Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex
EPISTOP
2 other identifiers
interventional
100
8 countries
9
Brief Summary
The primary objective of clinical part of EPISTOP project is to identify the clinical and molecular biomarkers of epileptogenesis in a prospective clinical study of patients with TSC. Secondary objective of the clinical part of EPISTOP is to compare the effects of standard antiepileptic treatment in patients diagnosed as having epilepsy after clinical seizures vs after electroencephalographic epileptiform discharges, in a randomized trial in TSC patients.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for not_applicable
Started Nov 2013
Longer than P75 for not_applicable
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2013
CompletedFirst Submitted
Initial submission to the registry
March 25, 2014
CompletedFirst Posted
Study publicly available on registry
March 28, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2018
CompletedApril 4, 2014
April 1, 2014
4.9 years
March 25, 2014
April 3, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
number of patients with epilepsy
Full analysis set comprises all patients participating in the study, including the control group. This set will be divided into subsets: control group, TSC patients with epilepsy, and TSC patients with no epilepsy. Among TSC patients with epilepsy, patients with well-controlled seizures and patients with drug-resistant epilepsy will be identified. In full analysis set the blood biomarkers will be analysed. Clinical analysis set will comprise of all TSC infants enrolled in the study and the clinical biomarkers of epileptogenesis (neuroimaging, vEEG, data from medical history) will be analysed in this set. Treatment analysis set will comprise of infants participating in the randomized part of the study and the efficacy of antiepileptic treatment in respect to the point of epilepsy diagnosis (electroencephalographic epileptiform discharges onset in group A and clinical seizures onset in group B) will be assessed in this set.
at 24 month of life
Study Arms (2)
epilepsy early diagnosis
EXPERIMENTALInfants that have epileptiform discharges on vEEG and no clinical seizures, if their parents/caregivers give consent, will enter the randomized part of the study. Those children will be randomized into two groups: group A will be diagnosed as having epilepsy after subclinical (electroencephalographic) epileptiform discharges, and the patients in group B will be diagnosed as epileptic after clinical seizures appear. All infants diagnosed with epilepsy will receive standard therapy with recommended first line antiepileptic drug starting from the day of diagnosis.
standard epilepsy diagnosis
EXPERIMENTALInfants that have epileptiform discharges on vEEG and no clinical seizures, if their parents/caregivers give consent, will enter the randomized part of the study. Those children will be randomized into two groups: group A will be diagnosed as having epilepsy after subclinical (electroencephalographic) epileptiform discharges, and the patients in group B will be diagnosed as epileptic after clinical seizures appear. All infants diagnosed with epilepsy will receive standard therapy with recommended first line antiepileptic drug starting from the day of diagnosis.
Interventions
Infants that have epileptiform discharges on vEEG and no clinical seizures, if their parents/caregivers give consent, will enter the randomized part of the study. Those children will be randomized into two groups: group A will be diagnosed as having epilepsy after subclinical (electroencephalographic) epileptiform discharges (based on epilepsy early diagnosis protocol), and the patients in group B will be diagnosed as epileptic after clinical seizures appear. All infants diagnosed with epilepsy will receive standard therapy with recommended first line antiepileptic drug starting from the day of diagnosis.
Eligibility Criteria
You may qualify if:
- male or female infants with a definite diagnosis of TSC (Roach criteria; Roach 1998 or DNA confirmed),
- age up to 4 months at the moment of enrolment,
- no clinical seizures seen by caregivers or on baseline videoEEG recording,
- written informed consent of caregivers. It is possible to give consent for the observational part of the study only. In this case, the child will not enter the randomized part of the study.
- male or female infants who have undergone routine MRI for reasons other than epilepsy and brain tumor or cortical defects,
- age up to 24 months at the moment of study entry,
You may not qualify if:
- any type of seizures observed till baseline visit,
- antiepileptic treatment at or prior to study entry,
- contraindications to MRI,
- any severe and/or uncontrolled medical condition that is considered by the investigator as possibly affecting the EPISTOP analyses or procedures.
- any sign or symptom suggesting TSC diagnosis,
- any type of seizures observed at study entry,
- antiepileptic treatment at study entry,
- history of seizures, with the exception of febrile seizures,
- any severe and/or uncontrolled medical condition that is considered by the investigator as possibly affecting the EPISTOP analyses or procedures.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Sergiusz Jozwiaklead
- University of Rome Tor Vergatacollaborator
- Institut National de la Santé Et de la Recherche Médicale, Francecollaborator
- Vrije Universiteit Brusselcollaborator
- General University Hospital, Praguecollaborator
- Charite University, Berlin, Germanycollaborator
- KU Leuvencollaborator
- Medical University of Viennacollaborator
- Rudolf Magnus Institute - University of Utrechtcollaborator
- Brigham and Women's Hospitalcollaborator
Study Sites (9)
Medizinische Universitaet Wien
Vienna, Austria
Vrije Universiteit Brussel
Brussels, Belgium
Katholieke Universiteit Leuven
Leuven, Belgium
Fakultni Nemocnice V Motole
Prague, Czechia
Institut National De La Sante et de la Recherche Medicale
Paris, France
Charite - Universitaetsmedizin Berlin
Berlin, Germany
Universita Degli Studi Di Roma Tor Vergata
Rome, Italy
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
Children's Memorial Health Institute
Warsaw, Poland
Related Publications (1)
van der Poest Clement E, Jansen FE, Braun KPJ, Peters JM. Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex. Paediatr Drugs. 2020 Feb;22(1):73-84. doi: 10.1007/s40272-019-00376-0.
PMID: 31912454DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Seriusz Jozwiak, Md, PhD
Children's Memorial Health Institute
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- DIAGNOSTIC
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- MD, PhD
Study Record Dates
First Submitted
March 25, 2014
First Posted
March 28, 2014
Study Start
November 1, 2013
Primary Completion
October 1, 2018
Study Completion
October 1, 2018
Last Updated
April 4, 2014
Record last verified: 2014-04