The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
This clinical research study is for women between the ages of 35 and 42 years who are having difficulty becoming pregnant and who would like to receive treatment within an assisted reproductive technology (ART) program in order to become pregnant. The purpose of this research study is to test the safety and effectiveness of an investigation drug called FE…
The purpose of this study is to study the effects of a new study drug known as Riociguat in people diagnosed with pulmonary hypertension (PH) associated with idiopathic interstitial pneumonia (IIP). Patients will be in the study for approximately 26 weeks in which they will take the pill Riogciguat three times a day.
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
This is a clinical research study designed to determine the safety and effectiveness of the TIVUS system, an investigational device used in the treatment of PAH. The TIVUS System is intended to treat patients with pulmonary artery hypertension by injuring the sympathetic nerves surrounding the pulmonary vasculature (denervation) using ultrasonic energy. The…
This study involves two infusions of either rituximab or placebo, two right heart catheterizations (6 months apart), and a variety of other clinical study procedures and exams.
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
The purpose of this study is to survey study participants to determine their knowledge and attitudes towards fertility, and interest in understanding fertility management options. Recruited participants will be contacted via e-mail and invited to participate in online surveys and have hormone markers measured at the Columbia University Medical Center.