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NCT Number: NCT07682129

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)

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.

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Key information

Conditions

Duchenne Muscular Dystrophy (DMD) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

4 year–20 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

University Hospital Gent, Ghent, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ENTR-601-44

Drug

ENTR-601-44 intravenous infusion

ENTR-601-45

Drug

ENTR-601-45 intravenous infusion

Primary outcomes

  1. 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.

Secondary outcomes

  1. Plasma concentration of study drug compounds and their final metabolite

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

  2. 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).

  3. 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).

  4. 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).

  5. 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).

  6. 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).

  7. 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).

  8. Anti-drug antibody (ADA) and anti-dystrophin antibody in serum

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

Sponsors and collaborators

Lead sponsor

Entrada Therapeutics, Inc.

Industry

Registry information

Official study title

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)

Acronym: ELEVATE-LTE

Important dates

Study start
2026
Primary completion
2032
Study completion
2032
First posted
Jul 2, 2026
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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