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Trial record 56 of 107 for:    PHENYTOIN

Pathophysiology Based Therapy of Early Onset Epileptic Encephalopathies (EE)

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ClinicalTrials.gov Identifier: NCT02968966
Recruitment Status : Recruiting
First Posted : November 21, 2016
Last Update Posted : April 18, 2019
Sponsor:
Information provided by (Responsible Party):
University Hospital Tuebingen

Brief 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. 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.

Condition or disease Intervention/treatment Phase
Seizure, Epileptic Other: Therapy regime Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Treatment A - if no positive response: Treatment B
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pathophysiologie Basierte Therapie Von früh Beginnenden Epileptischen Enzephalopathien
Actual Study Start Date : December 1, 2018
Estimated Primary Completion Date : May 2020
Estimated Study Completion Date : August 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy Seizures

Arm Intervention/treatment
Experimental: Therapy regime
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..
Other: Therapy regime
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.
Other Name: two medical products given in a predefined order




Primary Outcome Measures :
  1. Reduction of seizures [ Time Frame: one week ]
    Reduction of epileptic seizures within one treatment phase to 50% compared to baseline


Secondary Outcome Measures :
  1. Reduction of seizures stratified for genetic background [ Time Frame: one week ]
    Reduction of epileptic seizures within one treatment phase to 50% compared to baseline stratified for three gene mutations

  2. Reduction of epileptic activities or suppression phases [ Time Frame: one week ]
    Reduction of epileptic activities or suppression phases in EEG



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Ages Eligible for Study:   up to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02968966


Contacts
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Contact: Markus Wolff, Dr. 0049-7071-81391 markus.wolff@med.uni-tuebingen.de
Contact: Yvonne Weber, Prof. 0049-7071-80443 yvonne.weber@med.uni-tuebingen.de

Locations
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Germany
Universtiy Hospital Recruiting
Tübingen, Germany, 72076
Contact: Michael Alber, Dr.       michael.alber@med.uni-tuebingen.de   
Contact: Hanna Küpper, Dr.       hanna.kuepper@med.uni-tuebingen.de   
Sponsors and Collaborators
University Hospital Tuebingen
Investigators
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Principal Investigator: Markus Wolff, Dr. University Children's Hospital Tübingen

Study Data/Documents: Synopsis  This link exits the ClinicalTrials.gov site

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Responsible Party: University Hospital Tuebingen
ClinicalTrials.gov Identifier: NCT02968966     History of Changes
Other Study ID Numbers: AKF 357-0-0
First Posted: November 21, 2016    Key Record Dates
Last Update Posted: April 18, 2019
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Seizures
Epilepsy
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Diseases
Central Nervous System Diseases