Duchenne Muscular Dystrophy Clinical Trial
Official title:
A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.
This is a phase 2 multi center, multi national, open label, long term extension study. Up to
approximately 50 male subjects with Duchenne Muscular Dystrophy (DMD) who have previously
been treated with BMN 044 will be enrolled. Subjects will receive either IV infusions or SC
injections at pre-defined doses.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy
assessments will be conducted at regular intervals throughout the study.
Subjects will be permitted to continue in this study until the subject meets any of the
defined withdrawal criteria, BioMarin decides to halt the clinical development of BMN 044, or
BMN 044 receives marketing authorization.
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