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

May 15, 2026 updated by: Entrada Therapeutics, Inc.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Ghent, Belgium, 9000
        • Recruiting
        • University Hospital Gent
        • Principal Investigator:
          • Nicolas Deconinck
        • Contact:
      • Leuven, Belgium, 3000
        • Recruiting
        • UZ Leuven
        • Principal Investigator:
          • Liesbeth De Waele
        • Contact:
      • Liège, Belgium, 4000
        • Recruiting
        • Centre Hospitalier Régional de la Citadelle
        • Principal Investigator:
          • Aurore Daron
        • Contact:
  • Italy
      • Milan, Italy, 20132
        • Recruiting
        • IRCCS Ospedale San Raffaele
        • Contact:
        • Principal Investigator:
          • Stefano Previtali
      • Rome, Italy, 00165
        • Recruiting
        • Ospedale Pediatrico Bambino Gesù
        • Contact:
        • Principal Investigator:
          • Adele D'Amico
      • Rome, Italy, 00168
        • Recruiting
        • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
        • Principal Investigator:
          • Eugenio Maria Mercuri
        • Contact:
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Recruiting
        • Leids Universitair Medisch Centrum
        • Principal Investigator:
          • Erik Niks
        • Contact:
      • Nijmegen, Netherlands, 6525 GA
        • Recruiting
        • Stichting Radboud Universitair Medisch Centrum
        • Contact:
        • Principal Investigator:
          • Saskia Houwen
  • Spain
      • Barcelona, Spain, 08950
        • Recruiting
        • Hospital Sant Joan de Deu
        • Principal Investigator:
          • Andres Nascimento
        • Contact:
      • Barcelona, Spain, 08035
        • Recruiting
        • Hospital Universitario Vall d'Hebron
        • Contact:
        • Principal Investigator:
          • David Gomez Andres
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Recruiting
        • Leeds General Infirmary
        • Contact:
        • Principal Investigator:
          • Cristina Martos Lozano
      • Liverpool, United Kingdom, L122AP
        • Not yet recruiting
        • Alder Hey Children's NHS Foundation Trust
        • Principal Investigator:
          • Rajesh Madhu
        • Contact:
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital for Children
        • Principal Investigator:
          • Giovanni Baranello
        • Contact:
      • Manchester, United Kingdom, M13 9WL
        • Not yet recruiting
        • Royal Manchester Children's Hospital
        • Principal Investigator:
          • Gary McCullagh
        • Contact:
      • Oxford, United Kingdom, OX3 9DU
        • Recruiting
        • Oxford University Hospitals NHS Foundation Trust
        • Principal Investigator:
          • Laurent Servais
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ENTR-601-45
intravenous infusion every 6 weeks
Drug: ENTR-601-45
intravenous infusion
Placebo Comparator: Placebo
intravenous infusion every 6 weeks
Drug: ENTR-601-45 - matching placebo
intravenous infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period).
Time Frame: 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.
From baseline through End of Study (up to 62 weeks).

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period)
Time Frame: Baseline, End of Study (up to 62 weeks)
Ordinal scale with 0 as the minimum score and 34 as the maximum score (higher score - better outcome).
Baseline, End of Study (up to 62 weeks)
Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)
Time Frame: From baseline through End of Study (Up to 62 weeks).
From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A).
Time Frame: Baseline, End of Study (Up to 25 weeks)
Baseline, End of Study (Up to 25 weeks)
Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A).
Time Frame: Baseline, End of Study (Up to 25 weeks)
Baseline, End of Study (Up to 25 weeks)
Percent change from baseline to end of Part A in exon 45 skipping measured in muscle biopsy (Part A)
Time Frame: Baseline, End of Study (Up to 25 weeks)
Baseline, End of Study (Up to 25 weeks)
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)
Time Frame: From baseline through End of Study (Up to 62 weeks).
From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period).
Time Frame: Baseline, End of Study (up to 62 weeks)
Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period).
Time Frame: Baseline, End of Study (up to 62 weeks)
Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period).
Time Frame: Baseline, End of Study (up to 62 weeks)
Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period).
Time Frame: Baseline, End of Study (up to 62 weeks)
Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period).
Time Frame: Baseline, End of Study (up to 62 weeks)
Ordinal scale with 0 as the minimum score and 42 as the maximum score (higher score - better outcome).
Baseline, End of Study (up to 62 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Entrada Therapeutics, Inc.

Investigators

  • Study Director: Entrada Therapeutics Clinical Trials, Entrada Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 30, 2025

Primary Completion (Estimated)

March 1, 2029

Study Completion (Estimated)

March 1, 2029

Study Registration Dates

First Submitted

May 1, 2025

First Submitted That Met QC Criteria

June 17, 2025

First Posted (Actual)

June 26, 2025

Study Record Updates

Last Update Posted (Actual)

May 18, 2026

Last Update Submitted That Met QC Criteria

May 15, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ENTR-601-45-201
  • 2024-517499-39-00 (Ctis)
  • U1111-1316-6093 (Other Identifier: WHO)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

The datasets generated during and/or analysed during the current study are not expected to be made available due to due to the data's high commercial sensitivity.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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