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ENTR-601-45

Phase 1

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

No designations recorded

Clinical trial landscape

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)
NCT0703882424
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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 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 used for?

ENTR-601-45 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD) in patients amenable to exon 45 skipping. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.

Who is developing ENTR-601-45?

ENTR-601-45 is being developed by Entrada Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol TRDA. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for Duchenne Muscular Dystrophy.

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 or any other regulatory agency. The ongoing Phase 1 trial is recruiting participants to evaluate its safety and efficacy.

What clinical trials is ENTR-601-45 in?

ENTR-601-45 is being studied in a Phase 1 clinical trial registered as NCT07038824. This randomized, double-blind, placebo-controlled study is recruiting male participants aged 4 years and older with Duchenne Muscular Dystrophy amenable to exon 45 skipping. The trial is being conducted in Belgium, Italy, Netherlands, Spain, and the United Kingdom.

Is ENTR-601-45 the same as exon 45 skipping therapy?

ENTR-601-45 is designed for patients with Duchenne Muscular Dystrophy who are amenable to exon 45 skipping. This means the therapy targets a specific genetic mutation that can be addressed by skipping exon 45 of the dystrophin gene. It is a distinct investigational drug being developed by Entrada Therapeutics.