Anti-NMDA (N-Methyl D-Aspartate) Receptor EncephalitisNEOSIINCT06023160
Predicting Functional Outcome and Response to Therapy of Anti-NMDAR Encephalitis at Diagnosis
An observational study of Anti-NMDA (N-Methyl D-Aspartate) Receptor Encephalitis, run by Erasmus Medical Center at 1 site in Netherlands.
Trial summary
The goal of this international cohort study is to develop a prediction model for long-term outcome and response to first-line immunotherapy of anti-NMDAR Encephalitis, already at the moment of diagnosis. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Anti-NMDA (N-Methyl D-Aspartate) Receptor Encephalitis
- How many people
- 714 participants
- Where
- 1 siteNetherlands
- Study dates
- 1 Dec 2020to 1 Jul 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Retrospective data of five (inter)national anti-NMDAR encephalitis cohorts.
Who may be able to join
- Diagnosed with anti-NMDAR encephalitis (according to the Graus criteria)
Who cannot join
- Preceding Herpes Simplex encephalitis
- Premorbid dependency (modified Rankin Scale > 2)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Predicting Functional Outcome and Response to Therapy of Anti-NMDAR Encephalitis at Diagnosis: The NEOSII Score
The goal of this international cohort study is to develop a prediction model for long-term outcome and response to first-line immunotherapy of anti-NMDAR Encephalitis, already at the moment of diagnosis.
Read the detailed description
Anti-NMDARE is a severe, but treatable neurological condition, with considerable and variable long-term disability. The previously developed anti-NMDAR Encephalitis One-Year Functional Status (NEOS) score predicts outcome a month into treatment. To predict outcome and response to immunotherapy at the time of diagnosis would be a serious improvement. This would timely identify patients in need for aggressive treatment and avoid harmful side-effects in those with good outcome. International data from five anti-NMDAR encephalitis cohorts will be combined to attain these goals.
The investigators strive to have less than 10% missing data on all variables and will impute data were needed. The datasets will then be split - with equal distributions of cohorts and good/poor outcome - to develop (70%) and validate (30%) the NEOS2 model. The primary outcome is functioning one year after diagnosis. A secondary analysis is targeted to predict the effect of first-line therapy. Potentially relevant predictive variables are identified with a univariable analysis on the original data, confirmed with backwards selection on the imputed datasets. After checking for multicollinearity and linearity of the variables, identified variables are added to a mixed effects logistic regression model on the original and imputed datasets, to identify the final set of predictive variables. To make the models opportune for daily medical practice, the investigators will assign points to (categories of) the included variables, based on the coefficients.
- Keywords
- Prognosis, Immunotherapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Dutch National anti-NMDAR Encephalitis Cohort | Dutch National anti-NMDAR Encephalitis Cohort | |
| German anti-NMDAR Encephalitis Cohort (GENERATE) | German anti-NMDAR Encephalitis Cohort (GENERATE) | |
| Spanish anti-NMDAR Encephalitis Cohort | Spanish anti-NMDAR Encephalitis Cohort | |
| French anti-NMDAR Encephalitis Cohort | French anti-NMDAR Encephalitis Cohort | |
| Japanese anti-NMDAR Encephalitis Cohort | Japanese anti-NMDAR Encephalitis Cohort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional status one year after diagnosis, on the modified Rankin Scale.
The score on the modified Rankin Scale will be applied as the original ordinal scale as well as dichotomized, where scores 0-2 represent "independent functioning" (without or with symptoms) and scores 3-6 increasing levels of depencency
Measured: One year after diagnosis
Secondary outcomes (3)
Response to first-line immunotherapy
One point improvement (one point lower than at diagnosis) on the modified Rankin Scale (ranging from 0 - no symptoms - to 6 - deceased - as the most severe category) within two weeks from therapy commencement.
Measured: Two weeks after administration of first-line immunotherapy
Return to school or work
Time-to-event analysis on the ability to return to school or work during follow-up, including timing of resumption of work after diagnosis.
Measured: Throughout follow-up time after diagnosis (different per patient, depending on date of diagnosis). Average 3 years (min 0 - max 22 years)
Relapse rate
Time-to-event analysis on relapses during total available follow-up time, including time of relapse after diagnosis.
Measured: Throughout follow-up time after diagnosis (different per patient, depending on date of diagnosis). Average 3 years (min 0 - max 22 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2023
- Study start1 Dec 2020
- Primary completion1 Jul 2023
- Study completion1 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anti-NMDA (N-Methyl D-Aspartate) Receptor EncephalitisStudies of the same condition, open and under way.
- More trials run by Erasmus Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 714 participants (actual)
Sponsor and investigators
- Lead sponsor
- Erasmus Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Maarten J. Titulaer. MD, PhD, Erasmus Medical Center
- Investigators
- Maarten J. Titulaer, Assoc. Porf., Principal investigator, Erasmus Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06023160
- Sponsor's protocol ID
- NEOSII
- Acronym
- NEOSII
- Status last verified
- Mar 2025
- First submitted
- 19 Aug 2023
- Last updated
- 4 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predicting Functional Outcome and Response to Therapy of Anti-NMDAR Encephalitis at Diagnosis: The NEOSII Score
- ClinicalTrials.gov ID
- NCT06023160
- Acronym / sponsor ID
- NEOSII / NEOSII
- Keywords
- Prognosis, Immunotherapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 714 participants (actual)
- Lead sponsor
- Erasmus Medical CenterUniversity, hospital or other
- Study start
- 1 Dec 2020 (actual)
- Primary completion
- 1 Jul 2023 (actual)
- Study completion
- 1 Jul 2024 (actual)
- First posted
- 5 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this international cohort study is to develop a prediction model for long-term outcome and response to first-line immunotherapy of anti-NMDAR Encephalitis, already at the moment of diagnosis.
Detailed description
Anti-NMDARE is a severe, but treatable neurological condition, with considerable and variable long-term disability. The previously developed anti-NMDAR Encephalitis One-Year Functional Status (NEOS) score predicts outcome a month into treatment. To predict outcome and response to immunotherapy at the time of diagnosis would be a serious improvement. This would timely identify patients in need for aggressive treatment and avoid harmful side-effects in those with good outcome. International data from five anti-NMDAR encephalitis cohorts will be combined to attain these goals.
The investigators strive to have less than 10% missing data on all variables and will impute data were needed. The datasets will then be split - with equal distributions of cohorts and good/poor outcome - to develop (70%) and validate (30%) the NEOS2 model. The primary outcome is functioning one year after diagnosis. A secondary analysis is targeted to predict the effect of first-line therapy. Potentially relevant predictive variables are identified with a univariable analysis on the original data, confirmed with backwards selection on the imputed datasets. After checking for multicollinearity and linearity of the variables, identified variables are added to a mixed effects logistic regression model on the original and imputed datasets, to identify the final set of predictive variables. To make the models opportune for daily medical practice, the investigators will assign points to (categories of) the included variables, based on the coefficients.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Dutch National anti-NMDAR Encephalitis Cohort | – | –Dutch National anti-NMDAR Encephalitis Cohort |
| German anti-NMDAR Encephalitis Cohort (GENERATE) | – | –German anti-NMDAR Encephalitis Cohort (GENERATE) |
| Spanish anti-NMDAR Encephalitis Cohort | – | –Spanish anti-NMDAR Encephalitis Cohort |
| French anti-NMDAR Encephalitis Cohort | – | –French anti-NMDAR Encephalitis Cohort |
| Japanese anti-NMDAR Encephalitis Cohort | – | –Japanese anti-NMDAR Encephalitis Cohort |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Retrospective data of five (inter)national anti-NMDAR encephalitis cohorts.
Key inclusion criteria (1 listed)
- Diagnosed with anti-NMDAR encephalitis (according to the Graus criteria)
Key exclusion criteria (2 listed)
- Preceding Herpes Simplex encephalitis
- Premorbid dependency (modified Rankin Scale > 2)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Functional status one year after diagnosis, on the modified Rankin Scale.
Time frame: One year after diagnosis
The score on the modified Rankin Scale will be applied as the original ordinal scale as well as dichotomized, where scores 0-2 represent "independent functioning" (without or with symptoms) and scores 3-6 increasing levels of depencency
3 secondary outcomes
Response to first-line immunotherapy
Time frame: Two weeks after administration of first-line immunotherapy
Return to school or work
Time frame: Throughout follow-up time after diagnosis (different per patient, depending on date of diagnosis). Average 3 years (min 0 - max 22 years)
Relapse rate
Time frame: Throughout follow-up time after diagnosis (different per patient, depending on date of diagnosis). Average 3 years (min 0 - max 22 years)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maarten J. Titulaer, Assoc. Porf. principal investigator
Erasmus Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Anti-NMDA (N-Methyl D-Aspartate) Receptor Encephalitis, run by Erasmus Medical Center at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Functional status one year after diagnosis, on the modified Rankin Scale. (time frame: One year after diagnosis). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2025.