Lennox Gastaut SyndromePhase 3NCT05066217
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
A Phase 3 study testing Clemizole HCl in people with Lennox Gastaut Syndrome, run by Epygenix at 71 sites in 10 countries.
Trial summary
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCL (EPX-100) as adjunctive therapy in children and adult participants with Lennox-Gastaut syndrome (LGS). Read more about this study
- Who is it for?
- Ages 2 to 55 years
- Conditions the study is for
- Lennox Gastaut Syndrome
- What is being tested?
- Clemizole HCl
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 260 participants
- Where
- 71 sitesin 10 countries
- Study dates
- 9 Apr 2025to 1 Nov 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males or females, ages ≥2 to ≤55 years, at the time of Screening.
- Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent.
- Diagnosis of LGS, including:
- Evidence of at least one type of countable major motor seizure.
- History of electroencephalogram (EEG) consistent with LGS (abnormal background activity, and one of the following: 1) slow spike-wave discharges [<2.5 Hz], or 2) paroxysmal fast activity during sleep).
- Abnormal cognitive development.
- Onset of seizures at 11 years of age or younger.
Who cannot join
- Known sensitivity, allergy, or previous exposure to clemizole HCl.
- Known history of long QT syndrome or any significant history of a serious abnormality of the electrocardiogram (ECG) (e.g., recent myocardial infarction, clinically significant arrhythmia).
- Family history of sudden cardiac death, unexplained death, or death from a primary dysrhythmia potentially associated with QT prolongation in any family member.
- Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or progressive central nervous system disease, metabolic illness, recent anoxic episode within the last 6 months requiring resuscitation, or progressive degenerative disease or any other condition, which in the opinion of the investigator, could affect seizure control.
- Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
- Concomitant use of fenfluramine.
- Prior or concomitant use of lorcaserin.
Trial locations and contacts
71 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
71 sites in 10 countries
United States22 sites, 17 recruiting
- Recruiting
Atrium Health STRIVE Research
Charlotte, North Carolina, 28207
Jessica Lindley, Jessica.Lindley@atriumhealth.org
Ravi Singh, MD
- Recruiting
On-Site Clinical Solution
Charlotte, North Carolina, 28211
Ludny Saintil, 800-785-3150 ext. 198, ludny.santil@thermofischer.com
Robert Nahouraii
- Recruiting
Re:Cognition Health
Chicago, Illinois, 60611
Antonio Iglesias, MD, 312-971-3318, aiglesias@re-cognitionhealth.com
Antonio Iglesias, MD
- Recruiting
Children's Health Specialty Center
Dallas, Texas, 75235
Charuta Joshi
- Active, not recruiting
Henry Ford Hospital
Detroit, Michigan, 48202
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27705
Muhammad Zafar, MD, muhammad.zafar@duke.edu
Muhammad Zafar, MD
- Recruiting
UTHealth Houston
Houston, Texas, 77030
Antoinette Bernal-Tent, 713-500-7105, Antoinette.BernalTent@uth.tmc.edu
Mary Koenig, MD
- Active, not recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
- Recruiting
Norton Children's Medical Center
Louisville, Kentucky, 40202
Madeline McGinty-Holmes, 502-629-5568, madeline.mcginty-holmes@louisville.edu
Jaime Shoup, MD
- Recruiting
Tekton Research
Marlboro, New Jersey, 07746
Aimee Kinlan, 626-218-8294, aimee.kinlan@tektonresearch.com
Amor Mehta, MD
- Recruiting
University of Miami Miller School of Medicine
Miami, Florida, 33136
Brett Alonso Cardenas, 305-243-3100, bac301@med.miami.edu
Kamil Detyniecki, MD
- Recruiting
PPD Virtual-Science 37, Inc.
Morrisville, North Carolina, 27560
Victoria Taylor, 615-707-9148, victoria.taylor@science37.com
Antonio Iglesias, MD
- Recruiting
NYU Comprehensive Epilepsy Center - BRANY - PPDS
New York, New York, 10016-4852
Dana Price
- Recruiting
Weill Cornell Medicine/New York Presbyterian Hospital
New York, New York, 10021
Lundy Santil, 800-785-3150 ext. Ext. 198, ludny.saintil@thermofisher.com
Asim Shahid, los9075@med.cornell.edu
- Recruiting
Children's Nebraska
Omaha, Nebraska, 68114
Sookyong Koh, MD, PhD, 773-929-4445, skoh@childrensnebraska.org
Sookyong Koh
- Active, not recruiting
UC Irvine Medical Center
Orange, California, 92868
- Recruiting
AdventHealth Innovation Tower
Orlando, Florida, 32804
Holly Skinner, DO
- Suspended
Rare Disease Research
Orlando, Florida, 34746
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Eric Marsh, MD
- Recruiting
Minnesota Epilepsy Group, P.A.
Roseville, Minnesota, 55102
Sarah Ellis, MD, 651-377-8319, sellis@mnepilepsy.net
Stephanie Ahrens, MD
- Recruiting
Clinical Neurosciences Center
Salt Lake City, Utah, 84132
Angela Peters, MD
- Active, not recruiting
Pediatric Neurology and Epilepsy Specialists
Winter Park, Florida, 32789
China18 sites, 18 recruiting
- Recruiting
Peking University First Hospital - Changqiao Campus
Beijing, 100034
Yuwu Jiang
- Recruiting
Peking University First Hospital - Daxing Campus
Beijing, 100034
Yuwu Jiang
- Recruiting
Beijing Children's Hospital, Capital Medical University - PIN
Beijing, 100045
Hui Xiong
- Recruiting
The First Hospital of Jilin University
Changchun, 130021
Jianmin Liang
- Recruiting
Xiangya Hospital Central South University
Changsha, Hunan, 410008
Jing Peng
- Recruiting
Hunan Children's Hospital
Changsha, 410007
Sai Yang
- Recruiting
West China Second University Hospital, Sichuan University - Huaxi Campus
Chengdu, 610041
Rong Luo
- Recruiting
Chengdu Women's and Children's Central Hospital
Chengdu, 610073
Li Yan
- Recruiting
Children's Hospital of Chongqing Medical University
Chongqing, 400014
Li Jiang
- Recruiting
The Second Affiliated Hospital of Guangzhou Medical University
Guangzhou, 510260
Xiaorong Liu
- Recruiting
The Affiliated Hospital of Guizhou Medical University
Guiyang, 550004
Rong Ai
- Recruiting
Qilu Hospital of Shandong University
Jinan, 250012
Baomin Li
- Recruiting
Jiangxi Provincial Children's Hospital
Nanchang, 330006
Yong Chen
- Recruiting
Children's Hospital of Shanghai
Shanghai, Shanghai Municipality, 200062
Simei Wang
- Recruiting
Shenzhen Children's Hospital
Shenzhen, 518000
Dezhi Cao
- Recruiting
Wuhan Children's Hospital
Wuhan, 430015
Dan Sun
- Recruiting
The Second Affiliated Hospital of Xi'an Jiaotong University
Xi'an, 710004
Lin Yang
- Recruiting
Xuzhou Children's Hospital
Xuzhou, 221006
Xiaoming Liu
Italy7 sites, 7 recruiting
- Recruiting
Azienda Ospedaliera Universitaria Renato Dulbecco - AO Mater Domini
Catanzaro, 88100
Antonio Gambardella
- Recruiting
Azienda Ospedaliero Universitaria A Meyer
Florence, 50139
Renzo Guerrini
- Recruiting
IRCCS Azienda Ospedaliera Metropolitan
Genoa, Liguria, 16132
Flavio Villani
- Recruiting
IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147
Maria Margherita Mancardi
- Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Giuseppe Didato
- Recruiting
Fondazione Istituto Neurologico Nazionale Casimiro Mondino IRCCS
Pavia, 27100
Elena Tartara
- Recruiting
IRCCS Istituto Neurologico Mediterraneo Neuromed
Pozzilli, Molise, 86077
Alfredo D'Aniello
Spain6 sites, 6 recruiting
- Recruiting
Hospital de La Santa Creu i Sant Pau
Barcelona, 08025
Alba Sierra-Marcos
- Recruiting
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, 08035
Manuel Toledo Argany
- Recruiting
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, 28009
Victor Soto Insuga
- Recruiting
Hospital Regional Universitario de Malaga - Hospital General
Málaga, 29010
Pedro Serrano
- Recruiting
Hospital Universitario de Navarra
Pamplona, 31008
Sergio Aguilera Albesa
- Recruiting
Centro de Neurologia Avanzada
Seville, 41013
Juan Jesús Rodríguez Uranga, MD
India5 sites, 5 recruiting
- Recruiting
Royal Institute of Child Neurosciences
Ahmedabad, Gujarat, 380054
Nitish Vora
- Recruiting
Aster Malabar Institute of Medical Sciences Ltd
Calicut, Kerala, 673016
Smilu Mohanlal
- Recruiting
Yashoda Hospitals Hitec City
Hyderabad, Andhra Pradesh, 500081
Lokesh Lingappa
- Recruiting
Nizams Institute of Medical Sciences
Hyderabad, Andhra Pradesh, 500082
Surya Prabha Turaga
- Recruiting
Saachi Children Hospital
Surat, Gujarat, 395002
Ritesh Shah
Serbia4 sites, 4 recruiting
- Recruiting
Childrens University Hospital
Belgrade, 11000
Dimitrije Nikolic
- Recruiting
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
Belgrade, 11000
Biljana Vucetic Tadic
- Recruiting
University Clinical Center Nis
Niš, 18 000
Tatjana Tosic
- Recruiting
Children and Youth Health Care Institute of Vojvodina
Novi Sad, 21000
Tatjana Redzek Mudrinic
Poland3 sites, 3 recruiting
- Recruiting
Centrum Medyczne Plejady Magdalena Celinska
Krakow, Lesser Poland Voivodeship, 30-363
Marta Zolnowska, MD
- Recruiting
Neurosphera
Warsaw, Masovian Voivodeship, 02-829
Beata Zwolinska
- Recruiting
Premium Clinic Wrocław Centrum Medyczne
Wroclaw, Lower Silesian Voivodeship, 50-414
Monika Sluzewska-Niedzwiedz
Romania3 sites, 3 recruiting
- Recruiting
Centrul National Clinic De Recuperare Neuropsihomotorie Copii Robanescu-Padure
Bucharest, 041408
Madalina Leanca
- Recruiting
Emergency University Hospital
Bucharest, 050098
Ioana Raluca Mindruta
- Recruiting
Sf. Maria Children Emergency Clinical Hospital Iasi
Iași, 700309
Ioana Grigore
Turkey (Türkiye)2 sites, 2 recruiting
- Recruiting
Eskisehir Osmangazi Universitesi Saglik Uygulama ve Arastirma Hastanesi
Odunpazari, Eskişehir, 26040
Kursat Carman
- Recruiting
Karadeniz Teknik Universitesi Farabi Hastanesi
Ortahisar, 61080
Ali Cansu
Hungary1 site, 1 recruiting
- Recruiting
Debreceni Egyetem Klinikai Kozpont Nagyerdei Campus
Debrecen, Hajdú-Bihar, 4032
Monika Bessenyei
About this study
Official title: Multicenter, Randomized, Double-blind, Placebo-controlled Trial of Clemizole HCl as Adjunctive Therapy in Patients With Lennox-Gastaut Syndrome
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCL (EPX-100) as adjunctive therapy in children and adult participants with Lennox-Gastaut syndrome (LGS).
Read the detailed description
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole HCl as adjunctive therapy in children and adult participants with LGS.
The study will consist of an Observational Period, a Double-Blind (DB) Period, and an optional Open-Label Extension (OLE) Period.
- Conditions
- Lennox Gastaut Syndrome
- Being tested
- Clemizole HCl, Placebo
- Keywords
- Lennox Gastaut Syndrome, Lennox-Gastaut Syndrome, LGS, Clemizole HCl, Seizure
- Medical subject headings
- Lennox Gastaut Syndrome, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clemizole HClDrug
Also called EPX-100
Clemizole HCl will be administered as an oral solution.
PlaceboDrug
Placebo will be administered as an oral solution.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Participants will receive their first dose of study drug following randomization. Receives: Drug: Placebo |
| Double-blind clemizole HCl | Experimental | Participants will receive their first dose of study drug following randomization. Receives: Drug: Clemizole HCl |
| Open-label clemizole HCl | Experimental | Eligible participants who complete the DB Period will have the option to continue in the OLE Period, during which they will receive clemizole HCl for up to 3 years. Receives: Drug: Clemizole HCl |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in CMMS-28
Percent change in CMMS-28 from the Baseline Period through the end of the DB Period
Measured: From Baseline Period up to 16 weeks
Secondary outcomes (10)
Proportion of Participants with ≥50% Reduction in CMMS-28
Proportion of participants with ≥50% reduction in CMMS-28 from the Baseline Period through the end of the DB Period
Measured: From Baseline Period up to 16 weeks
Percent Change in CMMS-28 Seizure-free Days
Percent change in CMMS-28 seizure-free days from the Baseline Period through the end of the DB Period
Measured: From Baseline Period up to 16 weeks
Clinical Global Impression of Change (CGI-C) Score
CGI-C score at the end of the DB Period
Measured: Week 16
Caregiver Global Impression of Change (CaGI-C) Score
CaGI-C score at the end of the DB Period
Measured: Week 16
Caregiver Global Impression of Change in Seizure Intensity/Duration (CaGI-CSID) Score
CaGI-CSID score at the end of the DB Period
Measured: Week 16
Change in Quality of Life Inventory (QI)-Disability Score
Change in QI-disability score from Baseline to the end of the DB Period
Measured: From Baseline Period up to 16 weeks
Percent Change per 28 Days in the Number of Seizure Free Days
Percent change per 28 days in the number of seizure-free days (based on all seizure types) from the Baseline Period through the end of the DB Period
Measured: From Baseline Period up to 16 weeks
Percent Change in CMMS-28
Percent change in CMMS-28 from the Baseline Period through the end of the DB Maintenance Phase only
Measured: From Baseline Period up to 12 weeks
Proportion of Participants with ≥50% Reduction in CMMS-28
Proportion of participants with ≥50% reduction in CMMS-28 from the Baseline Period through the end of the DB Maintenance Phase only
Measured: From Baseline Period up to 12 weeks
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Incidence of TEAEs will be compared among the treatment groups
Measured: From the first dose administration of study drug up to end of the study, approximately up to 172 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2021
- Study start9 Apr 2025
- Primary completion1 Nov 2026 (estimated)
- Study completion1 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lennox Gastaut SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients are randomized 1:1 to clemizole HCl (EPX-100) or placebo.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 260 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Epygenix
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Harmony Biosciences Management, Inc.
- Responsible party
- Sponsor
- Investigators
- Amit Ray, MD, Study director, Harmony Biosciences Management, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05066217
- Sponsor's protocol ID
- EPX-100-003
- Status last verified
- Oct 2026
- First submitted
- 23 Sep 2021
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No