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

NCT06900049 | Recruiting Early Phase 1

• 1 other identifier: LE051-1
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Frequency of AEs, SAEs

  from day 1 to week 52 after treatment

Secondary Outcomes (7)

• Changes in North Star Ambulatory Assessment (NSAA) Scores Compared to Baseline

  from day 1 to week 52 after treatment

• Changes in 6-Minute Walk Distance Compared to Baseline

  from day 1 to week 52 after treatment

• Changes in Supine-to-Stand Time Compared to Baseline

  from day 1 to week 52 after treatment

• Changes in 4-Stair Climb Time Compared to Baseline

  from day 1 to week 52 after treatment

• Changes in 10-Meter Walk/Run Time Compared to Baseline

  from day 1 to week 52 after treatment

Study Arms (1)

LE051 treatment

EXPERIMENTAL

Single dose intravenous injection of LE051

Drug: LE051

Interventions

LE051 DRUG

LE051 dose escalation : dose 1 and dose 2.

LE051 treatment

Eligibility Criteria

• Age: 4 Years - 8 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead

Study Sites (1)

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

Shanghai, Shanghai Municipality, 200127, China

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Jiwen Wang

CONTACT

+86 189 1661 3192; wangjiwen@scmc.com.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 23, 2025
• First Posted: March 28, 2025
• Study Start: October 24, 2024
• Primary Completion (Estimated): September 20, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: April 2, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)