A Phase 2 Study of Emapalumab for Prevention of Liver Failure in Pediatric Indeterminate Severe Hepatitis Associated With T-Cell Activation: Severe Hepatitis Intervention With Emapalumab for Liver Disease
Children's Healthcare of Atlanta
Summary
The goal of this study is to see whether a medicine called emapalumab, a monoclonal antibody, is safe and works well for patients aged 1 year and older who have indeterminate severe hepatitis with T-cell activation and have not responded well to steroids or still need them. This study has a Screening Cohort and an Intervention Cohort. The Screening Cohort will enroll patients with severe hepatitis of all etiologies to observe and collect research samples. Patients enrolled on the Screening Cohort will be asked to participate on the Intervention Cohort if they develop steroid-refractory/ dependent indeterminate severe hepatitis (iSH-T). Additionally, patients may bypass the Screening Cohort and enroll directly in the Intervention Cohort if they already meet its eligibility criteria. Patients in the Intervention Cohort will be treated with emapalumab.
Description
This clinical protocol evaluates the use of emapalumab, an anti-IFN-γ monoclonal antibody, in children aged ≥1 year with steroid-refractory or steroid-dependent indeterminate severe hepatitis with T cell activation (iSH-T). This study aims to establish a safe and effective treatment pathway for a previously steroid-refractory/dependent subjects with iSH-T. Pediatric severe acute hepatitis is a rare but serious disorder that can rapidly progress to liver failure. Previously healthy children who develop severe acute hepatitis can unpredictably progress to pediatric acute liver failure (PALF) ov…
Eligibility
- Age range
- 1–30 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria (Screening Cohort): * Age: Participants must be ≥ 1 year of age at the time of screening. * Evidence of Severe Hepatic Injury of all etiology (not just iSH/ iSH-T): Serum alanine aminotransferase (ALT) ≥ 1000 U/L documented within 7 days prior to enrollment. Exclusion Criteria (Screening Cohort): * Any condition impairing informed consent/assent or protocol compliance Inclusion Criteria (Intervention Cohort; patients who meet Intervention Cohort eligibility can bypass Screening Cohort enrollment): * Steroid-refractory, steroid-dependent, or relapse of hepatitis after st…
Interventions
- DrugEmapalumab
Participants on the Intervention Cohort will receive a combination of immunomodulatory regimen using emapalumab and methylprednisolone (or prednisone equivalent), aimed at controlling severe immune-mediated hepatitis and preventing progression to liver failure.
Location
- Arthur M. Blank Children's Healthcare of AtlantaAtlanta, Georgia