Tests ENTR-601-45 in people with Duchenne muscular dystrophy
Tests ENTR-601-45 for safety and effects in young people with Duchenne.
Plain-language title generated from the registry record
Registry title: A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Key facts
- Where
- Leiden and Nijmegen, NetherlandsSee the 2 sites
- Phase
- Phase 1/2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys and young men with Duchenne muscular dystrophy caused by a gene change that can be treated by skipping exon 45. It tests the safety and effect of ENTR-601-45 against placebo.
- Age
- 4 to 20 years (Part A).
- Sex
- Males only (assigned male at birth).
- Disease stage
- Duchenne muscular dystrophy confirmed by a genetic test, with a dystrophin gene change amenable to exon 45 skipping. In Part A, people must be able to walk (ambulatory). They must have enough muscle for a biopsy.
- Earlier treatment
- Cannot have had any exon skipping therapy in the previous 12 months, or any gene therapy. Cannot have taken any experimental drug within 3 months before the first dose or within 5 half-lives (whichever is longer). Standard DMD care, such as corticosteroids, may continue.
- Biomarker or test result
- A confirmed variant in the dystrophin gene that is amenable to exon 45 skipping, reviewed by a central genetic counselor.
Main reasons for not taking part
- Exon skipping therapy within the previous 12 months, or any gene therapy
- Daytime ventilator dependence or invasive ventilation through a tracheostomy
- Acute illness within 4 weeks before the first dose
- Blood thinners, immunosuppressants (except corticosteroids for DMD) or HDAC inhibitors such as givinostat
- Clinically significant abnormal heart tracing (ECG) or QTcF above 450 msec
Read by machine on 9 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 4 and 20 years old?
Source: NCT07038824, who can take part
Where in Netherlands
- Leids Universitair Medisch Centrum (LUMC)Leiden, NetherlandsTaking people
- Radboud universitair medisch centrum StichtingNijmegen, NetherlandsTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Entrada Therapeutics, Inc.
- Trial code
- NCT07038824EU 2024-517499-39-00
- Record updated
- 13 August 2026
- Original description
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.Read full original descriptionShow less
A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping With an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)
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.
Inclusion: Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor; Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator; 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 Screening; Adequate muscle for obtaining tissue biopsy as assessed by the investigator; Other protocol-defined criteria apply.
Exclusion: Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements; 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; Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer); 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; Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from 30 days prior to screening and until the end of the study; 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; 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; Laboratory abnormalities; 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; Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy; Other protocol-defined criteria apply.