Pathophysiology Based Therapy of Early Onset Epileptic Encephalopathies

NCT02968966 | Withdrawn Phase 2 DMC

• 1 other identifier: AKF 357-0-0
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Seizure, Epileptic

Outcome Measures

Primary Outcomes (1)

• Reduction of seizures

  Reduction of epileptic seizures within one treatment phase to 50% compared to baseline

  one week

Secondary Outcomes (2)

• Reduction of seizures stratified for genetic background

  one week

• Reduction of epileptic activities or suppression phases

  one week

Study Arms (1)

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..

Other: Therapy regime

Interventions

Therapy regime OTHER

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.

Also known as: two medical products given in a predefined order

Therapy regime

Eligibility Criteria

• Age: Up to 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital Tuebingen: lead

Study Sites (1)

Universtiy Hospital

Tübingen, 72076, Germany

Location

MeSH Terms

Conditions

Seizures

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Markus Wolff, Dr.

  University Children's Hospital Tübingen

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Treatment A - if no positive response: Treatment B

Study Record Dates

• First Submitted: November 16, 2016
• First Posted: November 21, 2016
• Study Start: December 1, 2018
• Primary Completion: May 1, 2020
• Study Completion: August 1, 2020
• Last Updated: February 7, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)