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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
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
Where Is This Study? (5 UK sites)
Leeds General Infirmary
Leeds LS1 3EX, United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool L122AP, United Kingdom
Great Ormond Street Hospital for Children
London WC1N 3JH, United Kingdom
Royal Manchester Children's Hospital
Manchester M13 9WL, United Kingdom
Oxford University Hospitals NHS Foundation Trust
Oxford OX3 9DU, United Kingdom
