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A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy Clinical Trial

Official title:
A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

Clinical Trial Summary

The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.

Clinical Trial Description

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01957059
Study type Interventional
Source BioMarin Pharmaceutical
Contact
Status Terminated
Phase Phase 1/Phase 2
Start date June 2013
Completion date August 3, 2016
View Details
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