Health TrialsbyHealthPanel

Severe Hepatitis Intervention With Emapalumab for Liver Disease

ConditionHepatitis
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-30
SponsorChildren's Healthcare of Atlanta

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

Any condition impairing informed consent/assent or protocol compliance

Steroid-refractory, steroid-dependent, or relapse of hepatitis after stopping steroids and have an Indeterminate etiology of hepatitis based on standard of care, age appropriate evaluation

Age: ≥ 1 year

ALT ≥ 1000 U/L prior to initiation of any immune modulatory therapy

Trial design

Design model

Single group

Treatments tested in this trial

  • Emapalumab

    Drug

    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.

Treatment groups

28 Participants
are divided into 1 treatment group
Group A: Intervention CohortExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of Liver Failure

INR \>2 on two consecutive occasions without hepatic encephalopathy (HE), or INR ≥1.5 with evidence of HE

Time frame
30, 90, and 180 days after the start of emapalumab

Secondary outcomes

1

Complete Response (CR)

A complete response (CR) is defined by normalization or near-normalization of liver injury markers and hepatic function. Patients achieving CR will demonstrate an ALT \< 2.5X ULN together with an INR ≤ 1.2.

Time frame
throughout study participation/ up to 180 days
2

Cytokine and Chemokine Profiling

The cytokine milieu including interferon-γ, interferon-stimulated gene products, TNF-α, IL-6, IL-18, CXCL9, and others will be quantified to define the inflammatory environment at baseline and during treatment.

Time frame
through study participation/ up to 180 days
3

Emapalumab Immunologic Effects

Change in circulating activated CD8+ T cells from the initiation of emapalumab to Days 7, 14, 30, 60, 90, and 180 of study

Time frame
through study completion/ up to 180 days
4

Incidence and Severity of Adverse Events

Incidence of all adverse events of all grades as defined by CTCAE version 5.0

Time frame
from first emapalumab dose through study completion, up to 180 days

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Healthcare of Atlanta

Lead sponsor

Sobi, Inc.

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: