Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04003922
Recruitment Status : Not yet recruiting
First Posted : July 1, 2019
Last Update Posted : July 1, 2019
Sponsor:
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.

Layout table for study information
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
Estimated Study Start Date : October 1, 2019
Estimated Primary Completion Date : January 31, 2020
Estimated Study Completion Date : October 1, 2024

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 reduction 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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.
Criteria

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


Contacts
Layout table for location contacts
Contact: Anne Lépine anne.lepine@ap-hm.fr
Contact: DRS AP-HM drci@ap-hm.fr

Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
Layout table for investigator information
Study Director: Jean-Olivier ARNAUD Assistance Publique- Hôpitaux de Marseille
Layout table for additonal information
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: July 1, 2019
Last Verified: June 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Encephalitis
Inflammation
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Pathologic Processes
Adalimumab
Anti-Inflammatory Agents
Antirheumatic Agents