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Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy

NCT00428935 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2013-02-05

No results posted yet for this study

Summary

The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).

Conditions

Interventions

BIOLOGICAL

rAAV2.5-CMV-minidystrophin (d3990)

Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.

Sponsors & Collaborators

  • AskBio Inc

    collaborator INDUSTRY

  • Nationwide Children's Hospital

    lead OTHER

Principal Investigators

  • Jerry R. Mendell, MD · Nationwide Children's Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
15 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-03-31
Primary Completion
2009-03-31
Completion
2010-07-31

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 NCT00428935 on ClinicalTrials.gov