Dravet Syndrome MedDRA version: 20.0 Level: LLT Classification code 10073682 Term: Dravet syndrome System Organ Class: 100000004850
Conditions
Sponsors
Epygenix Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Eligible participants must fulfill the following inclusion criteria: 1. Male and female participants 2 years and older at time of consent. 2. Participant or parent/Legally Authorized Representative (LAR) willing and able to provide written informed consent, assent (if applicable) prior to initiation of any study related procedures. 3. Clinical diagnosis of Dravet Syndrome. Participants must have seizures which are not completely controlled by AEDs with the following criteria: • Onset of seizures prior to 18 months of age • Normal development at onset, • History of seizures that are generalized, unilateral clonic, and/or hemiclonic, • Brain MRI without cortical malformation (not including mild atrophy associated with the natural progression of Dravet Syndrome), and • Genetic mutation of the SCN1A gene must be documented. 4. The participant must be approved to participate by the Independent Reviewer, in collaboration with the PI. Participants will be approved for participation following review of the participant’s medical and seizure history, historical neuroimaging, historical EEGs, genetic report confirming SCN1A mutation, and review and classification of at least 28 days of baseline seizures. 5. =4 countable convulsive seizures within minimum 28-day screening/baseline period (e.g., hemiclonic, secondarily generalized tonic-clonic, generalized tonic-clonic, tonic, clonic, tonic/atonic (resulting in a drop), and focal with clear observable motor signs). 6. Participants should be on a stable regimen of AEDs =30 days prior to Visit 1 and generally in good health. 7. Participant or parent/ LAR is able and willing to maintain an accurate and complete daily seizure and medication diary for the duration of the trial. 8. Sexually active women of child-bearing potential (WCBP) must be using a medically acceptable method of birth control and have a negative serum or urine pregnancy test at the screening (Visit 1) and Randomization (Visit 2). 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. Are the trial subjects under 18? yes Number of subjects for this age range: 85 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 15 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: The presence of any of the following excludes a participant from study enrollment: 1. Known sensitivity, allergy, or previous exposure to EPX-100 (clemizole HCl). 2. Exposure to any investigational drug or device 35mmHg 7. Has any medical condition that, in the PI's judgment, is considered to be clinically significant and could potentially affect participant 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. 8. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of EPX-100 orally in divided doses as adjunctive therapy compared with placebo in participants with Dravet Syndrome, in terms of the mean percent change in countable convulsive seizure frequency (CCSF1) in the Titration and Maintenance (T+M) periods relative to baseline.;Secondary Objective: To evaluate the difference between EPX100 vs placebo in proportion of participants with >25% reduction in the mean CCSF1 in the T+M periods relative to baseline. To evaluate the difference between EPX100 vs placebo in proportion of participants with >50% reduction in the mean CCSF1 in the T+M periods relative to baseline. To evaluate the mean difference between EPX-100 vs placebo per 28-days in the percent change of all seizures in the T+M periods relative to the baseline. To describe the difference between EPX100 vs placebo in the number of countable convulsive seizure-free days in the T+M periods relative to baseline. To describe the difference between EPX100 vs placebo per 28-days in the reduction in episodes of status epilepticus 2, in the T+M periods relative to the baseline. To describe the incidence of rescue antiepileptic drug (AED) use between treatment arms, as measured by the number of days on rescue AEDs in the T+M periods relative to the baseline.;Primary end point(s): -To evaluate the efficacy of EPX-100 compared with placebo as adjunctive therapy in children and adult participantswith Dravet Syndrome, in terms of the mean percent change in countable convulsive seizure frequency (CCSF1) in the Titration and Maintenance (T+M) periods relative to baseline -The mean percent change between EPX-100 vs placebo in CCSF1 in the T+M periods relative to baseline.;Timepoint(s) of evaluation of this end point: 20 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The groups will be statistically compared on the following key secondary endpoints: • To describe the difference between EPX100 vs placebo in the number of countable convulsive seizure-free days in the T+M periods relative to baseline. • The number of countable convulsive seizure-free days in the T+M period relative to baseline. • To evaluate the difference between EPX100 vs placebo in proportion of participants with >50% reduction in the mean CCSF in the T+M periods relative to baseline. • The proportion of participants with >50% reduction in the mean CCSF in the T+M period relative to baseline. • To describe the total change in Seizure Severity using Hague Seizure Severity Scale (HASS). • The total HASS score in the T+M period relative to baseline. . To describe improvement in Clinical Global Impression (CGI). • The Clinical Global Impression (CGI) score, for each of the investigator and parent/caregiver assessments, in the T+M period. • To describe the total change in Quality of Life in Childhood Epilepsy short form (QOLCE-55). • The total score for the Quality of Life in Childhood Epilepsy (QOLCE-55) in the T+M period relative to baseline;Timepoint(s) of evaluation of this end point: 20 weeks | — |
Countries
Bulgaria, Canada, Georgia, Hungary, India, Poland, Romania, Spain, United Kingdom, United States
Contacts
Public ContactMary Shatzoff
Epygenix Therapeutics, Inc.
Outcome results
None listed