EpilepsyEPIMARKERNCT03486366
Application of Novel Diagnostic and Therapeutical Methods in Epilepsy and Neurodevelopmental Abnormalities in Children.
A study testing non interventional trial in people with Epilepsy, run by Medical University of Warsaw at 1 site in Poland.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 day to 16 yearsHealthy volunteers can take part
- Conditions the study is for
- Epilepsy
- What is being tested?
- non interventional trial
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 sitePoland
- Study dates
- 1 Apr 2017to 30 Mar 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 16 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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,
Show all 9Show fewer
- aged up to 16 years,
- seizure free, in whom a decision to withdraw antiepileptic drugs was made,
- written informed consent of caregivers.
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
Poland1 site, 1 recruiting
- Recruiting
Medical University of Warsaw
Warsaw, 05-091
Sergiusz Jóźwiak, Professor, +48 606 445 668, sergiusz.jozwiak@wum.edu.pl
Katarzyna Kotulska-Jóźwiak, Professor, +48 607 281 798, kotulska.jozwiak@gmail.com
About this study
Official title: Application of Novel Diagnostic and Therapeutical Methods in Epilepsy and Neurodevelopmental Abnormalities in Children Based on the Clinical and Cellular Model of the Mammalian Target of Rapamycin - mTOR Dependent Epilepsy.
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.
Read the detailed description
The clinical part of EPIMARKER is composed of 2 prospective studies (WP1 and WP2) of epilepsy progress in Tuberous Sclerosis Complex -TSC children, performed in 2 sites: Medical University of Warsaw -WUM and Children's Memoroal Health Institute - IPCZD, Poland.
WP1 and WP2 are designed to conduct the studies, but the resulting data and samples will be analyzed and used in Workpackage 3-12 (WP3-12).
CLINICAL STUDY in WP1 Inclusion criteria: 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.
Study overview: Each subject will be followed for 12 month. Epilepsy will be tracked with serial video EEG (vEEG) recordings and clinical investigations performed every 3 months. Apart from medical history of the patients, data from seizure diary, neuroimaging studies, and neuropsychological examinations will be collected. Blood samples for biomarkers studies will be collected at study entry, at the onset of drug- resistant seizures or after 6 months, whichever is applicable, and at the end of follow-up in all patients participating in the project. The data obtained in children responding, to standard and with drug- resistant seizures will be compare.
Sample size: We plan to enroll 60 TSC patients into WP1 of Epimarker in 12 months. Based upon our preliminary results and extensive experience with TSC, we predict that about 50% of patients will develop drug- resistant seizures, while 50% will respond to standard treatment (Jozwiak 2011) Study endpoints: the primary clinical endpoint of this study is a collection of a set of clinical, molecular, and EEG source data in all subjects.
CLINICAL STUDY in WP2 Inclusion criteria: 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, written informed consent of caregivers.
Study overview: Each subject will be followed for 12 months. Antiepileptic drugs will be withdrawn within 3 months in all subjects starting, at study entry. Epilepsy will be tracked by means of serial video EEG recordings and clinical investigations performed every 3 months. Apart from medical history of the patients, data from seizures diary, neuroimaging studies and neuropsychological examinations will be collected. Patients with recurrent seizures will receive antiepileptic treatment according to current standards. Blood samples for biomarkers study will be collected at study entry, at the end of drugs withdrawal, at the onset recurrent seizures and at the end of follow-up in all patient participating in the project. The data obtained in children seizure free at the end of follow-up and patients with recurrent seizures will be compare.
Sample size: We plan to enroll 60 TSC patients into WP2 of Epimarker in 12 months. Based upon our preliminary results and extensive experience with TSC, we predict that about 50% of patients will develop recurrent seizures, while 50% remain seizure free (unpublished data).
Study endpoints: the primary clinical endpoint of this study is a collection of a set of clinical, molecular, and EEG source data in all subjects.
- Conditions
- Epilepsy
- Being tested
- non interventional trial
- Keywords
- epilepsy, TSC, biomarkers, mTOR pathway
- Medical subject headings
- Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
non interventional trialOther
Also called other
other
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: non interventional trial |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2020
Secondary outcomes (1)
Cellular model of developing TSC brain
EPIMARKER will develop a innovative human cellular model of epilepsy in developing brain, which can be used for epilepsy biomarkers analyses and for investigations on novel therapeutic strategies in epilepsy.
Measured: 2020
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2018
- Study start1 Apr 2017
- Primary completion30 Mar 2020 (estimated)
- Study completion30 Mar 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EpilepsyStudies of the same condition, open and under way.
- More trials run by Medical University of WarsawThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of Warsaw
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Memorial Health Institute, Poland
- Responsible party
- Principal investigator, Sergiusz Jozwiak, Medical University of Warsaw
- Investigators
- Sergiusz Jóźwiak, professor, Study chair, Medical University of Warsaw
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03486366
- Sponsor's protocol ID
- Strategmed3/306306/4/NCBR/2017
- Acronym
- EPIMARKER
- Status last verified
- Mar 2018
- First submitted
- 26 Mar 2018
- Last updated
- 3 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Application of Novel Diagnostic and Therapeutical Methods in Epilepsy and Neurodevelopmental Abnormalities in Children Based on the Clinical and Cellular Model of the Mammalian Target of Rapamycin - mTOR Dependent Epilepsy.
- ClinicalTrials.gov ID
- NCT03486366
- Acronym / sponsor ID
- EPIMARKER / Strategmed3/306306/4/NCBR/2017
- Conditions
- Epilepsy
- Keywords
- epilepsy, TSC, biomarkers, mTOR pathway
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Medical University of WarsawUniversity, hospital or other
- Collaborators
- Children's Memorial Health Institute, Poland
- Study start
- 1 Apr 2017 (actual)
- Primary completion
- 30 Mar 2020 (estimated)
- Study completion
- 30 Mar 2020 (estimated)
- First posted
- 3 Apr 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The clinical part of EPIMARKER is composed of 2 prospective studies (WP1 and WP2) of epilepsy progress in Tuberous Sclerosis Complex -TSC children, performed in 2 sites: Medical University of Warsaw -WUM and Children's Memoroal Health Institute - IPCZD, Poland.
WP1 and WP2 are designed to conduct the studies, but the resulting data and samples will be analyzed and used in Workpackage 3-12 (WP3-12).
CLINICAL STUDY in WP1 Inclusion criteria: 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.
Study overview: Each subject will be followed for 12 month. Epilepsy will be tracked with serial video EEG (vEEG) recordings and clinical investigations performed every 3 months. Apart from medical history of the patients, data from seizure diary, neuroimaging studies, and neuropsychological examinations will be collected. Blood samples for biomarkers studies will be collected at study entry, at the onset of drug- resistant seizures or after 6 months, whichever is applicable, and at the end of follow-up in all patients participating in the project. The data obtained in children responding, to standard and with drug- resistant seizures will be compare.
Sample size: We plan to enroll 60 TSC patients into WP1 of Epimarker in 12 months. Based upon our preliminary results and extensive experience with TSC, we predict that about 50% of patients will develop drug- resistant seizures, while 50% will respond to standard treatment (Jozwiak 2011) Study endpoints: the primary clinical endpoint of this study is a collection of a set of clinical, molecular, and EEG source data in all subjects.
CLINICAL STUDY in WP2 Inclusion criteria: 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, written informed consent of caregivers.
Study overview: Each subject will be followed for 12 months. Antiepileptic drugs will be withdrawn within 3 months in all subjects starting, at study entry. Epilepsy will be tracked by means of serial video EEG recordings and clinical investigations performed every 3 months. Apart from medical history of the patients, data from seizures diary, neuroimaging studies and neuropsychological examinations will be collected. Patients with recurrent seizures will receive antiepileptic treatment according to current standards. Blood samples for biomarkers study will be collected at study entry, at the end of drugs withdrawal, at the onset recurrent seizures and at the end of follow-up in all patient participating in the project. The data obtained in children seizure free at the end of follow-up and patients with recurrent seizures will be compare.
Sample size: We plan to enroll 60 TSC patients into WP2 of Epimarker in 12 months. Based upon our preliminary results and extensive experience with TSC, we predict that about 50% of patients will develop recurrent seizures, while 50% remain seizure free (unpublished data).
Study endpoints: the primary clinical endpoint of this study is a collection of a set of clinical, molecular, and EEG source data in all subjects.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Workpackage1 WP1 | Other | Other: non interventional trialNon 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. |
| Workpackage2 WP2 | Other | Other: non interventional trialNon 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. |
Who may qualify
- Ages
- 1 day to 16 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| non interventional trialAlso: otherother | Other | Workpackage1 WP1, Workpackage2 WP2 |
Primary outcome
EEG and molecular data collection and integration into clinically applicable tool
Time frame: 2020
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.
1 secondary outcome
Cellular model of developing TSC brain
Time frame: 2020
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sergiusz Jóźwiak, professor study chair
Medical University of Warsaw
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing non interventional trial in people with Epilepsy, run by Medical University of Warsaw at 1 site in Poland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sergiusz Jóźwiak, professor (+48 606 445 668, sergiusz.jozwiak@wum.edu.pl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
EEG and molecular data collection and integration into clinically applicable tool (time frame: 2020). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2018.