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ENTR-601-44

Phase 2

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

Target and mechanism

Molecular targetDMD exon 44
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials2
Total Enrollment104

FDA Designations

No designations recorded

Clinical trial landscape

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
LOWJul 2, 2026NCT07682129NEW_TRIAL: changed
LOWJul 2, 2026NCT07682129NEW_TRIAL: changed
LOWJul 2, 2026NCT07682129NEW_TRIAL: changed

Frequently asked questions about ENTR-601-44

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

ENTR-601-44 is an investigational small molecule being developed for Duchenne Muscular Dystrophy (DMD) in patients amenable to exon 44 skipping. It is designed to target the DMD exon 44 region. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does ENTR-601-44 target?

ENTR-601-44 targets the DMD exon 44 region of the dystrophin gene. This target is relevant for patients with Duchenne Muscular Dystrophy who have mutations amenable to exon 44 skipping, a strategy intended to restore the reading frame of the dystrophin protein.

Who is developing ENTR-601-44?

ENTR-601-44 is being developed by Entrada Therapeutics, Inc., a biopharmaceutical company publicly traded under the ticker symbol TRDA. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for Duchenne Muscular Dystrophy.

What phase is ENTR-601-44 in?

ENTR-601-44 is in Phase 2 clinical development for Duchenne Muscular Dystrophy. It is an investigational drug, meaning it has not received FDA approval and is still undergoing clinical trials to assess its safety and effectiveness in patients.

What clinical trials is ENTR-601-44 in?

ENTR-601-44 is being studied in two clinical trials. NCT07037862 is a Phase 1 study recruiting 24 male participants with DMD amenable to exon 44 skipping. NCT07682129 is a Phase 2 long-term extension study with 80 participants, not yet recruiting. Both are controlled, randomized, and double-blind.

Is ENTR-601-44 the same as other exon-skipping drugs?

ENTR-601-44 is a distinct investigational drug developed by Entrada Therapeutics. It is part of a class of therapies targeting exon 44 skipping in Duchenne Muscular Dystrophy, but it is not the same as other approved or investigational exon-skipping agents. No alternative names for ENTR-601-44 have been disclosed.