Duchenne Muscular Dystrophy (DMD)Phase 1/2ELEVATE-45NCT07038824
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
A Phase 1/2 study testing ENTR-601-45 in people with Duchenne Muscular Dystrophy (DMD), run by Entrada Therapeutics, Inc. at 15 sites in 5 countries.
Trial summary
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. 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-601-45
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 15 sitesin 5 countries
- Study dates
- 30 Aug 2025to 1 Mar 2029
- Phase
- Phase 1/2
- 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
- Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.
- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
- Part A: 4-20 years of age, inclusive.
- Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening.
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
- Other protocol-defined criteria apply.
Who cannot join
- Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
- Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety.
- Use of the following medications :
- Prior or current treatment with any exon skipping therapy within the previous 12 months
- Prior or current treatment with any gene therapy
- Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from 30 days prior to screening and until the end of the study
Show all 13Show fewer
- Use of an immunosuppressant (other than systemic or oral corticosteroid for DMD condition) from 30 days prior to screening until the end of the study.
- Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from 30 days prior to screening until the end of the study
- Laboratory abnormalities.
- Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
- Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) >450 msec at Screening or prior to the first dose of study drug on Day 1.
- Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).
- Other protocol-defined criteria apply.
Trial locations and contacts
15 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
15 sites in 5 countries
United Kingdom5 sites, 5 recruiting
- Recruiting
Leeds General Infirmary
Leeds, LS1 3EX
Cristina Martos Lozano, +441133923113, c.martoslozano@nhs.net
Cristina Martos Lozano
- Recruiting
Alder Hey Children's NHS Foundation Trust
Liverpool, L122AP
Rajesh Madhu, +4401512284811, Rajesh.Madhu@alderhey.nhs.uk
Rajesh Madhu
- Recruiting
Great Ormond Street Hospital for Children
London, WC1N 3JH
Giovanni Baranello, 0039 02074059200, g.baranello@ucl.ac.uk
Giovanni Baranello
- Not yet recruiting
Royal Manchester Children's Hospital
Manchester, M13 9WL
Gary McCullagh, +44 161 701 2346, gary.mccullagh@cmft.nhs.uk
Gary McCullagh
- Recruiting
Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU
Laurent Servais, 07423213373, laurent.servais@paediatrics.ox.ac.uk
Laurent Servais
Belgium3 sites, 3 recruiting
- Recruiting
University Hospital Gent
Ghent, 9000
Nicolas Deconinck, +32 473 96 66 19, Nicolas.Deconinck@uzgent.be
Nicolas Deconinck
- Recruiting
UZ Leuven
Leuven, 3000
Liesbeth De Waele, +32 1634 3845, Liesbeth.dewaele@uzleuven.be
Liesbeth De Waele
- Recruiting
Centre Hospitalier Régional de la Citadelle
Liège, 4000
Aurore Daron, +3243218515, Aurore.daron@citadelle.be
Aurore Daron
Italy3 sites, 3 recruiting
- Recruiting
IRCCS Ospedale San Raffaele
Milan, 20132
Stefano Previtali, 0226435080, previtali.stefano@hsr.it
Stefano Previtali
- Recruiting
Ospedale Pediatrico Bambino Gesu
Rome, 00165
Adele D'Amico, +393888449868, adele2.damico@opbg.net
Adele D'Amico
- Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, 00168
Eugenio Maria Mercuri, 06 3015 4860, eugeniomaria.mercuri@policlinicogemelli.it
Eugenio Maria Mercuri
Netherlands2 sites, 2 recruiting
- Recruiting
Leids Universitair Medisch Centrum
Leiden, 2333 ZA
Erik Niks, +31 (0)71 5262197, E.H.Niks@lumc.nl
Erik Niks
- Recruiting
Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA
Jan Groothuis, +31 (0)243614892, jan.groothuis@radboudumc.nl
Jan Groothuis
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitario Vall d'Hebron
Barcelona, 08035
David Gomez Andres, +34620539379, david.gomezandres@vallhebron.cat
David Gomez Andres
- Recruiting
Hospital Sant Joan de Deu
Barcelona, 08950
Andres Nascimento, +34936009733, andres.nascimento@sjd.es
Andres Nascimento
About this study
Official title: A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping With an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-45 and placebo are both called study treatments.
The study has 2 parts: Part A: to evaluate if ENTR-601-45 is safe and to determine the best dose of ENTR-601-45 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-45 at the dose determined in Part A. Participants will be able to roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated.
Read more
Participants will:
- Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B
- Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, muscle biopsies and exercise 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 result 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-601-45, ENTR-601-45 - matching placebo
- Keywords
- Duchenne Muscular Dystrophy, DMD, 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-601-45Drug
intravenous infusion
ENTR-601-45 - matching placeboDrug
intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| ENTR-601-45 | Experimental | intravenous infusion every 6 weeks Receives: Drug: ENTR-601-45 |
| Placebo | Placebo comparator | intravenous infusion every 6 weeks Receives: Drug: ENTR-601-45 - matching placebo |
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 62 weeks).
Secondary outcomes (11)
Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)
Measured: From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A).
Measured: Baseline, End of Study (Up to 25 weeks)
Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A).
Measured: Baseline, End of Study (Up to 25 weeks)
Percent change from baseline to end of Part A in exon 45 skipping measured in muscle biopsy (Part A)
Measured: Baseline, End of Study (Up to 25 weeks)
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)
Measured: From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period).
Measured: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period).
Measured: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period).
Measured: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period).
Measured: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period)
Ordinal scale with 0 as the minimum score and 34 as the maximum score (higher score - better outcome).
Measured: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period).
Ordinal scale with 0 as the minimum score and 42 as the maximum score (higher score - better outcome).
Measured: Baseline, End of Study (up to 62 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2025
- Study start30 Aug 2025
- Primary completion1 Mar 2029 (estimated)
- Study completion1 Mar 2029 (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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 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
- NCT07038824
- Sponsor's protocol ID
- ENTR-601-45-201
- Other IDs
- 2024-517499-39-00, U1111-1316-6093
- Acronym
- ELEVATE-45
- Status last verified
- May 2026
- First submitted
- 1 May 2025
- Last updated
- 13 Aug 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 analysed during the current study are not expected to be made available due to due to the data's high commercial sensitivity.
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping With an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)
- ClinicalTrials.gov ID
- NCT07038824
- Acronym / sponsor ID
- ELEVATE-45 / ENTR-601-45-201
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, DMD, exon skipping therapy, oligonucleotide therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Entrada Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Aug 2025 (actual)
- Primary completion
- 1 Mar 2029 (estimated)
- Study completion
- 1 Mar 2029 (estimated)
- First posted
- 26 Jun 2025 (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 the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-45 and placebo are both called study treatments.
The study has 2 parts: Part A: to evaluate if ENTR-601-45 is safe and to determine the best dose of ENTR-601-45 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-45 at the dose determined in Part A. Participants will be able to roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated.
Participants will:
- Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B
- Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, muscle biopsies and exercise 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 result 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ENTR-601-45 | Experimental | Drug: ENTR-601-45intravenous infusion every 6 weeks |
| Placebo | Placebo comparator | Drug: ENTR-601-45 - matching placebointravenous 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 (6 listed)
- Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.
- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.
- Part A: 4-20 years of age, inclusive.
- Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening.
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
Key exclusion criteria (13 listed)
- Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
- Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety.
- Use of the following medications :
- Laboratory abnormalities.
- Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
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-601-45intravenous infusion | Drug | ENTR-601-45 |
| ENTR-601-45 - matching placebointravenous infusion | Drug | Placebo |
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 62 weeks).
Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.
11 secondary outcomes
Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)
Time frame: From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A).
Time frame: Baseline, End of Study (Up to 25 weeks)
Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A).
Time frame: Baseline, End of Study (Up to 25 weeks)
Percent change from baseline to end of Part A in exon 45 skipping measured in muscle biopsy (Part A)
Time frame: Baseline, End of Study (Up to 25 weeks)
Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)
Time frame: From baseline through End of Study (Up to 62 weeks).
Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period).
Time frame: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period).
Time frame: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period).
Time frame: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period).
Time frame: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period)
Time frame: Baseline, End of Study (up to 62 weeks)
Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period).
Time frame: Baseline, End of Study (up to 62 weeks)
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 13 Aug 2026.
- Individual participant dataSharing planned: No. The datasets generated during and/or analysed during the current study are not expected to be made available due to due to the data's high commercial sensitivity.
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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.
15 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ENTR-601-45 in people with Duchenne Muscular Dystrophy (DMD), run by Entrada Therapeutics, Inc. at 15 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 6 inclusion and 13 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: looking for participants now, as of the sponsor's last update on 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United Kingdom, Belgium, Italy, 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Each site lists its own contact: choose the nearest one under Trial locations. 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 62 weeks).). 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 13 Aug 2026.