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

January 5, 2024 updated by: Assistance Publique Hopitaux De Marseille

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.

Study Overview

Status

Recruiting

Conditions

Intervention / 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.

Study Type

Observational

Enrollment (Estimated)

40

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: DRS AP-HM
  • Phone Number: 33 0491381499
  • Email: drci@ap-hm.fr

Study Locations

  • France
      • Bordeaux, France
        • Not yet recruiting
        • CHU de Bordeaux
        • Contact:
          • Frédéric Villega
      • Brest, France
        • Not yet recruiting
        • CHU Brest
        • Contact:
          • Jérémie Lefranc
      • Lille, France
        • Not yet recruiting
        • Chru De Lille
        • Contact:
          • Sylvie Nguyen
      • Limoges, France
        • Not yet recruiting
        • CHU Limoges
        • Contact:
          • Cécile Laroche
      • Lyon, France
        • Not yet recruiting
        • Hospices Civils de Lyon
        • Contact:
          • Dorothée Ville
      • Marseille, France, 13005
        • Recruiting
        • Service Neuropédiatrie
        • Contact:
          • Anne Lepine
      • Montpellier, France
        • Not yet recruiting
        • CHU Montpellier
        • Contact:
          • Agathe ROUBERTIE
      • Nancy, France
        • Not yet recruiting
        • CHU de Nancy
        • Contact:
          • Louis Maillard
      • Paris, France
        • Not yet recruiting
        • Chu Necker Ap-Hp
        • Contact:
          • Rima Nabbout
      • Paris, France
        • Not yet recruiting
        • Chu Pitie Salpietriere Ap-Hp
        • Contact:
          • Sophie DUPONT
      • Paris, France
        • Not yet recruiting
        • CHU Robert Debré
        • Contact:
          • Stéphane Auvin
      • Paris, France
        • Not yet recruiting
        • Fondation Adolphe de Rothshild
        • Contact:
          • Christine Bulteau
      • Rennes, France
        • Not yet recruiting
        • CHU de Rennes
        • Contact:
          • Silvia Napuri
      • Strasbourg, France
        • Not yet recruiting
        • CHU Strasbourg
        • Contact:
          • Anne De Saint-Martin
      • Toulouse, France
        • Not yet recruiting
        • CHU Toulouse
        • Contact:
          • Claude Cances
      • Tours, France
        • Not yet recruiting
        • Chru De Tours
        • Contact:
          • Maximilien Perivier

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique Hopitaux De Marseille

Investigators

  • Study Director: Jean-Olivier ARNAUD, Assistance Publique- Hôpitaux de Marseille

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 21, 2020

Primary Completion (Estimated)

April 1, 2024

Study Completion (Estimated)

October 1, 2025

Study Registration Dates

First Submitted

June 20, 2019

First Submitted That Met QC Criteria

June 28, 2019

First Posted (Actual)

July 1, 2019

Study Record Updates

Last Update Posted (Estimated)

January 8, 2024

Last Update Submitted That Met QC Criteria

January 5, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2019-21
  • 2019-A00888-49 (Other Identifier: ANSM)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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