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

Epilepsy Clinical Trial

— EPIRARE  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05126914
• Other study ID #: P210776
• Status: Not yet recruiting
• Start date: December 2021
• Est. completion date: December 2028

Study information

• Verified date: October 2021
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Stephane AUVIN, Pr
• Phone: 0140033667
• Email: stephane.auvin[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 1000
• Est. completion date: December 2028
• Est. primary completion date: December 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 15 Years

Eligibility

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

Related Conditions & MeSH terms

• Dravet Syndrome
• Epilepsies, Myoclonic
• Epilepsy
• Epileptic Syndromes
• Spasms, Infantile
• Syndrome
• West Syndrome

Locations

Country	Name	City	State
France	Hopital Robert Debré - Neurologie	Paris

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Orphelia Pharma

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	rate of decrease in epileptic seizures	rate of decrease in epileptic seizures according to the treatments used based on the seizure calendar kept by the parents as part of the current care.	5 years