Under construction – not a live service
Sign in
Taking people in ChinaEarly phase 1

Tests LE051 injection in children with Duchenne muscular dystrophy

Tests LE051, given by a single injection, in children with Duchenne muscular dystrophy.

Plain-language title generated from the registry record
Registry title: Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy

Key facts

Where
Shanghai, ChinaSee the site
Phase
Early phase 1

AI summary: who the study is looking for

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for boys aged 4 to 8 with Duchenne muscular dystrophy whose gene test shows they can be treated with exon 51 skipping. It tests the safety and effect of a single drug infusion called LE051.

Age
4 to 8 years old, including 4 and 8, at the start of screening
Sex
Male only
Disease stage
Clinical diagnosis of Duchenne muscular dystrophy (DMD), confirmed by a gene test showing it can be treated by skipping exon 51
Earlier treatment
The trial studies DMD patients treated with exon 51 skipping therapy
Biomarker or test result
Gene test result confirming the DMD change can be treated by skipping exon 51

Main reasons for not taking part

  • Signs of heart failure: left ventricular ejection fraction (LVEF) below 40%
  • Average FVC (lung capacity) below 40% of the predicted value
  • 12-lead ECG QT interval (QTc) above 0.45 seconds

Read by machine on 9 November 2026.

Could this be for you?

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 4 and 8 years old?

Source: NCT06900049, who can take part

Where in China

  • Shanghai Children's Medical Center, Shanghai Jiao Tong University School of MedicineShanghai, ChinaTaking people

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Trial details

Who runs it
Shanghai Jiao Tong University School of Medicine
Trial code
NCT06900049
Record updated
2 April 2025
Original description
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 full original descriptionShow less

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.

Inclusion: 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: Clinical signs of heart failure: left ventricular ejection fraction (LVEF) <40%；; 12 lead ECG QT interval (QTc) >0.45 seconds; The average FVC percentage of the predicted value is less than 40%；.

ClinicalTrials.gov NCT06900049