Summary

Eligibility
for people ages 2-80 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Ernesto Gonzalez-Giraldo, MD
Headshot of Ernesto Gonzalez-Giraldo
Ernesto Gonzalez-Giraldo

Description

Summary

The purpose of this study is to compare the efficacy of EPX-100 against placebo as adjunctive therapy in 60 patients with Dravet Syndrome.

Official Title

A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Patients With Dravet Syndrome

Details

This is a global, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy of EPX-100 (clemizole hydrochloride) in children and young adults with Dravet Syndrome. The 20-week study begins with a 4-week Observational Phase which will establish seizure frequency and eligibility for treatment, followed by a 4-week Titration Phase to identify the maximum tolerated dose in each patient. Thereafter, the patient will enter into a 12-week Maintenance Phase. Safety chemistries, hematology, urinalysis, pharmacokinetics, and ECG's will be monitored throughout the 20-week study. The patient will have the opportunity to enter a 52-week Open-Label Extension phase at the end of the Maintenance Phase in which all patients will be administered EPX-100. The primary endpoint is the median percent change in the 28-day in countable convulsive seizure frequency in the final 4 weeks of the 12-week treatment period (Maintenance Phase) relative to the baseline (4-week Observational Phase).

Keywords

Dravet Syndrome Clemizole Hydrochloride Convulsive Seizure Pediatric epilepsy Dravet Epilepsies, Myoclonic Syndrome EPX-100 (Clemizole HCl)

Eligibility

You can join if…

Open to people ages 2-80

  1. Clinical diagnosis of Dravet Syndrome. Patients must have seizures which are not completely controlled by anti-epileptic drugs (AEDs) with the following criteria:
  2. Onset of frequent seizures prior to 18 months of age,
  3. Normal development at onset,
  4. History of seizures that are generalized, unilateral clonic, and/or hemi-clonic,
  5. Brain MRI without cortical malformation, and
  6. Genetic mutation of the sodium voltage-gated channel alpha subunit 1 (SCN1A) gene must be documented.
  7. The patient must be approved to participate by the PI after a review of the medical history, baseline seizure calendars and inclusion/

You CAN'T join if...

. The Independent Reviewer will confirm Dravet Syndrome diagnosis for each participant enrolled in the study.

  1. The patient must be approved to participate by the PI after review of the medical history, baseline seizure calendars and inclusion/exclusion criteria. The Independent Reviewer will confirm Dravet Syndrome diagnosis for each participant enrolled in the study and adjudicate all seizure types on the seizure calendar.
  2. Seizure criteria of ≥4 countable convulsive seizures (tonic-clonic, tonic, clonic, atonic, focal with observable motor signs) per 4-week baseline period (Observational Phase).
  3. Patients should be on a stable regimen of AEDs ≥30days prior to Visit 1 and generally in good health.
  4. Parent or Legal Authorised Representative (LAR) is willing and able to maintain an accurate and complete daily seizure calendar.
  5. Sexually active women of child-bearing potential (WCBP) must be using a medically acceptable method of birth control and have a negative urine pregnancy test at the screening visit. A WCBP is defined as a female who is biologically capable of becoming pregnant. A medically acceptable method of birth control includes intrauterine devices in place for at least 3 months, surgical sterilization, or adequate barrier methods (e.g., diaphragm and foam). Use of oral contraceptives in combination with another method (e.g., a spermicidal cream) is acceptable. In participants who are not sexually active, abstinence is an acceptable form of birth control and urine will be tested per protocol. Women who are of non-child-bearing potential, i.e., post-menopause, must have this condition captured in their medical history. Pregnant women are excluded from this study.
  6. Have parent or LAR available and willing to give written informed consent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.

Exclusion Criteria:

The presence of any of the following excludes a patient from study enrollment:

  1. Known sensitivity, allergy, or previous exposure to EPX-100 (clemizole HCl).
  2. Exposure to any investigational drug or device <90 days prior to screening or plans to participate in another drug or device trial at any time during the study.
  3. Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease.
  4. Concurrent use of drugs known to interfere with EPX-100, including moderate or severe inducers or inhibitors of CYP3A4/5/7. Specifically, concurrent use of carbamazepine, oxcarbazepine, phenytoin, and/or phenobarbital are excluded, as well as refraining from grapefruits and grapefruit juice during the study period.
  5. Prior or concurrent use of fenfluramine or lorcaserin.
  6. Has any medical condition that, in the Investigator's judgement, is considered to be clinically significant and could potentially affect patient safety or study outcome, including but not limited to: clinically significant cardiac disease (including angina, congestive heart failure, uncontrolled hypertension, and history of arrhythmias), renal, pulmonary, gastrointestinal, hematologic or hepatic conditions; or a condition that affects the absorption, distribution, metabolism, or excretion of drugs.
  7. Has an active suicidal plan/intent or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 3 years.

Locations

  • UCSF Medical Center accepting new patients
    San Francisco California 94158 United States
  • Child Neurology Consultants of Austin accepting new patients
    Austin Texas 78757 United States

Lead Scientist at UCSF

  • Ernesto Gonzalez-Giraldo, MD
    Dr. Ernesto Gonzalez-Giraldo is a pediatric neurologist who cares for children with epilepsy. He specializes in treating patients whose seizures have failed to respond to initial anti-seizure medications. He evaluates patients for epilepsy surgery, including the placement of devices that treat seizures by stimulating the vagus nerve or by responsive neurostimulation.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Epygenix
ID
NCT04462770
Phase
Phase 2 Research Study
Study Type
Interventional
Participants
Expecting 60 study participants
Last Updated