Omegaven as Alternative Parenteral Fat Nutrition

• Sponsor: University of South Florida (Other)
• Overall Status: Completed
• CT.gov ID: NCT03662282
• Collaborator: (none)
• Enrollment: 2
• Location: 1
• Arm: 1
• Actual Duration (Months): 2.5
• Patients Per Site Per Month: 0.8

Study Details

Study Description

Brief Summary

To provide Omegaven® as a compassionate treatment for critically ill infants with parenteral nutrition associated cholestasis

Condition or Disease	Intervention/Treatment	Phase
Cholestasis; Cholestasis of Parenteral Nutrition	Drug: Omegaven	Phase 3

Detailed Description

An intermediate population, Investigational New Drug study, to provide an alternate intravenous fat nutrition, Omegaven®, to help improve liver function while providing adequate nutrition for critically ill infants with parenteral nutrition associated cholestasis

Study Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Omegaven as Alternative Parenteral Fat Nutrition
• Actual Study Start Date: Oct 23, 2018
• Actual Primary Completion Date: Jan 7, 2019
• Actual Study Completion Date: Jan 7, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Drug - Omegaven® Therapy with Omegaven® will be initiated at the goal dose of 0.5-1/kg/day. The default is over 24 hours, but shorter intervals may be considered if a program of total parenteral nutrition cycling is recommended. Omegaven® will be infused intravenously through either a central or peripheral catheter.	Drug: Omegaven; Omegaven® fat emulsion will be used as a compassionate use treatment for critically ill infants with parenteral nutrition associated liver injury

Outcome Measures

Primary Outcome Measures

1. Number of Participant With Decrease in Direct Bilirubin Level [First month of treatment]

   Number of participant with decrease in direct bilirubin levels within 30 days of treatment

Secondary Outcome Measures

1. Number of Participant With Resolution of Direct Hyperbilirubinemia [2 months]

   Number of participant with Direct bilirubin level of <2mg/dL at discontinuation of treatment

2. Number of Participant With Preservation of Length [Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment]

   Monitoring of weekly length while the patient is receiving Omegaven® treatment

3. Number of Participant With Preservation of Head Circumference [Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment]

   Monitoring of weekly head circumference while the patient is receiving Omegaven® treatment

4. Number of Participant With Preservation of Normal Weight Gain [Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment]

   Monitoring of daily weight gain while the patient is on Omegaven®

Eligibility Criteria

Criteria

• Ages Eligible for Study: 14 Days and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Greater than 14 days old

• Has parenteral nutrition associated cholestasis defined as at least 2 consecutive direct bilirubin >2 mg/dL with anatomical or functional short gut (OR >4 mg/dL if intact intestine) obtained at least 1 week apart with a ratio of direct: total bilirubin > 0.4

• Patient is parenteral nutrition dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require parenteral nutrition for at least 3 more weeks

• Patient has not responded to other therapeutic approaches for parenteral nutrition-associated liver disease such as : cycling of parenteral nutrition, avoiding overfeeding, reduction/removal of copper and manganese from parenteral nutrition, advancement of enteral feeding, and use of Ursodiol

• Patient has been on at least 2 weeks of SMOFLIPIDs with no improvement or worsening of direct bilirubin levels

• Signed patient informed consent

• The patient is expected to have a reasonable possibility of survival

• No other known etiology of cholestasis other than parenteral nutrition-associated liver disease at the time of Omegaven® initiation

Exclusion Criteria:

• Known causes of cholestasis other than parenteral nutrition-associated liver disease including but not limited to Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 antitrypsin deficiency are present, prior to Omegaven® initiation

• Known fish or egg allergy

• Any of the contraindications to use of Omegaven®:

1. Active new infection at the time of initiation of Omegaven®

2. Hemodynamic instability

3. Recent use of medications with associated risk of bleeding, including NSAIDs

4. Active coagulopathy or bleeding

5. Platelet counts persistently under 30,000 despite transfusions

6. Unstable hyperglycemia

7. Impaired lipid metabolism (triglycerides >1000 mg/dL) while on 1 g/kg/day or less of Intralipid

8. History of severe hemorrhagic disorders (ie. hemophilia, Von Willebrand disease, etc.)

9. Unstable diabetes mellitus

10. Collapse and shock

11. Stroke/ Embolism

12. Cardiac infarction within the last 3 months

13. Undefined coma status

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	USF Health	Tampa	Florida	United States	33606

Sponsors and Collaborators

• University of South Florida

Investigators

• Principal Investigator: Thao Ho, DO, University of South Florida

Study Documents (Full-Text)

• Study Protocol - May 1, 2018

More Information

Publications

None provided.

• Responsible Party: University of South Florida
• ClinicalTrials.gov Identifier: NCT03662282
• Other Study ID Numbers: 00033519
• First Posted: Sep 7, 2018
• Last Update Posted: Jul 22, 2020
• Last Verified: Jul 1, 2019

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by University of South Florida: Omegaven; parenteral nutrition; Liver disease; Cholestasis
• Additional relevant MeSH terms: Cholestasis

Study Results

Participant Flow

Recruitment Details	Infants were recruited from a single NICU for compassionate use of Omegaven for cholestasis.
Pre-assignment Detail	Infants were screened based on inclusion and exclusion criteria. When there was a potential candidate, an IND application is submitted to FDA.

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Participants were screened based on inclusion and exclusion criteria. If and when a participant was qualified for Omegaven treatment, we sent FDA and application for IND approval. After the approval, the participant received Omegaven daily per protocol until cholestatsis resolved.
Period Title: Overall Study
STARTED	2
COMPLETED	2
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	These infants were more than one month old with history of long-term TPN dependence due to intestinal problem.
Overall Participants	2
Age (days) [Mean (Full Range) ]
Mean (Full Range) [days]	45
Sex: Female, Male (Count of Participants)
Female	1 50%
Male	1 50%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%
Not Hispanic or Latino	2 100%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	2 100%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (Count of Participants)
United States	2 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participant With Decrease in Direct Bilirubin Level
Description	Number of participant with decrease in direct bilirubin levels within 30 days of treatment
Time Frame	First month of treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Omegaven treatment group
Measure Participants	2
decrease in bilirubin level	2 100%
increase or no change in bilirubin level	0 0%

2. Secondary Outcome

Title	Number of Participant With Resolution of Direct Hyperbilirubinemia
Description	Number of participant with Direct bilirubin level of <2mg/dL at discontinuation of treatment
Time Frame	2 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Omegaven treatment group
Measure Participants	2
bilirubin <2mg/dL at discontinuing of Omegaven	1 50%
bilirubin >2mg/dL at discontinuing of Omegaven	1 50%

3. Secondary Outcome

Title	Number of Participant With Preservation of Length
Description	Monitoring of weekly length while the patient is receiving Omegaven® treatment
Time Frame	Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Omegaven treatment group
Measure Participants	2
Preservation of length growth	2 100%
fail in length growth	0 0%

4. Secondary Outcome

Title	Number of Participant With Preservation of Head Circumference
Description	Monitoring of weekly head circumference while the patient is receiving Omegaven® treatment
Time Frame	Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Omegaven treatment group
Measure Participants	2
preservation of head circumference	2 100%
Fail in head circumference growth	0 0%

5. Secondary Outcome

Title	Number of Participant With Preservation of Normal Weight Gain
Description	Monitoring of daily weight gain while the patient is on Omegaven®
Time Frame	Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Omegaven treatment group
Measure Participants	2
preservation of weight gain	2 100%
fail in weight gain	0 0%

Adverse Events

Time Frame	2 months
Arm/Group Title	Drug - Omegaven®
Arm/Group Description	Omegaven treatment group
All Cause Mortality
	Drug - Omegaven®
	Affected / at Risk (%)	# Events
Total	0/2 (0%)
Serious Adverse Events
	Drug - Omegaven®
	Affected / at Risk (%)	# Events
Total	0/2 (0%)
Other (Not Including Serious) Adverse Events
	Drug - Omegaven®
	Affected / at Risk (%)	# Events
Total	0/2 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.