Duchenne Muscular Dystrophy (DMD)Early Phase 1NCT06900049
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
A Early Phase 1 study testing LE051 in people with Duchenne Muscular Dystrophy (DMD), run by Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy. Read more about this study
- Who is it for?
- Ages 4 to 8 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- LE051
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 24 Oct 2024to 31 Dec 2026
- Phase
- Early Phase 1
- 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 8 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male，4-8 years old at the beginning of screening (including boundary values;
- DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No.51.
- The subjects and/or his guardian voluntary participate in this trial and can comprehend and sign ICF.
Who cannot join
- Clinical signs of heart failure: left ventricular ejection fraction (LVEF) <40%；
- The average FVC percentage of the predicted value is less than 40%；
- 12 lead ECG QT interval (QTc) >0.45 seconds.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200127
Jiwen Wang, +86 189 1661 3192, wangjiwen@scmc.com.cn
Jiwen Wang
About this study
Official title: Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
Read the detailed description
This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of LE051 after a single intravenous infusion in DMD patients, as well as the long-term safety and efficacy.
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder arising from mutations in the dystrophin gene, leading to muscle weakness, disability, and premature mortality. LE051, an investigational therapy, incorporates a ADAR recruiting RNA expression cassette targeting human exon 51 and is delivered via adeno-associated virus. By inducing exon 51 skipping, LE051 holds the potential to treat approximately 13% of DMD patients.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- LE051
- Keywords
- Duchenne Muscular Dystrophy
- 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.
LE051Drug
LE051 dose escalation : dose 1 and dose 2.
| Study group | Role | What they receive |
|---|---|---|
| LE051 treatment | Experimental | Single dose intravenous injection of LE051 Receives: Drug: LE051 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of AEs, SAEs
Measured: from day 1 to week 52 after treatment
Secondary outcomes (7)
Changes in North Star Ambulatory Assessment (NSAA) Scores Compared to Baseline
The North Star Ambulatory Assessment (NSAA) comprises 17 items yielding a total score between 0 and 34 points, with increased scores correlating with improved motor performance.
Measured: from day 1 to week 52 after treatment
Changes in 6-Minute Walk Distance Compared to Baseline
Measured: from day 1 to week 52 after treatment
Changes in Supine-to-Stand Time Compared to Baseline
Measured: from day 1 to week 52 after treatment
Changes in 4-Stair Climb Time Compared to Baseline
Measured: from day 1 to week 52 after treatment
Changes in 10-Meter Walk/Run Time Compared to Baseline
Measured: from day 1 to week 52 after treatment
Changes in Dystrophin Protein Expression Levels in Muscle Tissue Compared to Baseline
Measured: from day 1 to week 52 after treatment
Changes in the Percentage of Dystrophin-Positive Muscle Fibers Compared to Baseline
Measured: from day 1 to week 52 after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2025
- Study start24 Oct 2024
- Primary completion20 Sep 2026 (estimated)
- Study completion31 Dec 2026 (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.
- More trials run by Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Jiao Tong University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jiwen Wang, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06900049
- Sponsor's protocol ID
- LE051-1
- Status last verified
- Mar 2025
- First submitted
- 23 Mar 2025
- Last updated
- 2 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT06900049
- Acronym / sponsor ID
- LE051-1
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- 24 Oct 2024 (actual)
- Primary completion
- 20 Sep 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 28 Mar 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.
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
Detailed description
This is a single-arm, open-label study to evaluate the safety, tolerability, efficacy, pharmacokinetic, pharmacodynamic, and immune response of LE051 after a single intravenous infusion in DMD patients, as well as the long-term safety and efficacy.
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder arising from mutations in the dystrophin gene, leading to muscle weakness, disability, and premature mortality. LE051, an investigational therapy, incorporates a ADAR recruiting RNA expression cassette targeting human exon 51 and is delivered via adeno-associated virus. By inducing exon 51 skipping, LE051 holds the potential to treat approximately 13% of DMD patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LE051 treatment | Experimental | Drug: LE051Single dose intravenous injection of LE051 |
Who may qualify
- Ages
- 4 to 8 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male，4-8 years old at the beginning of screening (including boundary values;
- DMD subjects with a clinical diagnosis of DMD referred to the Duchenne Clinical Practice Guidelines for Progressive Muscular Dystrophy (2020 edition) and whose genetic test results were confirmed to be applicable to exon skipping at No.51.
- The subjects and/or his guardian voluntary participate in this trial and can comprehend and sign ICF.
Key exclusion criteria (3 listed)
- Clinical signs of heart failure: left ventricular ejection fraction (LVEF) <40%；
- The average FVC percentage of the predicted value is less than 40%；
- 12 lead ECG QT interval (QTc) >0.45 seconds.
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 |
|---|---|---|
| LE051LE051 dose escalation : dose 1 and dose 2. | Drug | LE051 treatment |
Primary outcome
Frequency of AEs, SAEs
Time frame: from day 1 to week 52 after treatment
7 secondary outcomes
Changes in North Star Ambulatory Assessment (NSAA) Scores Compared to Baseline
Time frame: from day 1 to week 52 after treatment
Changes in 6-Minute Walk Distance Compared to Baseline
Time frame: from day 1 to week 52 after treatment
Changes in Supine-to-Stand Time Compared to Baseline
Time frame: from day 1 to week 52 after treatment
Changes in 4-Stair Climb Time Compared to Baseline
Time frame: from day 1 to week 52 after treatment
Changes in 10-Meter Walk/Run Time Compared to Baseline
Time frame: from day 1 to week 52 after treatment
Changes in Dystrophin Protein Expression Levels in Muscle Tissue Compared to Baseline
Time frame: from day 1 to week 52 after treatment
Changes in the Percentage of Dystrophin-Positive Muscle Fibers Compared to Baseline
Time frame: from day 1 to week 52 after treatment
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 Apr 2025.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing LE051 in people with Duchenne Muscular Dystrophy (DMD), run by Shanghai Jiao Tong University School of Medicine at 1 site in China.
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 3 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 2 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Contact Jiwen Wang (+86 189 1661 3192, wangjiwen@scmc.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of AEs, SAEs (time frame: from day 1 to week 52 after treatment). 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 Apr 2025.