Gentamicin Treatment of Muscular Dystrophy

• Status: Completed
• Trial end date: 2001-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
• Treatments: Gentamicins

Criteria

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.