RecruitingPhase 1Phase 2NCT07038824

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

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)


Sponsor

Entrada Therapeutics, Inc.

Enrollment

24 participants

Start Date

Aug 30, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Sex: MALEMin Age: 4 YearsMax Age: 20 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called ENTR-601-45 and a drug called ENTR-601-45 - matching placebo for people with duchenne muscular dystrophy (dmd). The study is currently recruiting participants at 15 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGENTR-601-45

intravenous infusion

DRUGENTR-601-45 - matching placebo

intravenous infusion


Locations(15)

University Hospital Gent

Ghent, Belgium

UZ Leuven

Leuven, Belgium

Centre Hospitalier Régional de la Citadelle

Liège, Belgium

IRCCS Ospedale San Raffaele

Milan, Italy

Ospedale Pediatrico Bambino Gesu

Rome, Italy

Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Rome, Italy

Leids Universitair Medisch Centrum

Leiden, Netherlands

Stichting Radboud Universitair Medisch Centrum

Nijmegen, Netherlands

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Hospital Sant Joan de Deu

Barcelona, Spain

Leeds General Infirmary

Leeds, United Kingdom

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom

Great Ormond Street Hospital for Children

London, United Kingdom

Royal Manchester Children's Hospital

Manchester, United Kingdom

Oxford University Hospitals NHS Foundation Trust

Oxford, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT07038824


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