| CTRI Number |
CTRI/2026/01/100155 [Registered on: 01/01/2026] Trial Registered Prospectively |
| Last Modified On: |
09/09/2026 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Parallel Group, Placebo Controlled Trial |
|
Public Title of Study
|
A study to compare efficacy of EPX-100 in the treatment of Dravet syndrome |
|
Scientific Title of Study
|
A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants with Dravet Syndrome (ARGUS Trial) |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| 2024-518628-57-00 |
EudraCT |
| EPX-100-001 Amendment version 10.0 dated 23 May 2025 |
Protocol Number |
| NCT04462770 |
ClinicalTrials.gov |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Umakanta Sahoo |
| Designation |
Managing Director |
| Affiliation |
GCT Pharma Research (India) Pvt Ltd |
| Address |
7th Floor, Wing-B, Vatika Business Centre, Hiranandani Gardens, Powai,
Mumbai
MAHARASHTRA
400076
India |
| Phone |
912242369729 |
| Fax |
|
| Email |
U.Sahoo@gctrials.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Umakanta Sahoo |
| Designation |
Managing Director |
| Affiliation |
GCT Pharma Research (India) Pvt Ltd |
| Address |
7th Floor, Wing-B, Vatika Business Centre, Hiranandani Gardens, Powai,
Mumbai
MAHARASHTRA
400076
India |
| Phone |
912242369729 |
| Fax |
|
| Email |
U.Sahoo@gctrials.com |
|
Details of Contact Person
Public Query
|
| Name |
Umakanta Sahoo |
| Designation |
Managing Director |
| Affiliation |
GCT Pharma Research (India) Pvt Ltd |
| Address |
7th Floor, Wing-B, Vatika Business Centre, Hiranandani Gardens, Powai,
Mumbai
MAHARASHTRA
400076
India |
| Phone |
912242369729 |
| Fax |
|
| Email |
U.Sahoo@gctrials.com |
|
|
Source of Monetary or Material Support
|
| Epygenix Therapeutics, Inc
630 W. Germantown Pike, Suite 215 Plymouth Meeting, PA 19462 USA |
|
|
Primary Sponsor
|
| Name |
Epygenix Therapeutics, Inc |
| Address |
630 W. Germantown Pike, Suite 215 Plymouth Meeting, PA 19462 USA |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
| Name |
Address |
| Ms GCT Pharma Research India Pvt Ltd |
7th Floor, Wing-B, Vatika Business Centre, Hiranandani Gardens, Powai, Mumbai , Maharashtra (India) - 400076 |
|
|
Countries of Recruitment
|
Canada
China
Georgia
Hungary
India
Poland
Spain
United Kingdom
United States of America |
|
Sites of Study
|
| No of Sites = 8 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Sheffali Gulati |
All India Institute of Medical Sciences |
Room No. 805, Department of neurology, 8th Floor, A-Wing, Mother & Child Block, AIIMS, Ansari Nagar, New Delhi, Delhi-110029
New Delhi
DELHI |
98103 86847
sheffaligulati@gmail.com |
| Dr Vinayan K P |
Amrita Institute of Medical Sciences and Research |
Department of neurology,AIMS Ponekkara P.O,Kochi-682041
Ernakulam
KERALA |
9447800303
vinayankp@aims.amrita.edu |
| Dr Amarjeet Wagh |
Central India Cardiology Hospital & Research Institute |
Department of neurology, Pioneer Co-Op
Housing Society, Plot No.1, Gawande
Lay Out, Nagpur-440015, Maharashtra, India.
Mumbai
MAHARASHTRA |
77768 71744
amarjeet.wagh2001@gmail.com |
| Dr Maya Thomas |
Christian Medical College, Vellore |
Department of neurology, West Block 910, 1st Floor, Room No. 14, Paediatric Neurology Unit, Department of Neurological Sciences, Ida Scudder Road, Vellore-632004, Tamil Nadu
Vellore
TAMIL NADU |
99444 21996
maya@cmcvellore.ac.in |
| Dr Anaita Hegde |
Jaslok Hospital & Research Centre |
9th Floor, Department of neurology, Pediatric Research Room,
15, Dr. G. Deshmukh Marg, Mumbai,400026
Mumbai
MAHARASHTRA |
9820186155
anaitahegde@gmail.com |
| Dr Ajay Asranna |
National Institute of Mental Health and Neurosciences (NIMHANS) |
Department of neurology, Hosur Main Road, Lakkasandra, Ayappa Garden, Adugodi, Bengaluru, 560029, Karnataka
Bangalore
KARNATAKA |
89436 12626
ajayasranna@gmail.com |
| Dr Ritesh Shah |
Saachi Children Hospital |
Department of neurology, Prajapati Brahma Marg, Near Akashganga
Apartment, Sanskar Nagar, Majura Gate, Surat, Gujarat-395002.
Surat
GUJARAT |
99131 87560
Drriteshcshah@yahoo.co.in |
| Dr Pradnya Gadgil |
SRCC Children’s Hospital Managed by Narayana Health |
Department of neurology, 1A Haji Ali Park,
Keshavrao Khadye Marg, Mahalaxmi,
Mumbai-400034
Mumbai
MAHARASHTRA |
9820507577
pradnya_g@yahoo.com |
|
Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 9 |
| Name of Committee |
Approval Status |
| Ethics Committee Jaslok Hospital Research Centre |
Approved |
| Ethics Committee Jaslok Hospital Research Centre |
Approved |
| Ethics Committee Unique HospitalMultispeciality & Research Institute |
Approved |
| Institutional Ethics Committee of KLE Academy of Higher Education and Research |
Approved |
| Institutional Ethics Committee Rughwani Child Care Centre and Hospita |
Approved |
| Institutional Ethics Committee, AIIMS |
Approved |
| Institutional Ethics Committee, Amrita Institute of Medical Sciences and Research Centre |
Approved |
| Institutional Review Board, Christian Medical College |
Approved |
| NIMHANS Institute Ethics Committee Basic & Neuro Science Devision |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: G408||Other epilepsy and recurrent seizures, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
EPX-100 (Clemizole Hydrochloride) |
EPX-100 will be administered as an oral solution (5 mg/mL). |
| Comparator Agent |
Placebo |
Flavor and color-matching placebo will be administered as an oral solution |
|
|
Inclusion Criteria
|
| Age From |
2.00 Year(s) |
| Age To |
60.00 Year(s) |
| Gender |
Both |
| Details |
1.Male and female participants 2 years and older at time of consent.
2.Participant or parent or 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 DS. Participants must have seizures which are not completely controlled by antiepileptic drugs (AEDs) with the following criteria
Onset of seizures prior to 18 months of age,
Normal development at onset
History of at least one type of countable motor seizure
4.Brain magnetic resonance imaging (MRI) without cortical malformation (not including mild atrophy associated with the natural progression of DS),
Genetic mutation of the SCN1A gene must be documented.
|
|
| ExclusionCriteria |
| Details |
1.Known sensitivity, allergy, or previous exposure to clemizole HCl.
2.Exposure to any investigational drug or device less than 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 (this includes concomitant use of tetrahydrocannabinol and nonprescription cannabidiol preparations) or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease.
4.Concurrent use of lorcaserin. Note Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
5.Concurrent use of fenfluramine.
6.Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
|
|
|
Method of Generating Random Sequence
|
Other |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Participant and Investigator Blinded |
|
Primary Outcome
|
| Outcome |
TimePoints |
| The percent change in CMS-28 in the Titration+ Maintenance (T+M) Periods relative to baseline |
Percent change in countable motor seizure per 28 days in any of the Periods, Titration 4 weeks and Maintenance 12 weeks. |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| NIL |
NIL |
|
|
Target Sample Size
|
Total Sample Size="150"
Sample Size from India="45"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 3 |
|
Date of First Enrollment (India)
|
16/01/2026 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
07/10/2020 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="9"
Months="0"
Days="24" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Open to Recruitment |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
This is a multicenter, Phase 3, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of clemizole hydrochloride (EPX-100) as adjunctive therapy in children and adult participants with Dravet syndrome (DS). |