Duchenne Muscular Dystrophy (DMD)Phase 2ELEVATE-LTENCT07682129
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 study testing ENTR-601-44 and ENTR-601-45 in people with Duchenne Muscular Dystrophy (DMD), run by Entrada Therapeutics, Inc. at 17 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 20 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- ENTR-601-44 and ENTR-601-45
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 17 sitesin 5 countries
- Study dates
- Aug 2026to Mar 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 20 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
17 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 5 countries
United Kingdom6 sites
Leeds General Infirmary
Leeds, LS1 3EX
Alder Hey Children's NHS Foundation Trust
Liverpool, L122AP
Great Ormond Street Hospital for Children
London, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, M13 9WL
Freeman Hospital
Newcastle upon Tyne, NE1 3BZ
Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU
Italy4 sites
IRCCS Ospedale San Raffaele
Milan, 20132
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, 20162
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, 00168
Ospedale Pediatrico Bambino Gesu
Rome, 00165
Belgium3 sites
University Hospital Gent
Ghent, 9000
UZ Leuven
Leuven, 3000
Centre Hospitalier Régional de la Citadelle
Liège, 4000
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden, 2333 ZA
Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA
Spain2 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Sant Joan de Deu
Barcelona, 08950
About this study
Official 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)
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.
Read more
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)
- Being tested
- ENTR-601-44, ENTR-601-45
- Keywords
- Duchenne Muscular Dystrophy, DMD, Exon 44 skipping, Exon 45 skipping, exon skipping therapy, oligonucleotide therapy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ENTR-601-44Drug
ENTR-601-44 intravenous infusion
ENTR-601-45Drug
ENTR-601-45 intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| ENTR-601-44 | Experimental | ENTR-601-44 intravenous infusion every 6 weeks Receives: Drug: ENTR-601-44 |
| ENTR-601-45 | Experimental | ENTR-601-45 intravenous infusion every 6 weeks Receives: Drug: ENTR-601-45 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline through End of Study (up to 2 years).
Secondary outcomes (8)
Plasma concentration of study drug compounds and their final metabolite
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: From baseline through End of Study (up to 2 years).
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum
Measured: From baseline through End of Study (up to 2 years).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2026
- Study startAug 2026 (estimated)
- Primary completionMar 2032 (estimated)
- Study completionMar 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- 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.
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Entrada Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Entrada Therapeutics Clinical Trials, Study director, Entrada Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07682129
- Sponsor's protocol ID
- ENTR-601-DMD-202
- Other IDs
- 2025-525124-10-00, U1111-1334-9287, 2025
- Acronym
- ELEVATE-LTE
- Status last verified
- Jun 2026
- First submitted
- 11 Jun 2026
- Last updated
- 2 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT07682129
- Acronym / sponsor ID
- ELEVATE-LTE / ENTR-601-DMD-202
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, DMD, Exon 44 skipping, Exon 45 skipping, exon skipping therapy, oligonucleotide therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Entrada Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2026 (estimated)
- Primary completion
- Mar 2032 (estimated)
- Study completion
- Mar 2032 (estimated)
- First posted
- 2 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| ENTR-601-44 | Experimental | Drug: ENTR-601-44ENTR-601-44 intravenous infusion every 6 weeks |
| ENTR-601-45 | Experimental | Drug: ENTR-601-45ENTR-601-45 intravenous infusion every 6 weeks |
Who may qualify
- Ages
- 4 to 20 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ENTR-601-44ENTR-601-44 intravenous infusion | Drug | ENTR-601-44 |
| ENTR-601-45ENTR-601-45 intravenous infusion | Drug | ENTR-601-45 |
Primary outcome
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.
8 secondary outcomes
Plasma concentration of study drug compounds and their final metabolite
Time frame: 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).
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).
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).
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).
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).
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).
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum
Time frame: From baseline through End of Study (up to 2 years).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2026.
- Individual participant dataSharing planned: No. 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.
Related trials for Duchenne Muscular Dystrophy (DMD)
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Entrada Therapeutics Clinical Trials study director
Entrada Therapeutics, Inc.
17 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ENTR-601-44 and ENTR-601-45 in people with Duchenne Muscular Dystrophy (DMD), run by Entrada Therapeutics, Inc. at 17 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 2 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United Kingdom, Italy, Belgium, Netherlands, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jul 2026.