Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Pathophysiology Based Therapy of Early Onset Epileptic Encephalopathies (EE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date  ICMJE November 16, 2016
First Posted Date  ICMJE November 21, 2016
Last Update Posted Date April 18, 2019
Actual Study Start Date  ICMJE December 1, 2018
Estimated Primary Completion Date May 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 16, 2016)
Reduction of seizures [ Time Frame: one week ]
Reduction of epileptic seizures within one treatment phase to 50% compared to baseline
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02968966 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 16, 2016)
  • 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
  • Reduction of epileptic activities or suppression phases [ Time Frame: one week ]
    Reduction of epileptic activities or suppression phases in EEG
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pathophysiology Based Therapy of Early Onset Epileptic Encephalopathies
Official Title  ICMJE Pathophysiologie Basierte Therapie Von früh Beginnenden Epileptischen Enzephalopathien
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
Treatment A - if no positive response: Treatment B
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Seizure, Epileptic
Intervention  ICMJE 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
Study Arms  ICMJE 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..
Intervention: Other: Therapy regime
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 16, 2016)
25
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2020
Estimated Primary Completion Date May 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 12 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
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
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02968966
Other Study ID Numbers  ICMJE AKF 357-0-0
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party University Hospital Tuebingen
Study Sponsor  ICMJE University Hospital Tuebingen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Markus Wolff, Dr. University Children's Hospital Tübingen
PRS Account University Hospital Tuebingen
Verification Date October 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP