ESFstudy

JRCT ID: jRCTs031240436

Registered date:28/10/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: IFALD
• Date of first enrollment: 28/10/2024
• Target sample size: 15
• Study type: Interventional
• Intervention(s): Omegaven administered intravenously once daily over about 12 hours for 8 weeks

Outcome(s)

• Primary Outcome: The ratio of patients who were able to prevent or improve IFALD to all examinees

Secondary Outcome

(1)Rate of IFALD improvement
(2)Rate if IFALD preventionIFLAD prevention rate
(3)Rate of T/T ration > 0.02
(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

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: < 20age old
• Gender: Both

Include criteria

1. Patients who have been on parenteral nutrition (amino acids and lipid emulsion) for at least 2 weeks due to severe bowel dysfunction or significant intestinal loss and makes it difficult to establish enteral nutrition
2. 2. Patients who meet conditions (1)or (2) at the time of enrollment
(1)DB >1 mg/dL persists in any of the 2 measurements taken at intervals of at least 1 week apart within 4 weeks before enrollment
(2)AST, ALT, or gamma GTP (whichever is the same) > 2 times of normal range and persists in any of 2 measurements taken at intervals of at least 1 week within 4 weeks before enrollment
Patients with DB >=2 mg/dL and IFALD are also eligible
3. Regardless of gender
4. Age at enrollment must be less than 20-year-old
5. 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
6. Patients who can be hospitalized during the period of Omegaven treatment
7. 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 patients themselves and the legal guardian surrogate of the patients, and explanation using an assent document for patients

Exclude criteria

1. Patients who are expected to be able to gradually increase the amount of enteral nutrition 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, and 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 study within 28 days prior to 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 to 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

Related Information

• Primary Sponsor: Yohji Uehara
• Source(s) of Monetary Support: National center for child health and development