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The purpose of this study is to examine the effect of the investigational drug GBT440 on patients with sickle cell disease. The study will also explore whether the GBT440 can help slow down the destruction of red blood cells and improve the health of blood vessels in the brain.
Subject to certain exclusion criteria, this study is enrolling participants of both genders, ages 12 through 17, who have sickle cell disease.
Males and Females: Ages 12 through 17
Following an initial visit for a screening examination, selected participants will be provided with study pills to be taken three times a day during the study period. Certain dietary restrictions also apply. Participants will be required to keep a journal documenting medication intake. Participants’ health and response to the medication will be closely monitored through follow up visits over the course of 24 weeks, including additional blood tests and physical examinations.
Visits: Up to 12
Duration: Approximately 24 weeks
Global Blood Therapeutics
Study Description at National Institutes of Health
Cheryl A. Hillery, MD
For more information about the study or enrollment, please contact:
Angela Martino, BSN, RN
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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Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.