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Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term

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ClinicalTrials.gov Identifier: NCT04003922
Recruitment Status : Recruiting
First Posted : July 1, 2019
Last Update Posted : August 2, 2022
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille

Brief 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. This observational study is intended to document the long-term efficacy and safety of Adalimumab therapy in patients with Rasmussen encephalitis.

Condition or disease Intervention/treatment
Rasmussen Encephalitis Other: Adalimumab treatment

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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 40 participants
Observational Model: Other
Time Perspective: Other
Target Follow-Up Duration: 5 Years
Official Title: Observational Study on Patients With Rasmussen Encephalitis Treated With Adalimumab: Efficacy and Tolerance in the Short and Long Term
Actual Study Start Date : October 20, 2020
Estimated Primary Completion Date : October 2023
Estimated Study Completion Date : October 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Encephalitis
Drug Information available for: Adalimumab

Intervention Details:
  • Other: Adalimumab treatment
    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.

Primary Outcome Measures :
  1. 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.

  2. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
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.

Inclusion Criteria:

  • Confirmed diagnosis of Rasmussen Encephalitis
  • Patient aged more than 2 years
  • Compliant treatment with adalimumab

Exclusion Criteria:

  • Patient with a differential diagnosis
  • Patient suffering from Rassmussen Encephalitis but not treated with adalimumab
  • Patient who has not signed the informed consent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04003922

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Contact: Anne Lépine 0491387267 ext 33 anne.lepine@ap-hm.fr
Contact: DRS AP-HM 0491381499 ext 33 drci@ap-hm.fr

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Service Neuropédiatrie Recruiting
Marseille, France, 13005
Contact: Anne Lepine         
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
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Study Director: Jean-Olivier ARNAUD Assistance Publique- Hôpitaux de Marseille
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Responsible Party: Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier: NCT04003922    
Other Study ID Numbers: 2019-21
2019-A00888-49 ( Other Identifier: ANSM )
First Posted: July 1, 2019    Key Record Dates
Last Update Posted: August 2, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Pathologic Processes
Anti-Inflammatory Agents
Antirheumatic Agents