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ACTIVE NOT RECRUITING
NCT06392724
EARLY_PHASE1

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

Sponsor: Peking Union Medical College Hospital

View on ClinicalTrials.gov

Summary

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

Official title: A Single-arm, Open-label, Single-center Study to Evaluate the Safety and Tolerability of Intravenous GEN6050X Gene Therapy in Ambulatory Boys With Duchenne Muscular Dystrophy (DMD).

Key Details

Gender

MALE

Age Range

4 Years - 10 Years

Study Type

INTERVENTIONAL

Enrollment

3

Start Date

2024-07-05

Completion Date

2027-12

Last Updated

2025-07-29

Healthy Volunteers

No

Interventions

GENETIC

GEN6050X intravenous injection

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

Locations (1)

Peking Union Medical College Hospital

Beijing, China