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Immunosuppressive therapy for children with acute liver failure

A Phase 2b, Double-Blind, Three Arm, Randomized, Placebo Controlled Trial With Restricted Response Adaptive Randomization Testing the Efficacy and Safety of High Dose Methylprednisolone or Equine Anti-Thymocyte Globulin as Treatment for Acute Liver Failure in Pediatric Patients

Phase 2 Interventional National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · NCT04862221

This study is testing whether giving immunosuppressive treatments can help children with acute liver failure recover better than just supportive care alone.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment163 (estimated)
Ages1 Year to 18 Years
SexAll
SponsorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) NIH
Drugs / interventionschemotherapy
Locations24 sites (Los Angeles, California and 23 other locations)
Trial IDNCT04862221 on ClinicalTrials.gov

What this trial studies

The TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) trial is a multi-center, randomized, controlled trial aimed at evaluating the efficacy of immunosuppressive therapies in children suffering from acute liver failure. This phase 2b study will compare the effects of high-dose methylprednisolone and equine anti-thymocyte globulin against supportive care alone. The trial will enroll approximately 160 pediatric patients aged 1 to 18 years with acute liver failure of unknown cause, assessing their survival and recovery rates. The study employs a double-blind design and adaptive randomization to ensure robust results.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 1 to 18 years diagnosed with acute liver failure of less than 6 weeks duration.

Not a fit: Patients with active infections related to hepatitis or other specified viral infections may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could significantly improve survival rates and recovery outcomes for children with acute liver failure.

How similar studies have performed: Other studies have shown promise with immunosuppressive therapies in similar conditions, suggesting potential for success in this novel application.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Patient with liver injury of ≤ 6 weeks duration resulting in an international normalization ratio (INR) of ≥ 1.5 or \< 2.0 (not corrected by vitamin K) with evidence of hepatic encephalopathy (HE), INR of ≥ 1.5 or \< 2.0 for at least 7 days duration without evidence of 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

Where this trial is running

Los Angeles, California and 23 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Liver FailureFulminant Hepatic FailureHepatic EncephalopathyAcute Liver InjuryImmune Dysregulationhepatic insufficiencyliver diseasesliver failure
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.