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

NCT06900049 · Status: RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-04-02

No results posted yet for this study

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)

Interventions

DRUG

LE051

LE051 dose escalation : dose 1 and dose 2.

Sponsors & Collaborators

  • Shanghai Jiao Tong University School of Medicine

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
8 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-10-24
Primary Completion
2026-09-20
Completion
2026-12-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06900049 on ClinicalTrials.gov