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ENTR-601-44

Phase 2

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

ENTR-601-44 development status

Highest phase Phase 2
Registered trials 2 across 1 sponsor since Jun 2025

ENTR-601-44 target and mechanism

Molecular targetDMD exon 44
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment104

FDA Designations

RARE_PEDIATRIC_DISEASE

ENTR-601-44 clinical trials

ENTR-601-44 · 2 trials · 1 indication

Phase 2 1Phase 1 1
NCT07682129Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)Duchenne Muscular Dystrophy (DMD)
NOT YET_RECRUITING80 Analytics
PHASE2NOT YET_RECRUITING
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
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 2 years).

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

Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and Open Label (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 concentration of study drug compounds and their final metabolite
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in 10-Meter Walk/Run (10MWR)
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF)
From baseline through End of Study (up to 2 years).
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ENTR-601-44EXPERIMENTALENTR-601-44 intravenous infusion every 6 weeks
ENTR-601-45EXPERIMENTALENTR-601-45 intravenous infusion every 6 weeks
PlaceboPLACEBO_COMPARATORintravenous infusion every 6 weeks

Interventions

NameTypeDescription
ENTR-601-44DRUGENTR-601-44 intravenous infusion
ENTR-601-45DRUGENTR-601-45 intravenous infusion
ENTR-601-44 - matching placeboDRUGintravenous infusion
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Eligibility Criteria

Age Range4 Years to 20 Years
SexMALE
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: * Willing and able to provide consent (if at age of majority) or assent (if a minor) * Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201 * Males who are sexually active with a female partner of childbearing potential must agree to use condoms during sexual ...

Countries:BelgiumItalyNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWAug 14, 2026NCT07037862lastUpdatePostDate: changed
LOWAug 14, 2026NCT07037862lastUpdatePostDate: changed
LOWAug 14, 2026NCT07037862lastUpdatePostDate: changed

Frequently asked questions about ENTR-601-44

What is ENTR-601-44?

ENTR-601-44 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 44 skipping. The drug is in Phase 2 clinical development and has received a Rare Pediatric Disease designation from the FDA.

What is ENTR-601-44 used for in Duchenne Muscular Dystrophy?

ENTR-601-44 is being developed to treat Duchenne Muscular Dystrophy in patients whose genetic mutation is amenable to exon 44 skipping. DMD is a neuromuscular condition, and this therapy targets the specific exon 44 mutation subset of the patient population.

What does ENTR-601-44 target?

ENTR-601-44 targets DMD exon 44. By directing the drug at exon 44, the therapy is intended for Duchenne Muscular Dystrophy patients with mutations amenable to exon 44 skipping. It is a small molecule developed using Entrada Therapeutics' Endosomal Escape Vehicle platform.

Who is developing ENTR-601-44?

ENTR-601-44 is being developed by Entrada Therapeutics, Inc., which trades on the Nasdaq under the ticker TRDA. The company is advancing the candidate for Duchenne Muscular Dystrophy patients amenable to exon 44 skipping.

What phase is ENTR-601-44 in?

ENTR-601-44 is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA. The program includes a Phase 2 long-term extension study and a Phase 1 study, both of which are active.

What clinical trials is ENTR-601-44 in?

ENTR-601-44 is being evaluated in two clinical trials. NCT07037862 is a Phase 1 study in participants with Duchenne Muscular Dystrophy amenable to exon 44 skipping, and it is currently recruiting. NCT07682129 is a Phase 2 long-term extension study, ELEVATE-LTE, that is not yet recruiting.