NCT00451074

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: Male
• Age: 5 Years – 20 Years
• Healthy volunteers: Not accepted

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 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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Gentamicin infusions twice a week for six months	Gentamicin infusions twice a week

Timeline

• Start date: 2007-03-01
• Primary completion: 2009-07-01
• Completion: 2009-07-01
• First posted: 2007-03-23
• Last updated: 2012-03-23

Locations

3 sites across 1 country: United States