Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Recruiting
Early Phase 1
Interventional Study
Duchenne Muscular Dystrophy (DMD)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 4 – 8
- Sex
- Male
- Trial phase
- Early Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 12 (estimated)
- Sponsor
- Shanghai Jiao Tong University School of Medicine · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
Key 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 confirme…
Contacts