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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 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 80

Last updated 2026-07-02

No results posted yet for this study

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)

Interventions

DRUG

ENTR-601-44

ENTR-601-44 intravenous infusion

DRUG

ENTR-601-45

ENTR-601-45 intravenous infusion

Sponsors & Collaborators

  • Entrada Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Entrada Therapeutics Clinical Trials · Entrada Therapeutics, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
20 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-08-31
Primary Completion
2032-03-31
Completion
2032-03-31
FDA Drug
Yes

Countries

  • Belgium
  • Italy
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07682129 on ClinicalTrials.gov