NCT04240314View on ClinicalTrials.gov

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

January 15, 2020

Primary Completion Date

November 13, 2023

Study Completion Date

July 1, 2025

Conditions
Duchenne Muscular Dystrophy
Interventions
BIOLOGICAL

scAAV9.U7.ACCA

A single dose of scAAV9.U7.ACCA will be systemically delivered via a peripheral vein injection.

Trial Locations (1)

43205

Nationwide Children's Hospital, Columbus

Sponsors
All Listed Sponsors
collaborator

Audentes Therapeutics

INDUSTRY

collaborator

Astellas Pharma Inc

INDUSTRY

lead

Megan Waldrop

OTHER