Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy

NCT ID: NCT06900049

Last Updated: 2025-04-02

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-10-24
• Study Completion Date: 2026-12-31

Brief 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.

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)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

LE051 treatment

Single dose intravenous injection of LE051

Group Type: EXPERIMENTAL

LE051

Intervention Type: DRUG

LE051 dose escalation : dose 1 and dose 2.

Interventions

LE051

LE051 dose escalation : dose 1 and dose 2.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 8 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Shanghai Jiao Tong University School of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Jiwen Wang

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Jiwen Wang

Role: CONTACT

wangjiwen@scmc.com.cn

+86 189 1661 3192

Facility Contacts

Jiwen Wang

Role: primary

wangjiwen@scmc.com.cn

+86 189 1661 3192

Other Identifiers

LE051-1

Identifier Type: -

Identifier Source: org_study_id