Rasmussen EncephalitisNCT04003922
Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term
An observational study of Rasmussen Encephalitis, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial summary
Since the initial study of cases reported between 2009 and 2015, adalimumab has become in France the immunomodulatory reference treatment used after failure of corticosteroids and immunoglobulins before a possible recourse to the hemispherotomy. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Rasmussen Encephalitis
- What is being tested?
- Adalimumab treatment
- How many people
- 40 participants
- Where
- 1 siteFrance
- Study dates
- 21 Apr 2020to Dec 2027
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The implementation of a treatment with adalimumab in a patient with an ER requires the performance of a CPR within a reference center Epilepsies Rares if one complies with the rules of good clinical practice. Thus, all ER patients treated with adalimumab are referenced in a referral center. There are 7 reference centers in France, they are all partners of the study.
Who may be able to join
- Confirmed diagnosis of Rasmussen Encephalitis
- Patient aged more than 2 years
- Compliant treatment with adalimumab
Who cannot join
- Patient with a differential diagnosis
- Patient suffering from Rassmussen Encephalitis but not treated with adalimumab
- Patient who has not signed the informed consent
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Observational Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term
Since the initial study of cases reported between 2009 and 2015, adalimumab has become in France the immunomodulatory reference treatment used after failure of corticosteroids and immunoglobulins before a possible recourse to the hemispherotomy. This observational study is intended to document the long-term efficacy and safety of Adalimumab therapy in patients with Rasmussen encephalitis.
Read the detailed description
Rasmussen Encephalitis (ER) is a particularly severe chronic inflammatory brain disorder resulting in the progressive destruction of a hemisphere. It is a rare disease although at present no precise prevalence is available. It begins preferentially in children.
This inflammatory process is accompanied by a progressive loss of function of the affected hemisphere, associated with a pharmaco-resistant partial epilepsy. The diagnosis is based on a bundle of clinical, radiological and electroencephalographic arguments. CSF analysis directs diagnosis in 50% of cases.
No anti-epileptic treatment can stop seizures. Only hemispherotomy (surgical disconnection of a cerebral hemisphere) allows it but it is associated with definitive motor and cognitive deficits.
Over the last 20 years, new therapeutic trials have focused on immuno-modulatory treatments targeted at the T-lymphocyte pathway, including tacrolimus. Although they seem to be more effective than immunoglobulins or corticosteroids, it remains transient. In addition, the number of published cases is low. In this context, starting in 2009, it has been proposed to use adalimumab (Ab anti TNF) based on:
- 1 / study of a case index
- 2 / knowledge of the pathophysiology of Rasmussen Encephalitis. To date, very few data provide precise information on the efficacy or tolerance of the use of this product in the longer term. This information is essential to confirm the place of adalimumab in the therapeutic arsenal against Rasmussen encephalitis.
Thus, in the continuation of the work carried out previously (French study on the cases between 2009 and 2015), the aim of this research projet is to complete the follow-up of the patients who previously took part in in the first study and to establish the follow-up of the patients treated by adalimumab since then.
- Conditions
- Rasmussen Encephalitis
- Being tested
- Adalimumab treatment
- Medical subject headings
- Encephalitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adalimumab treatmentOther
Retrospective observational study initially and then prospective with inclusion of all the patients carrying an ER treated by Adalimumab in France. Follow-up will continue for one year if adalimumab is discontinued.
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficiency of the treatment measured by the reduction of the number of epileptic seizures
The change of epileptic seizures (Will be considered as a responder patient any patient with a 50% decrease in the number of seizures between the time of inclusion and 6 months after the start of treatment). This response to treatment is re-evaluated at 12 months then every 6 months for 5 years.
Measured: 5 years
Efficiency of the treatment measured by the stability if the cognitive assessment
Stability of the cognitive assessment: variation of less than 10 points on each of the different composite indices (QIT, ICV, IRF, IMT, IVT) of age-appropriate Weschler scales, between two cognitive assessments of 6 months.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2019
- Study start21 Apr 2020
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rasmussen EncephalitisStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe 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
- Other
- Time perspective
- Other
- Enrolment
- 40 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anne Lepine, Principal investigator, Assistance Publique - Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04003922
- Sponsor's protocol ID
- 2019-21
- Other IDs
- 2019-A00888-49
- Status last verified
- Oct 2026
- First submitted
- 20 Jun 2019
- Last updated
- 28 Oct 2026
- 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 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term
- ClinicalTrials.gov ID
- NCT04003922
- Acronym / sponsor ID
- 2019-21
- Conditions
- Rasmussen Encephalitis
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- 21 Apr 2020 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 1 Jul 2019 (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.
Since the initial study of cases reported between 2009 and 2015, adalimumab has become in France the immunomodulatory reference treatment used after failure of corticosteroids and immunoglobulins before a possible recourse to the hemispherotomy. This observational study is intended to document the long-term efficacy and safety of Adalimumab therapy in patients with Rasmussen encephalitis.
Detailed description
Rasmussen Encephalitis (ER) is a particularly severe chronic inflammatory brain disorder resulting in the progressive destruction of a hemisphere. It is a rare disease although at present no precise prevalence is available. It begins preferentially in children.
This inflammatory process is accompanied by a progressive loss of function of the affected hemisphere, associated with a pharmaco-resistant partial epilepsy. The diagnosis is based on a bundle of clinical, radiological and electroencephalographic arguments. CSF analysis directs diagnosis in 50% of cases.
No anti-epileptic treatment can stop seizures. Only hemispherotomy (surgical disconnection of a cerebral hemisphere) allows it but it is associated with definitive motor and cognitive deficits.
Over the last 20 years, new therapeutic trials have focused on immuno-modulatory treatments targeted at the T-lymphocyte pathway, including tacrolimus. Although they seem to be more effective than immunoglobulins or corticosteroids, it remains transient. In addition, the number of published cases is low. In this context, starting in 2009, it has been proposed to use adalimumab (Ab anti TNF) based on:
- 1 / study of a case index
- 2 / knowledge of the pathophysiology of Rasmussen Encephalitis. To date, very few data provide precise information on the efficacy or tolerance of the use of this product in the longer term. This information is essential to confirm the place of adalimumab in the therapeutic arsenal against Rasmussen encephalitis.
Thus, in the continuation of the work carried out previously (French study on the cases between 2009 and 2015), the aim of this research projet is to complete the follow-up of the patients who previously took part in in the first study and to establish the follow-up of the patients treated by adalimumab since then.
Study design
- Observational model
- Other
- Time perspective
- Other
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The implementation of a treatment with adalimumab in a patient with an ER requires the performance of a CPR within a reference center Epilepsies Rares if one complies with the rules of good clinical practice. Thus, all ER patients treated with adalimumab are referenced in a referral center. There are 7 reference centers in France, they are all partners of the study.
Key inclusion criteria (3 listed)
- Confirmed diagnosis of Rasmussen Encephalitis
- Patient aged more than 2 years
- Compliant treatment with adalimumab
Key exclusion criteria (3 listed)
- Patient with a differential diagnosis
- Patient suffering from Rassmussen Encephalitis but not treated with adalimumab
- Patient who has not signed the informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adalimumab treatmentRetrospective observational study initially and then prospective with inclusion of all the patients carrying an ER treated by Adalimumab in France. Follow-up will continue for one year if adalimumab is discontinued. | Other | – |
Primary outcome
Efficiency of the treatment measured by the reduction of the number of epileptic seizures
Time frame: 5 years
The change of epileptic seizures (Will be considered as a responder patient any patient with a 50% decrease in the number of seizures between the time of inclusion and 6 months after the start of treatment). This response to treatment is re-evaluated at 12 months then every 6 months for 5 years.
Efficiency of the treatment measured by the stability if the cognitive assessment
Time frame: 6 months
Stability of the cognitive assessment: variation of less than 10 points on each of the different composite indices (QIT, ICV, IRF, IMT, IVT) of age-appropriate Weschler scales, between two cognitive assessments of 6 months.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
Related trials for Rasmussen Encephalitis
RecruitingObservational study
Genetic and Electrophysiologic Study in Focal Drug-resistant Epilepsies
Drug-resistant Focal Epilepsies in Pediatric Population
Active, not recruitingPhase 2
Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
Autoimmune Disease, Neurologic Autoimmune Disease
RecruitingObservational study
Swiss Pediatric Inflammatory Brain Disease Registry (Swiss-Ped-IBrainD)
Optic Neuritis, Transverse Myelitis
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.
Anne Lepine principal investigator
Assistance Publique - Hôpitaux de Marseille
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 Rasmussen Encephalitis, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
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 3 inclusion and 3 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: under way, not looking for participants, as of the sponsor's last update on 28 Oct 2026. 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 France. 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?
This study is still under way. Results are not available in the source record yet.
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?
Efficiency of the treatment measured by the reduction of the number of epileptic seizures (time frame: 5 years). The study lists 2 primary outcomes in all. 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 28 Oct 2026; AzTrials last refreshed its copy on 4 Nov 2026.