Application of Novel Diagnostic and Therapeutical Methods in Epilepsy and Neurodevelopmental Abnormalities in Children.

NCT03486366 | Unknown

• 1 other identifier: Strategmed3/306306/4/NCBR/2017
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

Epilepsy affects 1% of the world's population and 6 million people in Europe. The estimated total cost of €20 billion in Europe in 2014 makes epilepsy a significant socioeconomic burden. Despite great progress in the management of epilepsy and increasing numbers of antiepileptic drugs, 30-40% of epilepsy patients are refractory to all available medications. Moreover, in childhood epilepsy is a causative factor of psychiatric and behavioral comorbidities, including developmental delay and autism spectrum disorder. In spite of multiple trials no reliable biomarker of epilepsy development has been identified. There are no studies on biomarkers of drug-resistance or epilepsy recurrence after the drug withdrawal. EPIMARKER is a first project, carried out in humans, which is going to examine in prospective way clinical, electroencephalographic and molecular biomarkers to produce an integrative tool useful in everyday diagnosis and treatment of epilepsy in children to prevent the development of drug-resistant epilepsy and its behavioral comorbidities as mental retardation and autism. The set of molecular biomarkers will be determined by quantitative transcriptomic and proteomic studies and validated in reprogrammed cellular models.

Conditions

Epilepsy

Keywords

epilepsy, TSC, biomarkers, mTOR pathway

Outcome Measures

Primary Outcomes (1)

• EEG and molecular data collection and integration into clinically applicable tool

  The EEG algorithm will include the data from automatic statistical analysis of prospectively recorded EEG signal and comprehensive clinical investigations. To achieve this goal, a special automatic system to detect and count clinically important EEG features will be developed.

  2020

Secondary Outcomes (1)

• Cellular model of developing TSC brain

  2020

Study Arms (2)

Workpackage1 WP1

OTHER

Non interventional trial: male or female children with a definite diagnosis of TSC (Roach 1998), aged up to 4 years, diagnosis of epilepsy established on the basis of clinical seizures or epileptiform changes on EEG within 1-7 days prior to baseline. We plan to enroll 60 TSC patients into WP1 to Epimarker in 12 months.

Other: non interventional trial

Workpackage2 WP2

OTHER

Non interventional trial: male or female children with a definite diagnosis of TSC (Roach criteria: Roach 1998) and epilepsy, aged up to 16 years, seizure free, in whom a decision to withdraw antiepileptic drugs was made. We plan to enroll 60 TSC patients into WP2 to Epimarker in 12 months. The data obtained in children seizure free at the end of follow-up and patients with recurrent seizures will be compare.

Other: non interventional trial

Interventions

non interventional trial OTHER

other

Also known as: other

Workpackage1 WP1; Workpackage2 WP2

Eligibility Criteria

• Age: 1 Day - 16 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• male or female children with a definite diagnosis of TSC (Roach 1998),
• aged up to 4 years,
• diagnosis of epilepsy established on the basis of clinical seizures or
• epileptiform changes on EEG within 1-7 days prior to baseline ,
• written informed consent of caregivers.
• male or female children with a definite diagnosis of TSC (Roach criteria: Roach 1998) with epilepsy,
• aged up to 16 years,
• seizure free, in whom a decision to withdraw antiepileptic drugs was made,
• written informed consent of caregivers.

Sponsors & Collaborators

• Medical University of Warsaw: lead
• Children's Memorial Health Institute, Poland: collaborator

Study Sites (1)

Medical University of Warsaw

Warsaw, 05-091, Poland

RECRUITING

MeSH Terms

Conditions

Epilepsy

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Sergiusz Jóźwiak, professor

  Medical University of Warsaw

  STUDY CHAIR

Central Study Contacts

Sergiusz Jóźwiak, professor

CONTACT

+48 606 445 668; sergiusz.jozwiak@wum.edu.pl

Katarzyna Kotulska- Jóźwiak, professor

CONTACT

+ 48 607 281 798; kotulska.jozwiak@gmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor Head of Department Neurology in Children Hospital

Model Details: With EPIMARKER we aim to develop clinically useful tools enabling accurate and early detection of risk of drug resistant or recurrent seizures and stablish the new therapeutic and preventative possibilities for epilepsy. The Epimarker algorithms will include selected data obtained in 3 unique models of epilepsy in developing brain. * EEG recordings and clinical data from a prospective study of drug-resistant epilepsy development in young children with TSC, * EEG recording and clinical data from a prospective study of epilepsy after antiepileptic drug withdrawal in children with TSC * Molecular biomarkers in both above clinical setting, established by means of a broad range of molecular analyses, including a diverse set of cutting edge analyses of blood samples, that will be obtained at study entry, at the onset of drug- resistance or recurrent epilepsy, and after 12-month follow-up and the verified in a Epimarker cellular model.

Study Record Dates

• First Submitted: March 26, 2018
• First Posted: April 3, 2018
• Study Start: April 1, 2017
• Primary Completion: March 30, 2020
• Study Completion: March 30, 2020
• Last Updated: April 3, 2018

Record last verified: 2018-03

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1)