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A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.

NCT06392724 · Status: ACTIVE_NOT_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-07-29

No results posted yet for this study

Summary

The study will evaluate the safety and tolerability of GEN6050X gene therapy in Duchenne muscular dystrophy (DMD) patients amenable to exon 50 skipping.

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Interventions

GENETIC

GEN6050X intravenous injection

GEN6050X is an intravenously administered human DMD exon 50 skipping base editing drug.

Sponsors & Collaborators

  • Peking Union Medical College Hospital

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2024-07-05
Primary Completion
2025-12-31
Completion
2027-12-31

Countries

  • China

Study Locations

More Related Trials

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 NCT06392724 on ClinicalTrials.gov