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Looking for participantsPhase1/Phase2

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

Sponsor: Entrada Therapeutics, Inc.

NCT ID: NCT07038824

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
ENTR-601-45 (drug), ENTR-601-45 - matching placebo (drug)
How long the study runs
Study runs about 43 months (dates as stated)
About the drug or intervention
ENTR-601-45 — drug: intravenous infusion · ENTR-601-45 - matching placebo — drug: intravenous infusion
Patient visit burden
Not specified by the sponsor

In plain English

This study is looking at a potential new treatment called ENTR-601-45 for people with Duchenne muscular dystrophy (DMD) whose particular genetic change may respond to a method called 'exon 45 skipping'. The study will check whether the treatment is safe and whether it works. It is run by Entrada Therapeutics, Inc.

Who can take part

  • A genetic test confirming DMD with a change in the dystrophin gene that may respond to exon 45 skipping, checked by a genetic counsellor
  • Assigned male at birth, with signs of Duchenne muscular dystrophy
  • Part A: aged 4 to 20 years
  • Part A: able to walk, with arm function measured using a test called the Performance of the Upper Limb version 2.0 (PUL 2.0)
  • Enough healthy muscle for a small tissue sample (biopsy) to be taken
  • Other criteria in the study plan also apply

Who may not be able to

  • Another serious health problem that could make taking part difficult
  • An acute (sudden) illness in the 4 weeks before the first dose
  • Previous or current exon skipping treatment in the past 12 months
  • Previous or current gene therapy
  • Taking blood-thinning medicines (anticoagulants, anti-thrombotics or anti-platelet drugs) from 30 days before screening until the end of the study
  • Taking immunosuppressant medicines (except corticosteroid tablets or injections for DMD) from 30 days before screening until the end of the study
  • Taking a medicine called an HDAC inhibitor, such as givinostat, from 30 days before screening until the end of the study
  • Unusual blood test results
  • Needing a ventilator during the day, or breathing support through a hole in the neck (tracheostomy)
  • An abnormal heart tracing (ECG) that the doctor considers important, or a certain heart tracing measurement (QTcF) over 450 milliseconds
  • Taking any experimental or investigational drug in the past 3 months, or within 5 half-lives of that drug (whichever is longer)
  • Other criteria in the study plan also apply

What taking part involves

  • • Taking the study drug ENTR-601-45
  • • Having a small muscle tissue sample (biopsy) taken
  • • Heart tracings (ECG) and blood tests as part of the checks

Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
4 Years to 20 Years
Who
Male
Number of participants
24
Started
2025-08-30
Last checked
2026-05

Plain English Summary

What is this study?

  • • Testing a new treatment for duchenne muscular dystrophy (dmd)
  • • Phase1/Phase2 - 24 participants
  • • This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition

Who can take part?

  • • Ages 4 Years to 20 Years
  • • Diagnosed with duchenne muscular dystrophy (dmd)
  • • Male only

Where?

  • • Leeds - Leeds General Infirmary
  • • Liverpool - Alder Hey Children's NHS Foundation Trust
  • • London - Great Ormond Street Hospital for Children
  • • Manchester - Royal Manchester Children's Hospital
  • • +1 more UK sites

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-45 and placebo are both called study treatments. The study has 2 parts: Part A: to evaluate if ENTR-601-45 is safe and to determine the best dose of ENTR-601-45 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-45 at the dose determined in Part A. Participants will be able to roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated. Participants will: * Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B * Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, muscle biopsies and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug. Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.

Duchenne Muscular Dystrophy (DMD)

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 4 Years - 20 Years
  • Who can join: Male only

What the study is looking for

  • ✓Genetic diagnosis of DMD and confirmed disease variant in the dystrophin gene amenable to exon 45 skipping as...
  • ✓Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the...
  • ✓Part A: 4-20 years of age, inclusive.
  • ✓Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at...
  • ✓Adequate muscle for obtaining tissue biopsy as assessed by the investigator.

Who cannot take part

  • ✗Any significant concomitant medical condition that might interfere with the ability to comply with protocol...
  • ✗Has an acute illness within 4 weeks prior to the first dose of the study treatment which may interfere with study...
  • ✗Use of the following medications :
  • ✗Prior or current treatment with any exon skipping therapy within the previous 12 months
  • ✗Prior or current treatment with any gene therapy
See the full criteria
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 investigator. 3. Part A: 4-20 years of age, inclusive. 4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening. 5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator. 6. Other protocol-defined criteria apply. Exclusion Criteria: 1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements. 2. Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety. 3. Use of the following medications : 1. Prior or current treatment with any exon skipping therapy within the previous 12 months 2. Prior or current treatment with any gene therapy 3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from 30 days prior to screening and until the end of the study 4. Use of an immunosuppressant (other than systemic or oral corticosteroid for DMD condition) from 30 days prior to screening until the end of the study. 5. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from 30 days prior to screening until the end of the study 4. Laboratory abnormalities. 5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy. 6. Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) \>450 msec at Screening or prior to the first dose of study drug on Day 1. 7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer). 8. Other protocol-defined criteria apply.

Where Is This Study? (5 UK sites)

Leeds General Infirmary

Leeds LS1 3EX, United Kingdom

Recruiting
Site contact (verified)
Cristina Martos LozanoPrincipal Investigator

Alder Hey Children's NHS Foundation Trust

Liverpool L122AP, United Kingdom

Recruiting
Site contact (verified)
Rajesh MadhuPrincipal Investigator

Great Ormond Street Hospital for Children

London WC1N 3JH, United Kingdom

Recruiting
Site contact (verified)
Giovanni BaranelloPrincipal Investigator

Royal Manchester Children's Hospital

Manchester M13 9WL, United Kingdom

NOT_YET_RECRUITING
Site contact (verified)
Gary McCullaghPrincipal Investigator

Oxford University Hospitals NHS Foundation Trust

Oxford OX3 9DU, United Kingdom

Recruiting
Site contact (verified)
Laurent ServaisPrincipal Investigator
Data sourced from ClinicalTrials.gov · Last verified: 2026-05