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

NCT04240314 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-09-11

Study results available
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Summary

Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.

Conditions

Interventions

BIOLOGICAL

scAAV9.U7.ACCA

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

Sponsors & Collaborators

  • Audentes Therapeutics

    collaborator INDUSTRY

  • Astellas Pharma Inc

    collaborator INDUSTRY

  • Megan Waldrop

    lead OTHER

Principal Investigators

  • Megan Waldrop, MD · Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
13 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-15
Primary Completion
2023-11-13
Completion
2025-07-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

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 NCT04240314 on ClinicalTrials.gov