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Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons

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ClinicalTrials.gov Identifier: NCT00451074
Recruitment Status : Completed
First Posted : March 23, 2007
Last Update Posted : March 23, 2012
Information provided by (Responsible Party):

Study Description
Brief Summary:
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 or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Gentamicin infusions twice a week for six months Phase 1

Detailed Description:
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.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations
Study Start Date : March 2007
Primary Completion Date : July 2009
Study Completion Date : July 2009

Arms and Interventions

Intervention Details:
    Drug: Gentamicin infusions twice a week for six months
    Gentamicin infusions twice a week

Outcome Measures

Primary Outcome Measures :
  1. In this phase 1 clinical trial, safety will be measured via gentamicin trough levels, audiology, and renal function tests. These lab tests will remain in the normal range while infusing gentamicin twice a week for 6 month. [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Determine if gentamicin given over six months improves muscle strength. [ Time Frame: 6 months ]
  2. Determine if gentamicin given over six months increases dystrophin binding at the muscle membrane. [ Time Frame: 6 months ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age 5-20 years
  • Duchenne muscular dystrophy documented by written report of stop codon mutation analysis of the dystrophin gene.
  • Subject is capable of cooperating for efficacy and safety testing
  • Absent dystrophin on muscle biopsy
  • Subjects may be untreated, taking prednisone or comparable corticosteroids
  • Subjects taking corticosteroids must be on the same dose for at least 3 months (90 days) prior to the start of the study.

Exclusion Criteria:

  • Known allergy to any aminoglycoside or sulfate compounds
  • Current use of potential nephrotoxic or ototoxic drug
  • Current use of corticosteroids has not been stable for 3 months (90) days
  • Known mutation at nucleotide 1555 in 12S rRNA gene of mitochondrial DNA (predisposes to aminoglycoside hearing loss and commercially available via Athena Diagnostics Lab). This DNA testing (Hearing susceptibility test) will be made available through funding from this grant.
  • Inability to hear within the range of 0 to 25 dB in any hearing frequency by pure tone audiometry
  • Cystatin C equal to or > 1.4mg/L
  • Other medical condition that would impede the conduct of study (e.g., congestive heart failure)
Contacts and Locations

Information from the National Library of Medicine

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): NCT00451074

United States, Arizona
Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea
Scottsdale, Arizona, United States, 85258
United States, Kansas
University of Kansas
Kansas City, Kansas, United States, 66160-0001
United States, Ohio
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205-2696
Sponsors and Collaborators
Nationwide Children's Hospital
National Institutes of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Principal Investigator: Jerry R. Mendell, M.D. The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital
More Information

Additional Information:
Responsible Party: Jerry R. Mendell, Director, Center for Gene Therapy, Nationwide Children's Hospital
ClinicalTrials.gov Identifier: NCT00451074     History of Changes
Other Study ID Numbers: NS043186
7R01NS043186 ( U.S. NIH Grant/Contract )
First Posted: March 23, 2007    Key Record Dates
Last Update Posted: March 23, 2012
Last Verified: March 2012

Keywords provided by Jerry R. Mendell, Nationwide Children's Hospital:
Stop codon mutations

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Anti-Bacterial Agents
Anti-Infective Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action