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Clemizole

Phase 3

Lennox Gastaut Syndrome | Small molecule | Other |Harmony Biosciences Holdings, Inc.|Last Updated: Apr 15, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment260
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05066217An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut SyndromePHASE3 RECRUITING 260Apr 9, 2025Nov 1, 2029Apr 15, 202620 United States
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Study Endpoints
Primary Endpoints
Percent Change in CMMS-28
From Baseline Period up to 16 weeks

Percent change in CMMS-28 from the Baseline Period through the end of the DB Period

Secondary Endpoints
Proportion of Participants with ≥50% Reduction in CMMS-28
From Baseline Period up to 16 weeks
Percent Change in CMMS-28 Seizure-free Days
From Baseline Period up to 16 weeks
Clinical Global Impression of Change (CGI-C) Score
Week 16
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORParticipants will receive their first dose of study drug following randomization.
Double-blind clemizole HClEXPERIMENTALParticipants will receive their first dose of study drug following randomization.
Open-label clemizole HClEXPERIMENTALEligible 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.
Interventions
NameTypeDescription
Clemizole HClDRUGClemizole HCl will be administered as an oral solution.
PlaceboDRUGPlacebo will be administered as an oral solution.
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Eligibility Criteria
Age Range2 Years — 55 Years
SexALL
Healthy VolunteersNo
Study Sites20

Key Inclusion Criteria: 1. Males or females, ages ≥2 to ≤55 years, at the time of Screening. 2. Participant/parent/legal authorized representative (LAR) willing and able to give written informed consent/assent. 3. Diagnosis of LGS, including: * Evidence of at least one type of countable major m...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT05066217studyFirstPostDate: changed