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)

NCT07682129 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 3 other identifiers: ENTR-601-DMD-2022025-525124-10-00U1111-1334-9287
• Study Type: interventional
• Target: 80
• Locations: 5 countries
• Sites: 17

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy; DMD; Exon 44 skipping; Exon 45 skipping; exon skipping therapy; oligonucleotide therapy

Outcome Measures

Primary Outcomes (1)

• Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period)

  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 Outcomes (8)

• Plasma concentration of study drug compounds and their final metabolite

  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)

  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)

  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)

  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)

  From baseline through End of Study (up to 2 years).

Study Arms (2)

ENTR-601-44

EXPERIMENTAL

ENTR-601-44 intravenous infusion every 6 weeks

Drug: ENTR-601-44

ENTR-601-45

EXPERIMENTAL

ENTR-601-45 intravenous infusion every 6 weeks

Drug: ENTR-601-45

Interventions

ENTR-601-44 DRUG

ENTR-601-44 intravenous infusion

ENTR-601-44

ENTR-601-45 DRUG

ENTR-601-45 intravenous infusion

ENTR-601-45

Eligibility Criteria

• Age: 4 Years - 20 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Entrada Therapeutics, Inc.: lead

Study Sites (17)

University Hospital Gent

Ghent, 9000, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Centre Hospitalier Régional de la Citadelle

Liège, 4000, Belgium

Location

IRCCS Ospedale San Raffaele

Milan, 20132, Italy

Location

Fondazione Serena Onlus - Centro Clinico NeMO Milano

Milan, 20162, Italy

Location

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

Roma, 00168, Italy

Location

Ospedale Pediatrico Bambino Gesu

Rome, 00165, Italy

Location

Leids Universitair Medisch Centrum

Leiden, 2333 ZA, Netherlands

Location

Stichting Radboud Universitair Medisch Centrum

Nijmegen, 6525 GA, Netherlands

Location

Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Location

Leeds General Infirmary

Leeds, LS1 3EX, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, L122AP, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

Freeman Hospital

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, OX3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Entrada Therapeutics Clinical Trials

  Entrada Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants with DMD who completed either clinical study ENTR-601-44-201 or ENTR-601-45-201 will begin the Long Term Extension (LTE) at the dose level they received upon completion of the open-label portion of the parent study. Dose escalation in the LTE study may be permitted based on emerging safety and efficacy data from the parent studies.

Study Record Dates

• First Submitted: June 11, 2026
• First Posted: July 2, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): March 1, 2032
• Study Completion (Estimated): March 1, 2032
• Last Updated: July 2, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

IT (4); GB (6); NL (2); ES (2); BE (3)