Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: LE051.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy (DMD). Basic parameters: 4 years — 8 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Evaluation of the Safety, Tolerability, and Efficacy of a Single Intravenous Injection of LE051 in Patients With Duchenne Muscular Dystrophy (DMD)
Overview
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.
Interventions
- Drug LE051
LE051 dose escalation : dose 1 and dose 2.
Primary outcome measures
- Frequency of AEs, SAEs [Time frame: from day 1 to week 52 after treatment]
Secondary outcome measures (7)
- 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]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine — Shanghai
Identifiers
NCT: NCT06900049 · LE051-1