drugset / Trial / NCT00451074
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
Phase 1
Completed
12 enrolled
Nationwide Children's Hospital
National Institute of Neurological Disorders and Stroke (NINDS) · collabNational Institutes of Health (NIH) · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2007-03
- Primary completion
- 2009-07
- Completion
- 2009-07
Publications
- Results Malik V, Rodino-Klapac LR, Viollet L, Mendell JR. Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy. Ther Adv Neurol Disord. 2010 Nov;3(6):379-89. doi: 10.1177/1756285610388693.
- Results Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Gentamicin | Small molecule | 7.5 mg/kg | Intravenous |