Gentamicin Treatment of Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT00005574 |
Recruitment Status :
Completed
First Posted : December 10, 2002
Last Update Posted : March 4, 2008
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This study will evaluate the antibiotic gentamicin for treating patients with muscular dystrophy caused by a specific genetic abnormality known as a nonsense mutation. In studies of mice with this type of muscular dystrophy, gentamicin treatment produced positive changes in muscle tissue.
Patients with Duchenne or Becker muscular dystrophy caused by nonsense mutations by may be eligible for this 2-week study. Before starting treatment, patients will have evaluations of muscle strength and general well being. Two muscle tissue samples will be taken by needle biopsy, under local anesthetic and sedation. Because of potential risks of hearing loss and kidney toxicity associated with gentamicin, patients will also have a hearing test and blood and urine tests for kidney function before starting treatment. (Currently, gentamicin is commonly prescribed for serious infections of the lungs, heart, and digestive and urinary tracts; adverse effects of hearing loss and kidney toxicity can occur with excessively high drug doses.)
Patients will be hospitalized during drug treatment. Gentamicin will be given intravenously (through a vein) once a day for 14 days. Blood samples will be collected daily to monitor drug levels and determine dosage adjustments, if necessary. Urine samples will be collected to assess kidney function. Hearing tests will be done on days 7 and 10.
On the last day of the study, hearing, kidney function, and muscle strength will be tested and the results compared with pre-treatment levels. Blood and muscle samples will also be taken again for pre-treatment comparison. Hearing, blood, urine, and muscle strength tests will be repeated one month after treatment ends for comparison with previous results.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Becker Muscular Dystrophy Duchenne Muscular Dystrophy | Drug: Gentamicin | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Enrollment : | 4 participants |
Primary Purpose: | Treatment |
Official Title: | Gentamicin Treatment of Patients With Muscular Dystrophy Due to Nonsense Mutations in Dystrophin |
Study Start Date : | February 2000 |
Study Completion Date : | January 2001 |


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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Diagnosis of DMD or Becker muscular dystrophy with confirmed dystrophin nonsense mutation.
Measurable limb or pulmonary weakness.
Signed consent.
Must not have a history of hypersensitivity reaction to an aminoglycoside.
Must not have abnormal baseline hearing.
Must not have abnormal baseline kidney function or serum creatinine level.
Must not be currently enrolled in another clinical trial.
Must not have recent (within past 3 months) initiation of prednisone or creatinine therapy.
Must not have a history of significant concomitant illness.
Must not have concomitant use of aminoglycoside or other nephrotoxic agent.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00005574
United States, Maryland | |
National Institute of Neurological Disorders and Stroke (NINDS) | |
Bethesda, Maryland, United States, 20892 |
ClinicalTrials.gov Identifier: | NCT00005574 |
Other Study ID Numbers: |
000083 00-N-0083 |
First Posted: | December 10, 2002 Key Record Dates |
Last Update Posted: | March 4, 2008 |
Last Verified: | January 2000 |
Becker Muscular Dystrophy Duchenne Muscular Dystrophy Muscle Disease Myopathy |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |
Genetic Diseases, X-Linked Gentamicins Anti-Bacterial Agents Anti-Infective Agents Protein Synthesis Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |