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

Information source: Nationwide Children's Hospital
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Duchenne Muscular Dystrophy

Intervention: Gentamicin infusions twice a week for six months (Drug)

Phase: Phase 1

Status: Completed

Sponsored by: Nationwide Children's Hospital

Official(s) and/or principal investigator(s):
Jerry R. Mendell, M.D., Principal Investigator, Affiliation: The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital


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.

Clinical Details

Official title: A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations

Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: 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.

Secondary outcome:

Determine if gentamicin given over six months improves muscle strength.

Determine if gentamicin given over six months increases dystrophin binding at the muscle membrane.

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.


Minimum age: 5 Years. Maximum age: 20 Years. Gender(s): Male.


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)

Locations and Contacts

Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea, Scottsdale, Arizona 85258, United States

University of Kansas, Kansas City, Kansas 66160-0001, United States

The Research Institute at Nationwide Children's Hospital, Columbus, Ohio 43205-2696, United States

Additional Information

Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital

Starting date: March 2007
Last updated: March 22, 2012

Page last updated: August 23, 2015

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