Children's Hospital of Philadelphia leads or participates in hundreds of clinical trials. Use this database to learn the purpose of these trials, find out who can participate and more.
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The purpose of this research study is to learn more about the safety and effects of CTX001 (the “Study Product”) in patients ages 18 to 35 years who have been diagnosed with severe sickle cell disease. This is an open-label study in which subjects will receive one dose of the Study Product. The goal of is to see if a single “dose” of the Study Product allows the body to increase the amount of hemoglobin F (HbF) while decreasing the effects of SCD. CTX001, which is created by changing (editing) the DNA in the subjects blood stem cells near a gene called BCL11A
This study hopes to revise the currently used criteria for diagnoses of Juvenile Idiopathic Arthritis (JIA). This study aims to follow children newly diagnosed with JIA for up to five years. We will ask for a small blood sample for the first two visits, and for participants to fill out a back assessment. We will also record disease information from your medical record.