COMparison Between Anakinra and Tocilizumab in NORSE - "COMBAT-NORSE"
NCT ID: NCT07281027
Last Updated: 2025-12-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
NOT_YET_RECRUITING
PHASE3
438 participants
INTERVENTIONAL
2026-03-01
2030-09-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
The Efficacy and Safety of Minocycline in the Treatment of Drug-resistant Epilepsy in NORSE Patients
NCT05958069
A Study of the Efficacy and Safety of Topiramate as "Add on" Treatment in Epilepsy Patients With Primary Generalized Tonic-clonic Seizures
NCT00236704
An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures
NCT06908356
Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension
NCT01963208
A Study of the Efficacy and Safety of Topiramate in Epilepsy Patients With Primary Generalized Tonic-clonic Seizures
NCT00236418
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Randomized Controlled Trial (RCT) Cohort
A randomized controlled cohort (RCT) of anakinra vs. tocilizumab (targeted immunotherapies) started up to and including 7 days after the onset of status epilepticus (SE)
Anakinra
SOC will be followed , Suggested Dose:
10 mg/kg/day IV, divided into 4 daily doses (q6h) Maximum dose: 400 mg/day
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
Observational Cohort
An observational cohort enrolling patients with acute cryptogenic NORSE who cannot be randomized or who are identified too late to be randomized by the end of day 7 .
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.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Anakinra
SOC will be followed , Suggested Dose:
10 mg/kg/day IV, divided into 4 daily doses (q6h) Maximum dose: 400 mg/day
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
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.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* In their usual state of health prior to their onset of SE.
* Presenting with NORSE as defined in the consensus criteria:
1. Refractory SE (failed 2 appropriately used anti-seizure medications) in a patient without active epilepsy or other pre-existing relevant neurological disorder and without an acute or active structural, toxic, or metabolic cause found in the first 72 hours.
2. Includes patients with any RSE, not just super-refractory SE.
3. Includes patients who ultimately are discovered to have a known etiology (infectious, autoimmune, genetic, etc.), as well as those who remain cryptogenic.
* Anakinra and/or tocilizumab are being planned or considered as part of standard clinical care.
* The onset of SE was in the prior 7 days at the time of enrollment.
Exclusion Criteria
* Contraindication to either anakinra or tocilizumab as listed in the prescribing information:
1. Known hypersensitivity to E. Coli-derived proteins, anakinra, tocilizumab, or any component of the products
2. Active serious infection at the time of initiation
3. Concomitant use of TNF blocking agents; absolute neutrophil count \< 2000; platelet count \< 100,000 per mm³; or ALT or AST \> 1.5 X the upper limit of normal
4. Elevated risk of GI perforation.
2 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Patient-Centered Outcomes Research Institute
OTHER
Yale University
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Lawrence Hirsch
Professor of Neurology
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Lawrence Hirsch, MD
Role: PRINCIPAL_INVESTIGATOR
Yale University
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Barrow Institute
Phoenix, Arizona, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Yale New Haven Hospital
New Haven, Connecticut, United States
Children's National (DC)
Washington D.C., District of Columbia, United States
University of Florida
Gainesville, Florida, United States
University of Chicago
Chicago, Illinois, United States
Northwestern University
Evanston, Illinois, United States
Mass General (MGH)
Boston, Massachusetts, United States
Beth Israel Deaconess
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
University of Nebraska
Lincoln, Nebraska, United States
New York University
New York, New York, United States
Columbia University
New York, New York, United States
Mount Sinai (NY)
New York, New York, United States
University of Cincinnati
Cincinnati, Ohio, United States
Cleveland Clinic
Cleveland, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center
Dallas, Texas, United States
Baylor/Texas Children's
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Western University
London, , Canada
Hospital for Sick Children
Toronto, , Canada
Salpêtrière
Paris, , France
University of Modena
Modena, , Italy
Seoul National University Hospital
Seoul, , South Korea
Karolinska Institute
Stockholm, , Sweden
Great Ormond Street Hospital
London, , United Kingdom
King's College
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
RD-2024C2-39648
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
2000041289
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.