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COMparison Between Anakinra and Tocilizumab in NORSE - "COMBAT-NORSE"

NCT07281027 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 438

Last updated 2026-05-12

No results posted yet for this study

Summary

The goal of this clinical trial is to find out whether two existing medications-anakinra and tocilizumab-can effectively treat a rare and life-threatening brain condition called NORSE (New-Onset Refractory Status Epilepticus). NORSE causes continuous seizures in previously healthy children and adults and does not respond to standard treatments. It often leads to long-term disability or death.

Doctors currently use anakinra and tocilizumab as second-line treatments when first-line therapies fail, but there is no clear evidence showing which drug works better or when it should be given. This study aims to answer those questions.

The study will enroll patients across 33 hospitals in the United States, Canada, Europe, and Asia.

It includes two groups:

1. Randomized Cohort Patients will be randomly assigned to receive either anakinra or tocilizumab within the first 7 days of their illness. Only patients whose doctors were already planning to use one of these medications as part of standard care will be eligible for randomization. Researchers will monitor their recovery and compare outcomes between the two treatments.
2. Observational Cohort Patients who cannot be randomized-usually because they were diagnosed too late-will still be followed to study how the timing of treatment affects recovery.

Participants will:

* Receive one of the two medications (depending on their group assignment).
* Take part in follow-up assessments over the course of one year, including medical evaluations and surveys. Some participants may be followed annually beyond one year.
* Optionally participate in a 60-minute interview to share their or their caregiver's experience with NORSE.

Conditions

  • New Onset Refractory Status Epilepticus
  • New-Onset Refractory Status Epilepticus
  • Febrile Infection-Related Epilepsy Syndrome (FIRES)

Interventions

DRUG

Anakinra

SOC will be followed , Suggested Dose: 10 mg/kg/day IV, divided into 4 daily doses (q6h) Maximum dose: 400 mg/day

DRUG

Tocilizumab

SOC will be followed, Suggested Dose: If \<30 kg: 12 mg/kg IV once every 2 weeks If ≥30 kg: 8 mg/kg IV once every 2 weeks Maximum dose: 800 mg per dose

OTHER

Standard medical treatment

For patients who could not be randomized by day 7, standard clinical care will be followed and patients will be followed prospectively and observationally.

Sponsors & Collaborators

  • Patient-Centered Outcomes Research Institute

    collaborator OTHER

  • Yale University

    lead OTHER

Principal Investigators

  • Lawrence Hirsch, MD · Yale University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-07-01
Primary Completion
2030-03-31
Completion
2030-09-30
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Italy
  • South Korea
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07281027 on ClinicalTrials.gov