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EDK060, dose A

Phase 1

Charcot-Marie-Tooth Disease, Type 1A | Small molecule | Other |Novartis AG|Last Updated: Apr 21, 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 Enrollment28
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07140614A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.PHASE1 RECRUITING 28 — — Sep 30, 2025Feb 26, 2028Apr 21, 20263 Canada
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Study Endpoints
Primary Endpoints
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
From baseline up to day 140

Incidence of AEs and SAEs by treatment group, including changes in vital signs, laboratory measures, neurological examinations and electrophysiology measures qualifying and reported as AEs.

Secondary Endpoints
EDK060 Plasma PK parameters - Cmax
From baseline up to day 140
EDK060 Plasma PK parameters - Tmax
From baseline up to day 140
EDK060 Plasma PK parameters - AUClast
From baseline up to day 140
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EDK060EXPERIMENTALDoses A; B; C; D
PlaceboPLACEBO_COMPARATORMatching placebo
Interventions
NameTypeDescription
EDK060, dose ADRUGEDK060, dose A, Single dose, IV infusion
EDK060, dose BDRUGEDK060, dose B, Single dose, IV infusion
EDK060, dose CDRUGEDK060, dose C, Single dose, IV infusion
EDK060, dose DDRUGEDK060, dose D, Single dose, IV infusion
PlaceboOTHERPlacebo
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Provide written informed consent before any assessment is performed. * Be male or female and 18 to 60 (inclusive) years of age at the time of screening. * Participant must have a clinical diagnosis of Charcot-Marie-Tooth Disease Type 1A (CMT1A) including verified documentation...

Countries:Canada
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07140614primaryCompletionDate: changed
LOWMay 24, 2026NCT07140614studyFirstPostDate: changed