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ENTR-601-45

Phase 2

Duchenne Muscular Dystrophy (DMD) | Small molecule | Neurology |Entrada Therapeutics, Inc.|Trials Updated: Aug 13, 2026

ENTR-601-45 development status

Highest phase Phase 2 (NCT07682129)
Phase scored for TRDAPhase 1
Registered trials 2 across 1 sponsor since Jun 2025

ENTR-601-45 target and mechanism

Molecular targetDMD exon 45
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24

FDA Designations

No designations recorded

ENTR-601-45 clinical trials

ENTR-601-45 · 1 trial · 1 indication

Phase 1 1
NCT07038824A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45Duchenne Muscular Dystrophy (DMD)
RECRUITING24 Analytics
PHASE1RECRUITING
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Duchenne Muscular Dystrophy (DMD)Unlock trial analytics

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 to 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)

LOWAug 14, 2026NCT07038824lastUpdatePostDate: changed
LOWAug 14, 2026NCT07038824lastUpdatePostDate: changed
LOWAug 14, 2026NCT07038824lastUpdatePostDate: changed

Frequently asked questions about ENTR-601-45

What is ENTR-601-45?

ENTR-601-45 is an investigational small molecule developed by Entrada Therapeutics for Duchenne Muscular Dystrophy (DMD). It is designed for patients whose disease is amenable to exon 45 skipping. It is currently in Phase 1 clinical development and has not been approved by the FDA.

What is ENTR-601-45 used for in Duchenne Muscular Dystrophy?

ENTR-601-45 is being developed for Duchenne Muscular Dystrophy (DMD) in patients whose mutations make them amenable to exon 45 skipping. It is an investigational therapy in Phase 1 testing and is not yet approved for any indication.

What does ENTR-601-45 target?

ENTR-601-45 targets DMD exon 45. By directing exon 45 skipping, the small molecule aims to address the underlying genetic cause of Duchenne Muscular Dystrophy in patients with mutations amenable to this approach. It is an investigational agent in Phase 1 development.

Who is developing ENTR-601-45?

ENTR-601-45 is being developed by Entrada Therapeutics, Inc., which trades on the Nasdaq under the ticker TRDA. The company is advancing the small molecule for Duchenne Muscular Dystrophy in patients amenable to exon 45 skipping.

What phase is ENTR-601-45 in?

ENTR-601-45 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 1 study is currently recruiting participants with Duchenne Muscular Dystrophy who are amenable to exon 45 skipping.

What clinical trials is ENTR-601-45 in?

ENTR-601-45 is being evaluated in one active clinical trial, NCT07038824, a Phase 1 study in participants with Duchenne Muscular Dystrophy amenable to exon 45 skipping. The trial is recruiting 24 male participants aged 4 years and older across Belgium, Italy, the Netherlands, Spain, and the United Kingdom.