The purpose of this study is to examine the causes and pathology of alopecia areata and other hair and skin disorders. Skin biopsies and blood and/or clinical information collected in the study will be used for research aiming at developing new options for the treatment of alopecia areata and other skin and hair disorders.
The purpose of this study is to determine if measuring blood levels of proteins called POMC, AgRP, and chromogrannin A (CGA) can be used to diagnose and track tumor growth and recurrence in patients that have ACTH-dependent Cushing's syndrome, non-secreting pituitary tumors, and other neuroendocrine tumors. We invite you to participate if you have: a…
Genetic studies help us to understand why some people develop certain diseases while others don't and also provide important clues about how best to treat the disease.
This study is for individuals who have Cushings disease and want to join voluntarily in this study to find out if a study drug called osilodrostat (LCI699) is safe and has beneficial effects in people who have Cushings disease. Cushings disease is a rare but debilitating disease. Cushings disease is caused by a benign tumor of the pituitary gland. The tumor…
We are conducting a research study on how psoriasis might be caused or worsened by high levels of the blood sugar-lowering hormone insulin. We are seeking people with plaque psoriasis, ages 18-65, who are overweight or obese with a body mass index of 25-35. Participants will have blood drawn and small skin biopsies taken from psoriasis plaques and nearby…
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…
The purpose of this study is to confirm the effectiveness and safety of LCI699 in treating patients with Cushing's disease. Patients will be treated with the invesitigational drug LCI699 at doses up to 60 mg a day, and possibly for a short period of time with placebo. A placebo is a dummy drug ("pill") with no active medicine inside.
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 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…