|
|
|
|
|
|
Sponsors and Collaborators: |
Nationwide Children's Hospital Asklepios Biopharmaceutical Inc. |
Information provided by: | Nationwide Children's Hospital |
ClinicalTrials.gov Identifier: | NCT00428935 |
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).
Condition | Intervention | Phase |
Duchenne Muscular Dystrophy |
Gene Transfer: rAAV vector expressing Mini-Dystrophin with CMV promoter |
Phase I |
Genetics Home Reference related topics: | Duchenne and Becker muscular dystrophy L1 syndrome |
MedlinePlus related topics: | Muscular Dystrophy |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double-Blind, Placebo Control, Single Group Assignment, Safety Study |
Official Title: | Phase 1 Clinical Trial of rAAV2.5-CMV-Mini-Dystrophin Gene Vector in Duchenne Muscular Dystrophy |
Estimated Enrollment: | 6 |
Study Start Date: | March 2006 |
Estimated Study Completion Date: | December 2008 |
This phase I randomized double blind dose escalation study investigates the safety and efficacy of the mini-dystrophin gene transferred to the biceps muscle for Duchenne muscular dystrophy patients, ages 5 to 12 years of age, using a recombinant adeno-associated virus. Eligible participants must have a known dystrophin gene mutation and may be concurrently treated with corticoid steroids. The mini-dystrophin gene or a placebo agent (normal saline or empty viral capsids) are injected directly into both biceps muscles while under conscious sedation. Following the gene transfer, patients are admitted to the hospital for 48 hours of observation followed by weekly outpatient visits at the Columbus Children's Hospital Neuromuscular Clinic. A bilateral muscle biopsy is preformed following 6 weeks with long term follow up will consisting of bi-annual visits for the next 2 years.
Ages Eligible for Study: | 5 Years to 15 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Ohio | |||||
Columbus Children's Hospital | |||||
Columbus, Ohio, United States, 43205 |
Nationwide Children's Hospital |
Asklepios Biopharmaceutical Inc. |
Principal Investigator: | Jerry R. Mendell, MD | Nationwide Children's Hospital |
Columbus Children's Research Institute 
  |
Asklepios BioPharmaceutical Inc. 
  |
Muscular Dystrophy Association 
  |
Study ID Numbers: | CCRI IRB05-00118 |
First Received: | January 26, 2007 |
Last Updated: | March 18, 2007 |
ClinicalTrials.gov Identifier: | NCT00428935 |
Health Authority: | United States: Food and Drug Administration |
|
|
|
|
|