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Study on the efficacy and safety of the omega 3 fatty acid for IFALD (ESFstudy)

ESFstudy (ESFstudy)

Yohji Uehara

National center for child health and development

2-10-1 Okura, Setagaya-ku, Tokyo

uehara-y@ncchd.go.jp

Yohji Uehara

National center for child health and development

2-10-1 Okura, Setagaya-ku, Tokyo

+81-334160181

uehara-y@ncchd.go.jp

Not Recruiting

Oct. 28, 2024

Interventional

single arm study

open(masking not used)

active control

single assignment

treatment purpose

1. Patients who may develop IFALD or patients who have developed IFALD. Patients who may develop IFALD are defined as those meeting criteria (1) - (3) below: (1) A nutritional state dependent on parenteral nutrition (amino acids and lipid emulsion) for more than 2 weeks due to severe bowel dysfunction or significant intestinal loss (2) Difficulty in establishing enteral nutrition (3) Meeting either (a) or (b) below: (a) DB levels consistently >= 1 mg/dL in two measurements taken at least one week apart within 4 weeks before registration (b) Hepatobiliary enzyme level (any one of AST, ALT, or gamma GTP) consistently >= 2 times standard value of the facility in two measurements taken at least one week apart within 4 weeks before registration. Patients who have developed IFALD are defined as those meeting criteria (1) - (3) below: (1) A nutritional state dependent on parenteral nutrition (amino acids and lipid emulsion) for more than 2 weeks due to severe bowel dysfunction or significant intestinal loss (2) Difficulty in establishing enteral nutrition (3) DB levels consistently >= 2 mg/dL for more than 2 weeks
2. Age at enrollment must be less than 20-year-old
3. Patients must be eligible for a washout period of at least 28 days (14 days in cases of less than 1 year old) if they have used a fish oil emulsion (Omegaven or SMOFlipid) within 28 days before obtaining consent
4. Patients who can be hospitalized during Omegaven treatment
5. Written informed consent (>= 18-year-old)
~7-year-old: Written informed consent from the legal guardian surrogate of the patients 7~16-year-old: Written informed consent from the patient's legal guardian surrogate and explanation using an assent document for patients
16~18-year-old: Written informed consent from both of the patients themselves and the legal guardian surrogate of the patients and explanation using an assent document for patients

1. Patients who are expected to be able to increase the amount of enteral nutrition gradually and to be able to terminate parenteral nutrition (both amino acids and lipid emulsions). For patients under 1-year-old, the amount of enteral nutrition is expected to reach 120 ml/kg/day and 100 kcal/kg/day. For patients over 1 year old, the amount of enteral nutrition does not reach the estimated energy requirement based on the dietary reference intake for Japanese (2020 Edition).
2. Patients with comorbidities that may cause cholestatic liver failure other than intestinal failure and parenteral nutrition (e.g., infection, biliary obstructive disease, hemorrhage, hemolytic jaundice) or patients suffering from or strongly suspected to have lipid metabolism abnormalities
3. Patients with irreversible severe hepatic dysfunction or failure who meet any of the following conditions
<Symptoms of portal hypertension>
(1)Untreatable thrombocytopenia (< 30000/mm3) or leukopenia (< 5000/mm3) associated with hypersplenism
(2)associated with varices
<Grade III or higher degree hepatic encephalopathy>
<untreatable coagulation abnormalities (PT INR >= 1.6)>
4. Patients who are scheduled for surgery not related to intestinal failure within 56 days of enrollment
5. Patients who have participated in other clinical trials or interventional studies within 28 days before obtaining consent
6. Patients with life-threatening respiratory failure, circulatory failure, shock, stroke, embolism, or undiagnosed coma
7. Contraindications as listed within the Appendix (U.S. version)
<Known hypersensitivity to fish or eggs or the active ingredient or excipients>
<Severe bleeding disorders due to potential effects on platelet aggregation>
<Hypertriglyceridemia (>= 1000 mg/dL) >
8. Pregnant or lactating patients
9. Other patients who the principal investigator deems inappropriate for this study

Both

IFALD

Omegaven administered intravenously once daily over about 12 hours for 8 weeks

IFALD, intestinal failure associated liver disease

The ratio of patients who were able to prevent or improve IFALD to all examinees

(1) Rate of IFALD improvement
(2) Rate if IFALD preventionIFLAD prevention rate
(3) Rate of patients with T/T ratio > 0.02 on the day after the last dose
(4) Change in APRI score and PELD score (MELD score for patients aged 12 years or older)
(5) Change in weight z-score before and after the last dose of Omegaven
(6) Rate of subjects who were able to prevent or alleviate IFALD and whose DB >= 2 mg/dL persisted for 2 weeks or more during the follow-up period

+81-3-3416-0181

Sept. 26, 2024

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