TReatment for ImmUne Mediated PathopHysiology

Launched by NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES (NIDDK) · Apr 23, 2021

Keywords

ClinConnect Summary

The TRIUMPH clinical trial is studying a new treatment approach for children with acute liver failure, a serious condition where the liver suddenly stops working properly. This trial aims to see if using medications that suppress the immune system, specifically corticosteroids or equine anti-thymocyte globulin, can help improve survival rates in these children. The study is open to children aged 1 to 17 who have liver injury lasting six weeks or less, along with specific blood test results indicating liver dysfunction.

If your child is eligible and agrees to participate, they will be randomly assigned to receive one of the two treatments or a control group and will be carefully monitored by healthcare professionals throughout the study. It's important to know that certain medical conditions, such as active infections or recent treatments, may prevent participation. This trial is currently recruiting participants, and your child's involvement could contribute to finding better treatments for this life-threatening condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Patient with liver injury of ≤ 6 weeks duration resulting in an international normalized ratio (INR) of ≥ 1.5 and \< 2.0 (not corrected by vitamin K) with evidence of hepatic encephalopathy (HE) or INR ≥ 2.0 without evidence of HE.
• • 2. Age is greater than or equal to 1 year and less than 18 years of age.
• • 3. Patient or their legally authorized representative(s) (LAR) must consent (and assent, if applicable) to be in the study and must have signed and dated an approved informed consent form which conforms to federal and institutional guidelines.
• • 4. Females of reproductive potential should not plan on conceiving children during the study and must agree to use a medically accepted form of contraception.
• Exclusion Criteria:
• • 1. Evidence of active infection with Hepatitis A, B, C, E or evidence of acute herpes simplex virus (HSV) or adenovirus infection
• • 2. Travel within the past 3 months to an area highly endemic for Hepatitis E
• • 3. Diagnosis of hemophagocytic lymphohistiocytosis (HLH) Note: Patients with a history of consanguinity and/or central nervous system (CNS) dysfunction that is exaggerated compared to the degree of liver dysfunction (as judged by the site investigator) will not be enrolled until results of rapid genetic testing are available. Turn-around time for genetic testing results is estimated to be 72-96 hours.
• • 4. Aplastic anemia as defined by standardized criteria \[1\] diagnosed prior to enrollment
• • 5. Diagnosis of autoimmune Hepatitis (AIH)
• • 6. Diagnosis of acute Wilson disease
• • 7. Diagnosis of inborn error of metabolism Note: Suspicion of metabolic disease is not an exclusion for entry into the Trial.
• • 8. Diagnosis of acute drug or toxin-induced liver injury
• • 9. History of recreational drug use within the past 4 weeks
• • 10. Therapy with an immunosuppressive agent, including chemotherapy, biological therapies or an experimental drug or device within the past 6 weeks
• • 11. Liver injury due to ischemia
• • 12. Liver dysfunction diagnosed more than 6 weeks prior to screening
• • 13. History of allergy to horse dander
• • 14. Sepsis
• • 15. Imminent risk of death as judged by the clinical site investigator, including but not limited to; signs of cerebral herniation at the time of enrollment and presence of intractable arterial hypotension
• • 16. Solid organ or stem cell transplant recipient
• • 17. Pregnant or breast-feeding at the time of proposed study entry
• • 18. Clinical AIDS or HIV positive
• • 19. History of any form of malignant neoplasm and/or tumors treated within five years prior to study entry (other than non-melanoma skin cancer or in situ cervical cancer) or where there is current evidence of recurrent or metastatic disease
• • 20. Received a live-virus vaccine within 4 weeks of study entry
• • 21. Patients with positive respiratory secretion testing for respiratory viral infection including SARS-CoV-2, influenza and respiratory syncytial virus only if they also have declining respiratory function
• • 22. Psychiatric or addictive disorders that would preclude obtaining informed consent/assent
• • 23. Patient is unwilling or unable to adhere with study requirements and procedures
• • 24. Currently receiving other experimental therapies