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.
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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Gentamicin infusions twice a week
Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea
Scottsdale, Arizona, United States
University of Kansas
Kansas City, Kansas, United States
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States
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
Determine if gentamicin given over six months improves muscle strength.
Time frame: 6 months
Determine if gentamicin given over six months increases dystrophin binding at the muscle membrane.
Time frame: 6 months
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