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The purpose of this study is to determine if established parenteral nutrition (PN) associated liver disease can be reversed or its progression halted by using a parenteral fat emulsion prepared from fish oil as measured by normalization of serum levels of hepatic enzymes and bilirubin.
The primary objective of this study is to evaluate the need for and time to small bowel transplantation or liver transplantation. This will be calculated as both the age at time of primary transplant as well as the length of time from initial evaluation to transplant.Secondary objectives are to determine if there are improvements in clinical measures associated with established parenteral nutrition- associated liver disease (PNALD). These will be determined by measurement of total and direct bilirubin levels, platelet count, serum albumin, and changes in both length and weight growth curves.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Omegaven™ | Experimental | This study will be a prospective, non-randomized, open-label study of Omegaven™ for provision of parenteral lipid calories. The study cohort, receiving the parenteral nutrition (PN) lipid at 1g/kg/day, will be compared to historical controls at University of Nebraska Medical Center (UNMC) where parenteral lipid calories were provided exclusively through soybean-based formulations. The study is planned to enroll 100 patients. The Intestinal Rehabilitation Program at UNMC sees between 20 and 30 new pediatric patients per year, with almost all being PN-dependent and over 75% presenting with a bilirubin ≥ 2mg/dL. Based on these calculations, we estimate 4-5 years to enroll 100 patients. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Omegaven™ | Drug | 10% Omegaven™, 50 or 100 mL bottle; 1gram/kg/day and is infused over 12-24 hours. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients With Progression to Small Bowel Transplantation. | Whether or not the subject had an intestine-containing transplant or not | Bi-weekly x4, then monthly until the completion of the study, for an overall average of 5.5 years follow-up for the cohort |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects With Reversal of Biochemical Cholestasis | Of patients starting with elevated total serum bilirubin, the number that progress to normalization of serum total bilirubin in routine bloodwork | weekly x 4, then bi-weekly x4, then monthly until the completion of the study, with an average follow-up of 5.5 years for the study cohort |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David F Mercer, MD, PhD | University of Nebraska | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Nebraska Medical Center | Omaha | Nebraska | 68105 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 15659701 | Background | Alwayn IP, Gura K, Nose V, Zausche B, Javid P, Garza J, Verbesey J, Voss S, Ollero M, Andersson C, Bistrian B, Folkman J, Puder M. Omega-3 fatty acid supplementation prevents hepatic steatosis in a murine model of nonalcoholic fatty liver disease. Pediatr Res. 2005 Mar;57(3):445-52. doi: 10.1203/01.PDR.0000153672.43030.75. Epub 2005 Jan 19. | |
| 10022591 |
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Patient recruitment began May 1, 2008. The study was closed October 6, 2017
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| ID | Title | Description |
|---|---|---|
| FG000 | Omegaven™ | This study will be a prospective, non-randomized, open-label study of Omegaven™ for provision of parenteral lipid calories. The study cohort, receiving the PN lipid at 1g/kg/day, will be compared to historical controls at UNMC where parenteral lipid calories were provided exclusively through soybean-based formulations. The study is planned to enroll 100 patients. The Intestinal Rehabilitation Program at UNMC sees between 20 and 30 new pediatric patients per year, with almost all being PN-dependent and over 75% presenting with a bilirubin ≥ 2mg/dL. Based on these calculations, we estimate 4-5 years to enroll 100 patients. Omegaven™: 10% Omegaven™, 50 or 100 mL bottle; 1gram/kg/day and is infused over 12-24 hours. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Omegaven™ | This study will be a prospective, non-randomized, open-label study of Omegaven™ for provision of parenteral lipid calories. The study cohort, receiving the PN lipid at 1g/kg/day, will be compared to historical controls at UNMC where parenteral lipid calories were provided exclusively through soybean-based formulations. The study is planned to enroll 100 patients. The Intestinal Rehabilitation Program at UNMC sees between 20 and 30 new pediatric patients per year, with almost all being PN-dependent and over 75% presenting with a bilirubin ≥ 2mg/dL. Based on these calculations, we estimate 4-5 years to enroll 100 patients. Omegaven™: 10% Omegaven™, 50 or 100 mL bottle; 1gram/kg/day and is infused over 12-24 hours. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age at treatment start |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Patients With Progression to Small Bowel Transplantation. | Whether or not the subject had an intestine-containing transplant or not | Posted | Count of Participants | Participants | Bi-weekly x4, then monthly until the completion of the study, for an overall average of 5.5 years follow-up for the cohort |
|
Adverse events were collected over the term of the study, from May 1, 2009 until closure October 6, 2017. Each patient was assessed for treatment related adverse events from the initiation of therapy with Omegaven until 4 weeks after completion of therapy. This time was variable for each subject based on treatment length, with an overall average of 27 weeks follow-up for the cohort
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Omegaven™ | This study will be a prospective, non-randomized, open-label study of Omegaven™ for provision of parenteral lipid calories. The study cohort, receiving the PN lipid at 1g/kg/day, will be compared to historical controls at UNMC where parenteral lipid calories were provided exclusively through soybean-based formulations. The study is planned to enroll 100 patients. The Intestinal Rehabilitation Program at UNMC sees between 20 and 30 new pediatric patients per year, with almost all being PN-dependent and over 75% presenting with a bilirubin ≥ 2mg/dL. Based on these calculations, we estimate 4-5 years to enroll 100 patients. Omegaven™: 10% Omegaven™, 50 or 100 mL bottle; 1gram/kg/day and is infused over 12-24 hours. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| David Mercer | University of Nebraska Medical Center | 402-559-6955 | dmercer@unmc.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 5, 2009 | May 23, 2019 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D002779 | Cholestasis |
| D006963 | Hyperphagia |
| D008107 | Liver Diseases |
| ID | Term |
|---|---|
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D012817 | Signs and Symptoms, Digestive |
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| ID | Term |
|---|---|
| C568345 | fish oil triglycerides |
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| Van Aerde JE, Duerksen DR, Gramlich L, Meddings JB, Chan G, Thomson AB, Clandinin MT. Intravenous fish oil emulsion attenuates total parenteral nutrition-induced cholestasis in newborn piglets. Pediatr Res. 1999 Feb;45(2):202-8. doi: 10.1203/00006450-199902000-00008. |
| 16029913 | Background | Gura KM, Parsons SK, Bechard LJ, Henderson T, Dorsey M, Phipatanakul W, Duggan C, Puder M, Lenders C. Use of a fish oil-based lipid emulsion to treat essential fatty acid deficiency in a soy allergic patient receiving parenteral nutrition. Clin Nutr. 2005 Oct;24(5):839-47. doi: 10.1016/j.clnu.2005.05.020. |
| 18310188 | Background | 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. |
| 19179884 | Background | Diamond IR, Sterescu A, Pencharz PB, Kim JH, Wales PW. Changing the paradigm: omegaven for the treatment of liver failure in pediatric short bowel syndrome. J Pediatr Gastroenterol Nutr. 2009 Feb;48(2):209-15. doi: 10.1097/MPG.0b013e318182c8f6. |
| 23201707 | Derived | Mercer DF, Hobson BD, Fischer RT, Talmon GA, Perry DA, Gerhardt BK, Grant WJ, Botha JF, Langnas AN, Quiros-Tejeira RE. Hepatic fibrosis persists and progresses despite biochemical improvement in children treated with intravenous fish oil emulsion. J Pediatr Gastroenterol Nutr. 2013 Apr;56(4):364-9. doi: 10.1097/MPG.0b013e31827e208c. |
| Standard Deviation |
| months |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Gestational Age (weeks) | Mean | Standard Deviation | weeks |
|
| Weight (kg) at Treatment Start | Mean | Standard Deviation | kg |
|
| Weight Z Score at Treatment Start | The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean | Mean | Standard Deviation | z-score |
|
| Length (cm) at Treatment Start | Mean | Standard Deviation | cm |
|
| Length Z Score at Treatment Start | The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean | Mean | Standard Deviation | z score |
|
| Total Bilirubin (mg/dL) at Treatment Start | Mean | Standard Deviation | mg/dL |
|
| Direct Bilirubin (mg/dL) at Treatment Start | Mean | Standard Deviation | mg/dL |
|
| Serum aspartate aminotransferase (AST) at Treatment Start | Mean | Standard Deviation | U/L |
|
| Serum alanine aminotransferase (ALT) at treatment Start | Mean | Standard Deviation | U/L |
|
| Alkaline Phosphatase at Treatment Start | Mean | Standard Deviation | U/L |
|
| Parenteral Nutrition (PN) Calories at Treatment Start (kCal/kg/d) | Mean | Standard Deviation | kCal/kg/d |
|
| Enteral Calories at Treatment Start (kCal/kg/d) | Mean | Standard Deviation | kCal/kg/d |
|
| Underlying Diagnosis | Count of Participants | Participants |
|
|
|
| Secondary | Number of Subjects With Reversal of Biochemical Cholestasis | Of patients starting with elevated total serum bilirubin, the number that progress to normalization of serum total bilirubin in routine bloodwork | Posted | Count of Participants | Participants | weekly x 4, then bi-weekly x4, then monthly until the completion of the study, with an average follow-up of 5.5 years for the study cohort |
|
|
|
| 7 |
| 72 |
| 0 |
| 72 |
| 0 |
| 72 |
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| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |