Evaluating Bortezomib for Severe Autoimmune Encephalitis

A Multicenter Randomized, Controlled, Double-blinded Trial to Evaluate Efficacy and Safety of Bortezomib in Patients With Severe Autoimmune Encephalitis

Phase 2 Interventional Jena University Hospital · NCT03993262

This study is testing if bortezomib can help people with severe autoimmune encephalitis feel better by targeting the cells that produce harmful antibodies.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment50 (estimated)
Ages18 Years and up
SexAll
SponsorJena University Hospital Academic / other
Drugs / interventionsrituximab, chemotherapy, cyclophosphamide
Locations17 sites (München, Bavaria and 16 other locations)
Trial IDNCT03993262 on ClinicalTrials.gov

What this trial studies

This trial aims to evaluate the efficacy and safety of bortezomib, a proteasome inhibitor, in patients diagnosed with severe autoimmune encephalitis characterized by autoantibodies against neuronal surface proteins. Currently, there are no specific therapies for this condition, and existing treatments often provide insufficient relief. The study will involve administering bortezomib to target the antibody-producing plasma cells, potentially leading to improved clinical outcomes. Participants will be monitored for both efficacy and safety throughout the trial.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with clinically diagnosed severe autoimmune encephalitis and detectable autoantibodies.

Not a fit: Patients who are pregnant, breastfeeding, or have certain acute diseases or malignancies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a targeted therapy option for patients suffering from severe autoimmune encephalitis.

How similar studies have performed: While bortezomib has shown promise in small case series for similar conditions, this approach is still considered novel for treating autoimmune encephalitis.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Clinically diagnosed severe autoimmune encephalitis (defined as mRS ≥ 3) with autoantibodies to neuronal surface proteins in cerebrospinal fluid and / or serum
* Pretreatment with rituximab
* Age ≥18 years
* signed informed consent
* Women of childbearing potential (up to 2 years after menopause): negative pregnancy test

Exclusion Criteria:

* pregnancy/breast-feeding
* acute infiltrative pulmonary and pericardial disease
* malignant tumor under current chemotherapy
* Simultaneous participation in another intervention study
* Previous participation in this study
* Known hypersensitivity to an ingredient of the investigational product
* Continued therapy with glucocorticoids / rituximab during the study duration (last dose must be administered before the first dose of the investigational product)

Where this trial is running

München, Bavaria and 16 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Autoimmune Encephalitisautoimmune diseaseautoimmune encephalitisbortezomibNMDARLGI1encephalitis
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.