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Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents

NCT ID: NCT05126914

Last Updated: 2021-11-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-12-31

Study Completion Date

2028-12-31

Brief Summary

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Rare epilepsies as a whole account for 20-30% of epilepsies, but knowledge about prognostic factors is currently limited. This means that it is difficult to provide adequate information to families at diagnosis and during follow-up. Prognostic factors are also important for management as they can have an impact on the patient's outcome (time to intervention, choice of one molecule over another, etc.). Finally, few treatments are currently available for these epilepsies. One of the limitations to the development of treatments is the lack of real life data as it is difficult to create reliable primary endpoints such as the rate of patients becoming seizure free naturally compared to a therapeutic intervention.

The aim of this real-life study is to evaluate the response to treatment as well as to see the evolution of cognitive and psychiatric comorbidities. As explained above, there are very few randomised trials except for 3 rare epilepsies (infantile spasm syndrome, Dravet syndrome, Lennox-Gastaut syndrome). This has led to the virtual absence of management recommendations, including for the three syndromes mentioned above, where attempts at treatment algorithms have been proposed, although these have not been able to be considered as evidence-based recommendations.

As a result, there is some diversity in the management of rare epilepsies from one centre to another. However, this diversity in management can be an asset in a real-life study. This will make it possible to compare different management methods, both in terms of seizure control and medium-term outcome.

Detailed Description

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Conditions

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Epilepsy West Syndrome Dravet Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* Diagnosis for rare epilepsy (based on ORPHA codes)
* holders of parental authority not opposed
* Be followed in one of the declared centers of the study

Exclusion Criteria

* opposition from the holders of parental authority or the patient
Maximum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Orphelia Pharma

INDUSTRY

Sponsor Role collaborator

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Stephane AUVIN, Pr

Role: STUDY_DIRECTOR

Hopital Robert Debré - Assistance Publique Hopitaux de Paris

Locations

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Hopital Robert Debré - Neurologie

Paris, , France

Site Status

Countries

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France

Central Contacts

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Stephane AUVIN, Pr

Role: CONTACT

Phone: 0140033667

Email: [email protected]

Blandine DOZIERES, Dr

Role: CONTACT

Email: [email protected]

Other Identifiers

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P210776

Identifier Type: -

Identifier Source: org_study_id