9
Participants
Start Date
June 30, 2013
Primary Completion Date
August 3, 2016
Study Completion Date
August 3, 2016
Regimen Selection Phase Group 2
"All doses of BMN053 will be administered as IV infusions. The proposed doses are as follows:~• 3 mg/kg"
Regimen Selection Phase Group 3
"All doses of BMN053 will be administered as IV infusions. The proposed doses are as follows:~• 4-6 mg/kg"
Treatment Phase Group 4
All doses of PRO053 will be administered as IV infusions. The proposed doses will be decided upon completion of the Regimen Selection Phase of Groups 2 and 3
Regimen Selection Phase Group 1 (COMPLETED)
All doses of BMN053 have been administered as subcutaneous injections.
Dosing Extension
All doses of PRO053 will be administered as IV infusions. The proposed doses will be decided upon completion of the Regimen Selection Phase of Groups 2 and 3 and the Treatment Phase Group 4.
UZ Leuven, Campus Gasthuisberg, Leuven
Institut de Myologie, Paris
Policlinico Universitario Agostino Gemelli, Rome
Leids Universitair Medisch Centrum, Leiden
Great Ormond Street Hospital for Children, London
Institute of Genetic Medicine International Centre for Life, Newcastle
Lead Sponsor
BioMarin Pharmaceutical
INDUSTRY