Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE) (ELEVATE-LTE)

June 26, 2026 updated by: Entrada Therapeutics, Inc.

A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)

This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).

The investigational medicines are currently being investigated in multiple ascending dose parent studies. After participants complete their respective parent study, there is a need to understand the effects of long-term administration of ENTR-601-44 and ENTR-601-45.

Participants enrolling in this study will begin this long-term extension (LTE) study at the dose level they received upon completion of the parent study with possible dose escalation in the LTE study based on emerging safety and efficacy data from the parent studies.

Participants will:

  • Receive study treatment in the form of multiple intravenous (IV) infusions (slow injections) into a vein over the course of several weeks
  • Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and excersice 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 results 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

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

80

Phase

  • Phase 2

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
        • University Hospital Gent
      • Leuven, Belgium, 3000
        • UZ Leuven
      • Liège, Belgium, 4000
        • Centre Hospitalier Regional de la Citadelle
  • Italy
      • Milan, Italy, 20132
        • IRCCS Ospedale San Raffaele
      • Milan, Italy, 20162
        • Fondazione Serena Onlus - Centro Clinico NeMO Milano
      • Roma, Italy, 00168
        • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
      • Rome, Italy, 00165
        • Ospedale Pediatrico Bambino Gesù
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leids Universitair Medisch Centrum
      • Nijmegen, Netherlands, 6525 GA
        • Stichting Radboud Universitair Medisch Centrum
  • Spain
      • Barcelona, Spain, 08035
        • Hospital Universitario Vall d'Hebron
      • Barcelona, Spain, 08950
        • Hospital Sant Joan De Deu
  • United Kingdom
      • Leeds, United Kingdom, LS1 3EX
        • Leeds General Infirmary
      • Liverpool, United Kingdom, L122AP
        • Alder Hey Children's NHS Foundation Trust
      • London, United Kingdom, WC1N 3JH
        • Great Ormond Street Hospital for Children
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Children's Hospital
      • Newcastle upon Tyne, United Kingdom, NE1 3BZ
        • Freeman Hospital
      • Oxford, United Kingdom, OX3 9DU
        • Oxford University Hospitals NHS Foundation Trust

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:

  • Willing and able to provide consent (if at age of majority) or assent (if a minor)
  • Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201
  • Males who are sexually active with a female partner of childbearing potential must agree to use condoms during sexual intercourse

Exclusion Criteria:

  • Any change from the applicable parent study eligibility criteria, including safety events during the parent study, that in the opinion of the investigator in consultation with the medical monitor and/or sponsor designee precludes safe use of study drug
  • Participant has a condition or circumstance that in the view of the investigator places the subject at high risk of poor treatment compliance or for not completing the study

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: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ENTR-601-44
ENTR-601-44 intravenous infusion every 6 weeks
Drug: ENTR-601-44
ENTR-601-44 intravenous infusion
Experimental: ENTR-601-45
ENTR-601-45 intravenous infusion every 6 weeks
Drug: ENTR-601-45
ENTR-601-45 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 2 years).
Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.
From baseline through End of Study (up to 2 years).

Secondary Outcome Measures

Outcome Measure
Time Frame
Plasma concentration of study drug compounds and their final metabolite
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in 10-Meter Walk/Run (10MWR)
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF)
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in Timed 4-Stair Climb (4SC)
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in stride velocity 95th centile (SV95C)
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in North Star Ambulatory Assessment (NSAA)
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Change from parent study Part A and OL Period baselines to LTE EOS in Performance of the Upper Limb v2.0 (PUL 2.0)
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum
Time Frame: From baseline through End of Study (up to 2 years).
From baseline through End of Study (up to 2 years).

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 (Estimated)

August 1, 2026

Primary Completion (Estimated)

March 1, 2032

Study Completion (Estimated)

March 1, 2032

Study Registration Dates

First Submitted

June 11, 2026

First Submitted That Met QC Criteria

June 26, 2026

First Posted (Actual)

July 2, 2026

Study Record Updates

Last Update Posted (Actual)

July 2, 2026

Last Update Submitted That Met QC Criteria

June 26, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ENTR-601-DMD-202
  • 2025-525124-10-00 (Ctis)
  • U1111-1334-9287 (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 analyzed during the current study are not expected to be made available 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.

Clinical Trials on Duchenne Muscular Dystrophy (DMD)

Clinical Trials on ENTR-601-44

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Panama

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe