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)

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)

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07682129

Acronym

ELEVATE-LTE

Enrollment

Registered

2026-07-02

Start date

2026-08-01

Completion date

2032-03-01

Last updated

2026-07-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

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

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.

Interventions

DRUGENTR-601-44

ENTR-601-44 intravenous infusion

DRUGENTR-601-45

ENTR-601-45 intravenous infusion

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

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.

Eligibility

Sex/Gender

MALE

Age

4 Years to 20 Years

Healthy volunteers

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

Design outcomes

Primary

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

Measure	Time frame
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum	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)	From baseline through End of Study (up to 2 years).
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 Performance of the Upper Limb v2.0 (PUL 2.0)	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).

Countries

Belgium, Italy, Netherlands, Spain, United Kingdom

Contacts

STUDY_DIRECTOREntrada Therapeutics Clinical Trials

Entrada Therapeutics, Inc.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 3, 2026