Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsors and Collaborators: |
Nationwide Children's Hospital National Institutes of Health (NIH) |
---|---|
Information provided by: | Nationwide Children's Hospital |
ClinicalTrials.gov Identifier: | NCT00451074 |
The purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.
Condition | Intervention | Phase |
---|---|---|
Duchenne Muscular Dystrophy |
Drug: Gentamicin infusions twice a week for six months |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Dose Comparison, Single Group Assignment, Safety Study |
Official Title: | A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations |
Estimated Enrollment: | 12 |
Study Start Date: | March 2007 |
Estimated Study Completion Date: | June 2009 |
Estimated Primary Completion Date: | June 2009 (Final data collection date for primary outcome measure) |
The primary purpose of this second cohort is to see if the IV Medication, gentamicin, is safe to give twice a week for six months to boys with Duchenne muscular dystrophy (DMD). Secondarily, we want to know if gentamicin can help strengthen the muscles of boys with DMD who have a particular type of genetic mutation known as a stop codon. The gentamicin is thought to allow for "read-through" of this type of mutation which would allow for the production of dystrophin, a protein which is lacking in boys with DMD.
Ages Eligible for Study: | 5 Years to 20 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Laurence M. Viollet, Ph.D. | (614) 722-2238 | Laurence.Viollet@nationwidechildrens.org |
Contact: Christopher J. Shilling, MS | (614) 722-6960 | Chris.Shilling@nationwidechildrens.org |
United States, Arizona | |
Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea | Recruiting |
Scottsdale, Arizona, United States, 85258 | |
Contact: Richardo D Bien-Willner, BS 480-314-1007 ext 1026 rwillner@azneuromuscular.org | |
Principal Investigator: Kumaraswamy Sivakumar, MD, MRCP | |
United States, Kansas | |
University of Kansas | Recruiting |
Kansas City, Kansas, United States, 66160-0001 | |
Contact: Victoria Watts, BSN, RN 913-588-5479 vwatts@kumc.edu | |
Principal Investigator: Richard J Barohn, MD, FAAN | |
United States, Ohio | |
The Research Institute at Nationwide Children's Hospital | Recruiting |
Columbus, Ohio, United States, 43205-2696 | |
Contact: Laurence Viollet, Ph.D. 614-722-2238 laurence.viollet@nationwidechildrens.org | |
Contact: Christopher J. Shilling, M.S. (614) 722-2238 chris.shilling@nationwidechildrens.org | |
Principal Investigator: Jerry R. Mendell, M.D. |
Principal Investigator: | Jerry R. Mendell, M.D. | The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital |
Responsible Party: | Nationwide Children's Hospital ( Jerry R. Mendell, MD ) |
Study ID Numbers: | NS043186, NS043186 |
Study First Received: | March 21, 2007 |
Last Updated: | November 7, 2008 |
ClinicalTrials.gov Identifier: | NCT00451074 |
Health Authority: | United States: Institutional Review Board |
Stop codon mutations |
Muscular dystrophy, Duchenne and Becker type Muscular Dystrophies Muscular Diseases Becker's muscular dystrophy Muscular Disorders, Atrophic Musculoskeletal Diseases Neuromuscular Diseases |
Genetic Diseases, Inborn Muscular Dystrophy, Duchenne Gentamicins Genetic Diseases, X-Linked Duchenne muscular dystrophy Atrophy Muscular dystrophy |
Protein Synthesis Inhibitors Anti-Infective Agents Anti-Bacterial Agents Molecular Mechanisms of Pharmacological Action |
Therapeutic Uses Nervous System Diseases Enzyme Inhibitors Pharmacologic Actions |