Status:
COMPLETED
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
Lead Sponsor:
Nationwide Children's Hospital
Collaborating Sponsors:
National Institutes of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
5-20 years
Phase:
PHASE1
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.
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 t...
Eligibility Criteria
Inclusion
- 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
- 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)
Key Trial Info
Start Date :
March 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2009
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT00451074
Start Date
March 1 2007
End Date
July 1 2009
Last Update
March 23 2012
Active Locations (3)
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1
Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea
Scottsdale, Arizona, United States, 85258
2
University of Kansas
Kansas City, Kansas, United States, 66160-0001
3
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205-2696