Overview

TReatment for ImmUne Mediated PathopHysiology

Status:
Not yet recruiting
Trial end date:
2026-01-01
Target enrollment:
0
Participant gender:
All
Summary
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure. The study will determine if suppressing inflammatory responses with either corticosteroids or equine anti-thymocyte globulin therapy improves survival for children with this rare, life-threatening condition.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborator:
Ann & Robert H Lurie Children's Hospital of Chicago
Treatments:
Antilymphocyte Serum
Diphenhydramine
Methylprednisolone
Methylprednisolone Acetate
Methylprednisolone Hemisuccinate
Prednisolone
Prednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Promethazine
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. Positive test result for the severe acute respiratory syndrome coronavirus 2
(SARS-CoV-2) infection

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