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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
7
Universitair Ziekenhuis Leuven
Leuven, Belgium
S.Anna Hospital
Ferrara, Italy
Policlinico Univsersitario Agostino Gemelli
Rome, Italy
Leiden University Medical Center
Leiden, Netherlands
Number of subjects with 1 or more treatment emergent adverse events following BMN044 dosing
Time frame: Through study completion, an average of 1 year
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Drottning Silvias Barn- ochungdomssjukhus
Gothenburg, Sweden