A Phase 2 interventional study of Bortezomib and Placebo in Autoimmune Encephalitis, sponsored by Jena University Hospital. Recruiting at 17 sites in Germany. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-09-04.
Sponsored by Jena University Hospital · Phase 2, Interventional, and Treatment
Autoimmune Encephalitis is a disorder of the central nervous system caused by bodily substances, called antibodies. Antibodies normally help the body to prevent infections. However, in this disorder, the antibodies turn against the body itself and especially against cells in the brain and disturb the normal brain function. They are therefore called autoantibodies.
There is no specific therapy for patients with autoimmune encephalitis so far. At the moment, the symptoms are treated with approved medications such as cortisone and immunotherapies also used in oncology. These therapies are unspecified and aim to reduce the number of autoantibodies and to contain the autoimmune process. In this trial we aim to test a new therapy option: in this therapy the body cells producing autoantibodies will be specifically targeted by a substance called bortezomib.
The trial addresses patients with severe autoimmune encephalitis. The aim of the trial is to evaluate the efficacy and safety of bortezomib in patients with severe autoimmune encephalitis.
Autoimmune encephalitis is characterized by autoantibodies against neuronal surface antigens like the NMDA (N-methyl-D-aspartate) receptor or LGI1 (Leucin-rich glioma inactivated protein 1). So far, no specific therapy exists for this disease. Actual treatment includes combination therapies aiming for a reduction of pathogenic antibodies and containing the autoimmune process. In first line, patients are treated with plasmapheresis and cortisone. In second line, Rituximab and/or cyclophosphamide are administered. The response to these treatments are, however, often delayed and insufficient.
Therefore, we need a specific therapy aiming at the antibody-producing plasma cells.
Bortezomib is a proteasome inhibitor which interferes with NF-kB (nuclear factor kB) and the ubiquitin proteasome signaling pathway. Bortezomib acts preferably on cells with high protein synthesis - like plasma cells - and induces cell death in these cells. Bortezomib is used since more than a decade in chemotherapy of the multiple myeloma. Additionally, it is reported for systemic autoimmune diseases like lupus erythematodes that bortezomib leads to a depletion of plasma cells and therefore reduces the number of pathogenic antibodies and improves clinical outcome. The therapeutic potential of bortezomib for NMDAR encephalitis is described in a first case series with 5 patients.
123 studies on the registry are indexed under Autoimmune Diseases of the Nervous System; 59 are open to participants now.
This study's planned enrollment of 50 is above the median of 40 across 63 interventional studies indexed under Autoimmune Diseases of the Nervous System.
Browse Autoimmune Diseases of the Nervous System studies →Jena University Hospital is the lead sponsor of 64 studies on the registry; 20 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
1 to 3 cycles Bortezomib with 1,3mg/m2 body surface s.c. + 20mg dexamethasone p.o. on days 1, 4, 8 and 11 (= 1 cycle)
Drug: Bortezomib
1 to 3 cycles placebo (NaCl solution) s.c. + 20mg dexamethasone p.o. on days 1, 4, 8 and 11 (= 1 cycle)
Drug: Placebo
1 to 3 cycles Bortezomib with 1,3mg/m2 body surface s.c. + 20mg dexamethasone p.o. on days 1, 4, 8 and 11 (= 1 cycle)
1 to 3 cycles placebo (NaCl solution) s.c. + 20mg dexamethasone p.o. on days 1, 4, 8 and 11 (= 1 cycle)
Also known as: isotonic NaCl solution
modified Rankin-Score (mRS)
modified Rankin-Score from 0 = no symptoms to 6 = death
Time frame: 17 weeks after first administration of the study drug
modified Rankin-Score (mRS)
modified Rankin-Score from 0 = no symptoms to 6 = death
Time frame: 3, 6, 9 and 13 weeks after first administration of the study drug; GCS Score also 17 weeks after first administration of the study drug
Length of in-hospital stay / length of ICU stay
Number of days in hospital or on ICU for each patient from first administration of the study drug until 17 weeks after first administration of the study drug
Time frame: until 17 weeks after first administration of the study drug
Immune response
Antibody titer (in serum and liquor) and cellular immune response (FACS analysis of liquor)
Time frame: at study start and 17 weeks after first administration of the study drug
neurocognitive function assessed by Montreal Cognitive Assessment
total score of the Montreal Cognitive Assessment (MoCA) (0 to max. 30 points = best possible result)
Time frame: at study start and 17 weeks after first administration of the study medication
neurocognitive function assessed by Mini-Mental Status Test
total score of the Mini-Mental Status Test (MMST) (0 to max 30 points = best possible result)
Time frame: at study start and 17 weeks after first administration of the study medication
neurocognitive function assessed by Rey Auditory Verbal Learning Test
total score of the Rey Auditory Verbal Learning Test (RAVLT) (memory performance assessed by 3 word lists which are read to the patient and should be recalled and repeated by the patient; different proceeding for the 3 word lists)
Time frame: at study start and 17 weeks after first administration of the study medication
neurocognitive function assessed by Neuropsychiatric Inventory Questionnaire
total score of the Neuropsychiatric Inventory Questionnaire (NPI) (0 = best score to max 36 (patient) or 60 (caregiver)
Time frame: at study start and 17 weeks after first administration of the study medication
safety of Bortezomib regarding polyneuropathy, increase of liver enzymes and secondary infections
number of polyneuropathy cases, number of increased liver enzymes, number of secondary infections
Time frame: until 17 weeks after first administration of the study drug
safety of Bortezomib regarding polyneuropathy
number of polyneuropathy cases
Time frame: until 17 weeks after first administration of the study drug
safety of Bortezomib regarding increase of liver enzymes
number of increased liver enzyme values
Time frame: until 17 weeks after first administration of the study drug
Secondary infections due to Bortezomib
number of secondary infections
Time frame: until 17 weeks after first administration of the study drug
Hematotoxicity events due to Bortezomib
number of hematotoxicity events
Time frame: until 17 weeks after first administration of the study drug
Gastrointestinal toxicity due to Bortezomib
number of gastrointestinal toxicity events
Time frame: until 17 weeks after first administration of the study drug
total Glasgow Coma Scale (GCS)
GCS from 3 to 15 points (sum of 3 subscores eye response (1 to 4 points), motor response (1 to 6 points), verbal response (1 to 5 points); highest score = best score; 1= worst score)
Time frame: 3, 6, 9, 13 and 17 weeks after first administration of the study drug
Destruction marker UCH-L1 (Ubiquitin carboxy-terminal hydrolase L1) in serum and liquor
Analysis of destruction marker UCH-L1 in serum and liquor
Time frame: at baseline visit and 17 weeks after first administration of the study drug
Destruction marker Neurofilament light chain (in serum and liquor)
Analysis of destruction marker Neurofilament light chain in serum and liquor
Time frame: at baseline visit and 17 weeks after first administration of the study drug
Destruction markers GFAP (glial fibrillary acidic protein) in serum and liquor
Analysis of destruction marker GFAP in serum and liquor
Time frame: at baseline visit and 17 weeks after first administration of the study drug
Destruction marker TAU proteins in serum and liquor
Analysis of destruction marker TAU in serum and liquor
Time frame: at baseline visit and 17 weeks after first administration of the study drug
Plan to share: Undecided — It is not yet decided in which way and which data exactly will be shared with other researchers.
Started May 2020 (actual). Primary completion is estimated for Sep 2026, 76 months after the start.
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Studies of the drug tested here: Bortezomib
Autoimmune Diseases of the Nervous System →
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