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ENTR-601-45

Phase 1

Duchenne Muscular Dystrophy (DMD) | Small molecule | Neurology |Entrada Therapeutics, Inc.|Last Updated: May 18, 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_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07038824A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45PHASE1 RECRUITING 24 — — Aug 30, 2025Mar 1, 2029May 18, 202615 Belgium, Italy +3
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Study Endpoints
Primary Endpoints
Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period).
From baseline through End of Study (up to 62 weeks).

Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.

Secondary Endpoints
Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)
From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A).
Baseline, End of Study (Up to 25 weeks)
Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A).
Baseline, End of Study (Up to 25 weeks)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ENTR-601-45EXPERIMENTALintravenous infusion every 6 weeks
PlaceboPLACEBO_COMPARATORintravenous infusion every 6 weeks
Interventions
NameTypeDescription
ENTR-601-45DRUGintravenous infusion
ENTR-601-45 - matching placeboDRUGintravenous infusion
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Eligibility Criteria
Age Range4 Years — 20 Years
SexMALE
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: 1. Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor. 2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigato...

Countries:BelgiumItalyNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07038824primaryCompletionDate: changed
LOWMay 24, 2026NCT07038824studyFirstPostDate: changed