfter baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.	Drug	Omegaven Therapy

Primary outcome

Improvement of liver dysfunction as measured by time to achieve 50 % decrease in direct bilirubin
Time frame: weekly then biweekly data collection
Direct bilirubin will be collected at baseline, then weekly for 30 days, and then biweekly, thereafter, up to an expected average of 108 weeks

3 secondary outcomes

a) Maintenance of nutritional status
Time frame: Labwork will be collected at baseline, then weekly for the first month. Thereafter, a lipid panel will be collected every 2 months, complete metabolic panel every 2 weeks, and essential fatty acid profile monthly, up to an expected average of 108 weeks
Occurrence of potential adverse side effects
Time frame: biweekly labwork up to an expected average of 108 weeks
c) Resolution of liver dysfunction
Time frame: weekly complete metabolic panel for the first month and then biweekly thereafter, up to an expected average of 108 weeks

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2012.
Background: PubMed 17344923Christensen RD, Henry E, Wiedmeier SE, Burnett J, Lambert DK. Identifying patients, on the first day of life, at high-risk of developing parenteral nutrition-associated liver disease. J Perinatol. 2007 May;27(5):284-90. doi: 10.1038/sj.jp.7211686. Epub 2007 Mar 8.
Background: PubMed 18504595Diamond IR, Sterescu A, Pencharz PB, Wales PW. The rationale for the use of parenteral omega-3 lipids in children with short bowel syndrome and liver disease. Pediatr Surg Int. 2008 Jul;24(7):773-8. doi: 10.1007/s00383-008-2174-0. Epub 2008 May 27.
Background: PubMed 16473076Kelly DA. Intestinal failure-associated liver disease: what do we know today? Gastroenterology. 2006 Feb;130(2 Suppl 1):S70-7. doi: 10.1053/j.gastro.2005.10.066.
Background: PubMed 16818533Gura KM, Duggan CP, Collier SB, Jennings RW, Folkman J, Bistrian BR, Puder M. Reversal of parenteral nutrition-associated liver disease in two infants with short bowel syndrome using parenteral fish oil: implications for future management. Pediatrics. 2006 Jul;118(1):e197-201. doi: 10.1542/peds.2005-2662.
Background: PubMed 8468653Moss RL, Das JB, Ansari G, Raffensperger JG. Hepatobiliary dysfunction during total parenteral nutrition is caused by infusate, not the route of administration. J Pediatr Surg. 1993 Mar;28(3):391-6; discussion 396-7. doi: 10.1016/0022-3468(93)90238-g.
Background: PubMed 16843991Chen WJ, Yeh SL, Huang PC. Effects of fat emulsions with different fatty acid composition on plasma and hepatic lipids in rats receiving total parenteral nutrition. Clin Nutr. 1996 Feb;15(1):24-8. doi: 10.1016/s0261-5614(96)80257-3.
Background: PubMed 20038849de Meijer VE, Le HD, Meisel JA, Gura KM, Puder M. Parenteral fish oil as monotherapy prevents essential fatty acid deficiency in parenteral nutrition-dependent patients. J Pediatr Gastroenterol Nutr. 2010 Feb;50(2):212-8. doi: 10.1097/MPG.0b013e3181bbf51e.
Background: PubMed 11071594Colomb V, Jobert-Giraud A, Lacaille F, Goulet O, Fournet JC, Ricour C. Role of lipid emulsions in cholestasis associated with long-term parenteral nutrition in children. JPEN J Parenter Enteral Nutr. 2000 Nov-Dec;24(6):345-50. doi: 10.1177/0148607100024006345.
Background: PubMed 16844003Yeh SL, Chen WJ, Huang PC. Effects of fish oil and safflower oil emulsions on diet-induced hepatic steatosis in rats receiving total parenteral nutrition. Clin Nutr. 1996 Apr;15(2):80-3. doi: 10.1016/s0261-5614(96)80024-0.
Background: PubMed 18310188Gura KM, Lee S, Valim C, Zhou J, Kim S, Modi BP, Arsenault DA, Strijbosch RA, Lopes S, Duggan C, Puder M. Safety and efficacy of a fish-oil-based fat emulsion in the treatment of parenteral nutrition-associated liver disease. Pediatrics. 2008 Mar;121(3):e678-86. doi: 10.1542/peds.2007-2248.
Background: PubMed 19661785Puder M, Valim C, Meisel JA, Le HD, de Meijer VE, Robinson EM, Zhou J, Duggan C, Gura KM. Parenteral fish oil improves outcomes in patients with parenteral nutrition-associated liver injury. Ann Surg. 2009 Sep;250(3):395-402. doi: 10.1097/SLA.0b013e3181b36657.

Related trials for Short Bowel Syndrome

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
Short Bowel Syndrome
Not yet recruitingPhase 2
Efficacy and Safety of TRA16 Administered Orally in Patients With Enteric Hyperoxaluria (EH).
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Active, not recruitingObservational study
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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.

Palmetto Health Children's Hospital

Columbia, United States

803-434-3034

terra.varner@palmettohealth.org

Terra R Varner, PharmD principal investigator

Palmetto Health Children's Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Omegaven Therapy in people with Short Bowel Syndrome and Cholestasis, run by Amarnath, Rathna, M.D. 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 6 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: status not confirmed, as of the sponsor's last update on 3 Dec 2012. 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 phase mean?

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?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Improvement of liver dysfunction as measured by time to achieve 50 % decrease in direct bilirubin (time frame: weekly then biweekly data collection). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01739517, which also lists 11 linked publications.

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 3 Dec 2012.