261
Participants
Start Date
May 31, 2013
Primary Completion Date
November 25, 2015
Study Completion Date
November 25, 2015
BMN 165
After informed consent, eligible subjects will be randomized (1:1) to titrate up to one of two dose regimens: 20 mg/day or 40 mg/day. All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the Induction Period, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly BMN 165 dose to a daily dose regimen of 20 mg/day or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 mg/day or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until a minimum of approximately 26 weeks or a maximum of 36 weeks in the study.
Icahn School of Medicine at Mount Sinai Medical Center, New York
Albany Medical College, Albany
University of Rochester, Rochester
Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center, Pittsburgh
St. Christopher's Hospital for Children, Philadelphia
Emory Universty, Decatur
University of Florida Clinical Research Center, Gainesville
University of Miami Health System, Miami
University of South Florida, Tampa
Vanderbilt University Medical Center, Nashville
Weisskopf Child Evaluation Center / University of Louisville, Louisville
University of Kentucky Medical Center, Lexington
University Hospital Cleveland, Case Medical Center, Cleveland
Riley Children's Hospital, Indianapolis
Wayne State University, Detroit
Children's Hospital of Wisconsin, Milwaukee
Ann and Robert H Lurie Children's Hospital of Chicago, Chicago
Washington University Center for Applied Research Sciences, St Louis
University of Missouri, Columbia
University of Nebraska Medical Center, Omaha
University of Oklahoma Health Sciences Center, Oklahoma City
University of Texas Health Science at Houston, Houston
The Children's Hospital Colorado, Aurora
University of Utah, Salt Lake City
University of California, Altman Clinical and Translational Research Institute, La Jolla
UCSF Benioff Children's Hospital Oakland, Oakland
Oregon Health & Science University, Portland
University of Washington Medical Center, Seattle
Boston Children's Hospital, Boston
Cooper Health Systems, Camden
Atlantic Health System - Morristown Medical Center, Morristown
Lead Sponsor
BioMarin Pharmaceutical
INDUSTRY