A Study to Gather Information About Overall Occurrence and New Cases of Dravet and Lennox-Gastaut Syndromes in Children, Teenagers and Adults in Spain

NCT05982717 | Completed

• 1 other identifier: TAK-935-5003
• Study Type: observational
• Target: 237
• Locations: 1 country
• Sites: 4

Brief Summary

The main aims of this study are to gather information about how many children, teenagers and adults in Spain have been diagnosed with Dravet syndrome and Lennox-Gastaut syndrome as well as to learn about the number of new Dravet syndrome and Lennox-Gastaut syndrome cases in persons in Spain. Participants' data will be taken from their medical records (charts), which were already collected as a part of their routine care in public hospitals in Spain between 01 January 2021 and 31 December 2022.

Conditions

Dravet Syndrome (DS); Lennox-Gastaut Syndrome (LGS)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (4)

• One-Year Period Prevalence of DS

  Number of participants with DS over a period of one year will be assessed.

  Up to 12 months

• Incidence of DS

  Number of new participants diagnosed annually with DS yearly.

  At two different 12 month periods (in consecutive years at Months 12 and 24)

• One-Year Period Prevalence of LGS Based on Stricter Diagnosis Criterion

  Number of participants with LGS over a period of one year based on stricter diagnosis criterion will be assessed.

  Up to 12 months

• Incidence of LGS Based on Stricter Diagnosis Criterion

  Number of new participants diagnosed annually with LGS based on stricter diagnosis criterion.

  At two different 12 month periods (in consecutive years at Months 12 and 24)

Secondary Outcomes (28)

• One-Year Period Prevalence of LGS Based on Wider Diagnosis Criterion

  Up to 12 months

• Incidence of LGS Based on Wider Diagnosis Criterion

  At two different 12 month periods (in consecutive years at Months 12 and 24)

• One-Year Period Prevalence of Paediatric DS

  Up to 12 months

• One-Year Period Prevalence of Paediatric LGS Based on Stricter and Wider Diagnoses Criteria

  Up to 12 months

• One-Year Period Prevalence of Adult DS

  Up to 12 months

Study Arms (1)

Participants With DS or LGS

Participants with a medical record of diagnosis with either DS or LGS that are treated in the participating hospitals and reside in the reference area of these hospitals will be included and data will be retrospectively collected for the period between 01 January 2021 to 31 December 2022.

Other: No intervention

Interventions

No intervention OTHER

As this is an observational study, no intervention will be administered.

Participants With DS or LGS

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants diagnosed with DS or LGS at public hospitals in Spain will be enrolled in the study.

You may qualify if:

• A. Diagnosis criteria for DS:
• All the following criteria must be met: i. Seizures onset within 1-20 months (usually within the first year of life). ii. Normal initial development prior to presentation (no cognitive or behavioural disability before the onset of seizures) followed by behaviour and cognitive impairment.
• iii. Recurrent focal clonic (hemiclonic) febrile and afebrile seizures (which often alternate sides from seizure to seizure), focal to bilateral tonicclonic, and/or generalized clonic seizures.
• And at least one of the following criteria must be met: i. Emergence of other seizure type, including atypical absence seizures, myoclonic seizures, atonic seizures, or non-tonic-clonic status epilepticus between 1-4 years.
• ii. Seizures triggered by fever due to illness or vaccinations, hot baths, sudden temperature changes, high level of activity, or by strong lighting or exposure to certain visual patterns.
• iii. Mutations or copy number variants in the SCN1A gene.
• B. Diagnosis criteria for LGS:
• Given the uncertainties associated with the diagnosis of this condition, two different criteria will be used, a stricter criterion, intended to identify "pure" Lennox-Gastaut syndrome participants, and a wider criterion, intended to also include the so-called Lennox-Gastaut-like participants.
• Lennox-Gastaut syndrome - stricter criteria:
• All the following criteria must be met: i. Seizures onset before 18 years of age, typically from 1 to 8 years. ii. Progressive development/cognition impairment after seizures onset. iii. Tonic seizures. iv. At least one additional seizure: generalised tonic-clonic seiures, atypical absence seizures, atonic seizures, myoclonic seizures, focal impaired awareness, epileptic spams, or non-convulsive status epilepticus v. Slow (\<2.5 hertz \[Hz\]) spike-and-wave EEG pattern. vi. Paroxysmal fast activity (10 Hz or greater) in sleep.
• Lennox-Gastaut syndrome - wider criteria:
• At least one the following criteria must be met:
• i. Tonic seizures. ii. Multiple types of seizures, including generalised tonic-clonic seizures, atypical absence seizures, atonic seizures, myoclonic seizures, myoclonic-atonic seizures, focal seizures, epileptic spams, or nonconvulsive status epilepticus.
• And at least one the following criteria must be met:
• i. Slow (\<2.5 Hz) spike-and-wave EEG pattern. ii. Paroxysmal fast activity (10 Hz or greater) in sleep.

You may not qualify if:

• Participants with epileptic condition other than DS or LGS.
• Participants with DS or LGS not residents in the reference area of the hospital.

Sponsors & Collaborators

• Takeda: lead

Study Sites (4)

Complejo Hospitalario Universitario de Vigo

Vigo, Pontevedra, 36312, Spain

Location

H. Universitario Infantil Niño Jesús

Madrid, 28009, Spain

Location

H. Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Universitari i Politècnic La Fe

Valencia, 46026, Spain

Location

Related Links

• To obtain more information on the study, click this link.

MeSH Terms

Conditions

Epilepsies, Myoclonic; Lennox Gastaut Syndrome

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2023
• First Posted: August 9, 2023
• Study Start: October 26, 2023
• Primary Completion: April 9, 2025
• Study Completion: April 10, 2025
• Last Updated: April 17, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

ES (4)