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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Friedrich-Schiller-Universitaet Jena

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

13 May 2020 → ongoing

Decision date (initial)

2024-06-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Bundesministerium für Forschung, Technologie und Raumfahrt (BMFTR)

External identifiers

EU CT number

2024-514494-21-00

EudraCT number

2019-001423-12

ClinicalTrials.gov

NCT03993262

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

Evaluation of the efficacy and safety of bortezomib in patients with severe autoantibody-positive autoimmune encephalitis

Conditions and MedDRA coding

autoimmune encephalitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Clinically diagnosed severe autoimmune encephalitis (defined as mRS ≥ 3)
2. Autoantibodies against neuronal surface proteins in cerebrospinal fluid or serum serum, detection must not be older than 4 weeks, calculated before randomization
3. Pre-treatment with rituximab
4. Age ≥ 18 years
5. Written informed consent of the patient or the patient “under witness” (if the patient cannot write for motor reasons) cannot write themselves) or the legal representative (=guardian) or the authorized representative
6. Potentially fertile patient (up to 2 years after menopause): negative pregnancy test

Exclusion criteria 8

1. Lactation
2. Acute infiltrative lung disease
3. Acute infiltrative pericardial disease
4. Malignant tumor under ongoing or newly started chemotherapy
5. Concurrent participation in another intervention study
6. Previous participation in this study
7. Known hypersensitivity to any ingredient of the investigational product
8. Continued therapy with glucocorticoids/rituximab during the duration of the study (last administration must be completed before first administration of investigational product)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. mRS 17 weeks after first administration of the investigational product

Secondary endpoints 6

1. mRS and GCS 3, 6, 9 and 13 weeks after first administration of the investigational product; GCS 17 weeks after first administration of the investigational product
2. Length of stay in hospital/intensive care unit
3. Antibody titers and destruction markers (in serum and cerebrospinal fluid), cellular immune response (FACS, in cerebrospinal fluid) at the baseline visit and 17 weeks after first administration of the investigational product
4. Neurocognitive function (MoCA, MMST, VLMT and NPI) at the baseline visit and 17 weeks after the first visit and 17 weeks after first administration of the investigational product
5. Number of all (serious) adverse events within 17 weeks after the first 17 weeks after first administration of the investigational product
6. Bortezomib safety with regard to polyneuropathy, increase in liver enzymes liver enzymes, hematotoxicity, gastrointestinal toxicity and secondary infections.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Friedrich-Schiller-Universitaet Jena

Sponsor organisation

Friedrich-Schiller-Universitaet Jena

Address

Am Klinikum 1, Lobeda Lobeda

City

Jena

Postcode

07747

Country

Germany

Scientific contact point

Organisation

Friedrich-Schiller-Universitaet Jena

Contact name

Prof. Dr. Christian Geis

Public contact point

Organisation

Friedrich-Schiller-Universitaet Jena

Contact name

Prof. Dr. Christian Geis

Locations

1 EU/EEA country · 17 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications