Clinical trial NCT04398667: European Non-interventional Study on Refractory Epilepsy With Developmental Delay
Official title: A European Non-interventional Study to Understand the Criteria Used and the Time Required for the Clinical Diagnosis in Participants With Refractory Epilepsies Associated With Developmental Delay
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Refractory Epilepsy
Development Delay
Keywords: Observational
Retrospective
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
Collaborators: name: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
First posted: 2020-05-21
Enrollment: 93
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with rare epilepsy syndromes up to the date of the most recent visit at the study site.

Brief summary: This is a multinational, multicentre, non-interventional, retrospective data collection (manual Medical Chart Review).

Detailed description: This is a multinational, multicentre, non-interventional, retrospective data collection (manual Medical Chart Review). The identified local site staff (or trained independent data abstractors, if requested by the site) will review the medical records of all participants meeting the eligibility criteria, who have provided a signed inform consent form (ICF) for data collection and analysis. For participants who are less than 18 years of age, in addition to the assent form being signed by the participant, an informed consent would be obtained from parental or legally authorized representative.

In line with the retrospective nature of the study: there is no assignment of a participant to a particular therapeutic strategy; no additional diagnostic or monitoring procedures shall be applied to the participants; epidemiological methods shall be used for the analysis of collected data; the prescription of any medication for the treatment of the epileptic syndrome occur before the collection of the data in this study and is clearly separated from the decision to include the participant in the study.

Eligibility criteria: Inclusion Criteria:

* Age ≥2 years
* Disease history of at least 24 months from date of first seizure
* Medical history of epilepsy associated with moderate to severe intellectual disability, cognitive developmental delay or cognitive regression
* Medical history of onset of seizures in early childhood (≤ 8 years)
* Failure of adequate trials of two tolerated and appropriately chosen and used Anti-Epileptic Drug (AED) schedules (whether as monotherapies or in combination) to achieve sustained seizure freedom
* Ongoing refractory epilepsy
* MRI and EEG data are available for the participant
* Participants (or their parents/ legal representative as appropriate) have provided written informed consent / assent form to collect the data specified.

Exclusion Criteria:

* Normal cognitive development.
* Any acute symptomatic seizures in participants with underlying developmental delay.
* Any progressive intellectual and neurological deterioration conditions.

Primary outcomes:
measure: Proportion of Participants With and Without Diagnosis of an Epilepsy Syndrome
timeFrame: 5 months
measure: Understand the Clinical Practice Criteria
timeFrame: 5 months
measure: Clinical Practice Diagnostic Criteria Compared to ILAE
timeFrame: 5 months
measure: Understand the Time to Formal Diagnosis
timeFrame: 5 months
measure: Unclassified Epilepsy
timeFrame: 5 months
Secondary outcomes:
Other outcomes:
Locations:
city: Angers
country: France
facility: Unité de neuropédiatrie, CHU Angers
city: La Force
country: France
facility: Department of Neurology - Foundation John Bost
city: Paris
country: France
facility: Hospital St Louis DRCI, CHU Bicêtre
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Berlin
country: Germany
facility: Institut der Diagnostik der Epilepsien
city: Berlin
country: Germany
facility: Zentrum für Sozialpädiatrie und Neuropädiatrie (DBZ), Vivantes Klinikum
city: Bielefeld
country: Germany
facility: Gesellschaft für Epilepsieforschung e. V.
city: Bonn
country: Germany
facility: Klinik für Epileptologie, Universitätsklinikum Bonn
city: Frankfurt
country: Germany
facility: Klinik Für Kinder- und Jugendmedizin, Neuropadiatrie und Epilepsiezentrum
city: Greifswald
country: Germany
facility: Department of Neurology / Epileptology, University Medicine Greifswald
city: Kiel
country: Germany
facility: Klinik für Kinder-und Jugendmedizin II, Universitätsklinikum Schleswig-Holstein
city: Kork
country: Germany
facility: Epilepsiezentrum Kork
city: Köln
country: Germany
facility: Klinik für Neurologie, Heilig Geist-Krankenhaus GmbH
city: Magdeburg
country: Germany
facility: Department of Neurologie University of Magdeburg
city: Ravensburg
country: Germany
facility: Abteilung für Epileptologie, ZfP Südwürttemberg
city: Genova
country: Italy
facility: I.R.C.C.S. Giannina Gaslini
city: Troina
country: Italy
facility: Oasi Research Institute
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Ruber Internacional
Study officials:
References: