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The purpose of this multi-center study is to determine the best dose of the investigational drug, BPS804 to treat osteogenesis imperfecta, also known as “brittle bone disease.” This will be done by measuring the effects on bones of different doses of BPS804 compared to receiving no drug (placebo).
Subject to certain exclusion criteria, the study accepts pediatric candidates ages 18 and older, who have osteogenesis imperfecta Type I, III or IV with a confirmed defect in the COL1A1/COL1A2 genes, and who have had one or more fractures in the past 24 months.
Males and Females: 18 years and older
Following a screening period that will last no more than 28 days for test results, participants will be randomly assigned to receive one of three doses of BPS804 or a placebo. Dosages will be provided intravenously during visits once a month for 12 months. During certain study visits, participants will receive physical examinations and various other tests, such as electrocardiograms, bone scans and hearing exams. When treatment with the study medicine stops, participants will have an end-of-treatment visit and one additional follow-up visit.
Duration: About 15 months
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Danielle Black, BS
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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