Seizure, EpilepticPhase 2EENCT02968966
Pathophysiology Based Therapy of Early Onset Epileptic Encephalopathies
A Phase 2 study testing Therapy regime in people with Seizure, Epileptic, run by University Hospital Tuebingen at 1 site in Germany.
Trial summary
Genetic epileptic encephalopathies (EEs) are a group of very rare and severe, pharmaco-resistant epilepsy forms characterized by an early onset, e.g. first years of life, and an often severe developmental delay. Read more about this study
- Who is it for?
- Ages Up to 12 months
- Conditions the study is for
- Seizure, Epileptic
- What is being tested?
- Therapy regime
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteGermany
- Study dates
- 1 Dec 2018to Aug 2020
- Phase
- Phase 2
- 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 Up to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- highly active epilepsy (≥ 1 seizure per day)
- epilepsy with onset 0-3 months of age
- pharmaco-resistant epilepsy (2 or more standard anticonvulsive medications tried before)
- recently max. two stable anticonvulsive drugs for minimum 4 days before study start
- patients under continuous monitoring control
- patients younger than 1 year of age
Who cannot join
- high grade cardial rhythm disorders
- severe liver, renal and electrolyte blood parameter changes
- metabolic or lesional origin of epilepsy (metabolic screening results and cranial MRI available)
- parallel participation in other studies (must be finished two month before study start)
- missing informed consent
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pathophysiologie Basierte Therapie Von früh Beginnenden Epileptischen Enzephalopathien
Genetic epileptic encephalopathies (EEs) are a group of very rare and severe, pharmaco-resistant epilepsy forms characterized by an early onset, e.g. first years of life, and an often severe developmental delay. Genetic defects were found in different ion channels such as potassium or sodium channels explaining well the pathological neuronal hyperexcitability leading to seizures. Further mutations were also found in proteins relevant for cell structure, DNA/RNA processing or the synaptic vesicular metabolism. Specific and individualized therapies have not been established neither in the clinical routine nor in controlled studies. The goal of this monocentric non-blinded non-placebo controlled phase IIb study is the evaluation of the effectivity of anticonvulsive drugs specifically working on the ion channels defective in some subtypes of EEs in order to establish a standard and individualized therapy for these rare diseases based on the specific genetic defect.
Read the detailed description
During the study, the sodium channel blockers phenytoin and lacosamide and the potassium channel blocker kinidinsulfate will be given under standardized conditions to patients with an early onset and pharmaco-resistant genetic epilepsy with and without mutations in the potassium channels KCNT1 and KCNQ2 and the sodium channel gene SCN2A. The primary endpoint will be a significant seizure reduction under trial medication compared to baseline. Secondary endpoints will be the improvement of electroencephalographic characteristics of the respective EEs.
- Conditions
- Seizure, Epileptic
- Being tested
- Therapy regime
- Medical subject headings
- Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Therapy regimeOther
Also called two medical products given in a predefined order
Patient will receive Phenytoin, if no success is obtained, Vimpat is given. In case of success after one of the treatments, the endpoint is reached. Success is defined as reduction of seizures to 50% compared to baseline.
| Study group | Role | What they receive |
|---|---|---|
| Therapy regime | Experimental | Two medical drugs will be administered in a predefined order (1. Phenhydan® (Phenytoin), 2. Lacosamide (Vimpat®) to investigate whether this enables an effective reduction of seizures in early onset epileptic encephalopathies.. Receives: Other: Therapy regime |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of seizures
Reduction of epileptic seizures within one treatment phase to 50% compared to baseline
Measured: one week
Secondary outcomes (2)
Reduction of seizures stratified for genetic background
Reduction of epileptic seizures within one treatment phase to 50% compared to baseline stratified for three gene mutations
Measured: one week
Reduction of epileptic activities or suppression phases
Reduction of epileptic activities or suppression phases in EEG
Measured: one week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2016 (estimated)
- Study start1 Dec 2018
- Primary completionMay 2020 (estimated)
- Study completionAug 2020 (estimated)
Why it stopped: no patients recruited
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Seizure, EpilepticStudies of the same condition, open and under way.
- More trials run by University Hospital TuebingenThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Treatment A - if no positive response: Treatment B
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Markus Wolff, Dr., Principal investigator, University Children's Hospital Tübingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02968966
- Sponsor's protocol ID
- AKF 357-0-0
- Acronym
- EE
- Status last verified
- Feb 2020
- First submitted
- 16 Nov 2016
- Last updated
- 7 Feb 2020
- Results posted
- No
- Data monitoring committee
- Yes
- 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 7 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pathophysiologie Basierte Therapie Von früh Beginnenden Epileptischen Enzephalopathien
- ClinicalTrials.gov ID
- NCT02968966
- Acronym / sponsor ID
- EE / AKF 357-0-0
- Conditions
- Seizure, Epileptic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: no patients recruited
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- 1 Dec 2018 (actual)
- Primary completion
- May 2020 (estimated)
- Study completion
- Aug 2020 (estimated)
- First posted
- 21 Nov 2016 (estimated)
- 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.
Genetic epileptic encephalopathies (EEs) are a group of very rare and severe, pharmaco-resistant epilepsy forms characterized by an early onset, e.g. first years of life, and an often severe developmental delay. Genetic defects were found in different ion channels such as potassium or sodium channels explaining well the pathological neuronal hyperexcitability leading to seizures. Further mutations were also found in proteins relevant for cell structure, DNA/RNA processing or the synaptic vesicular metabolism. Specific and individualized therapies have not been established neither in the clinical routine nor in controlled studies. The goal of this monocentric non-blinded non-placebo controlled phase IIb study is the evaluation of the effectivity of anticonvulsive drugs specifically working on the ion channels defective in some subtypes of EEs in order to establish a standard and individualized therapy for these rare diseases based on the specific genetic defect.
Detailed description
During the study, the sodium channel blockers phenytoin and lacosamide and the potassium channel blocker kinidinsulfate will be given under standardized conditions to patients with an early onset and pharmaco-resistant genetic epilepsy with and without mutations in the potassium channels KCNT1 and KCNQ2 and the sodium channel gene SCN2A. The primary endpoint will be a significant seizure reduction under trial medication compared to baseline. Secondary endpoints will be the improvement of electroencephalographic characteristics of the respective EEs.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Treatment A - if no positive response: Treatment B
| Arm | Type | Receives |
|---|---|---|
| Therapy regime | Experimental | Other: Therapy regimeTwo medical drugs will be administered in a predefined order (1. Phenhydan® (Phenytoin), 2. Lacosamide (Vimpat®) to investigate whether this enables an effective reduction of seizures in early onset epileptic encephalopathies.. |
Who may qualify
- Ages
- Up to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- highly active epilepsy (≥ 1 seizure per day)
- epilepsy with onset 0-3 months of age
- pharmaco-resistant epilepsy (2 or more standard anticonvulsive medications tried before)
- recently max. two stable anticonvulsive drugs for minimum 4 days before study start
- patients under continuous monitoring control
Key exclusion criteria (5 listed)
- high grade cardial rhythm disorders
- severe liver, renal and electrolyte blood parameter changes
- metabolic or lesional origin of epilepsy (metabolic screening results and cranial MRI available)
- parallel participation in other studies (must be finished two month before study start)
- missing informed consent
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 |
|---|---|---|
| Therapy regimeAlso: two medical products given in a predefined orderPatient will receive Phenytoin, if no success is obtained, Vimpat is given. In case of success after one of the treatments, the endpoint is reached. Success is defined as reduction of seizures to 50% compared to baseline. | Other | Therapy regime |
Primary outcome
Reduction of seizures
Time frame: one week
Reduction of epileptic seizures within one treatment phase to 50% compared to baseline
2 secondary outcomes
Reduction of seizures stratified for genetic background
Time frame: one week
Reduction of epileptic activities or suppression phases
Time frame: one week
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2020.
- 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.
Markus Wolff, Dr. principal investigator
University Children's Hospital Tübingen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Therapy regime in people with Seizure, Epileptic, run by University Hospital Tuebingen at 1 site in Germany.
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 6 inclusion and 5 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: withdrawn, as of the sponsor's last update on 7 Feb 2020. 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 Germany. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reduction of seizures (time frame: one week). 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 7 Feb 2020.