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…
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…
This study is being done to determine if REGN2810 (anti-PD-1) in combination with ipilimumab (anti-CTLA-4) plus initial platinum-based doublet chemotherapy, or REGN2810 in combination with ipilimumab will prolong how long you live without your disease progressing compared to the standard of care pembrolizumab (anti-PD-1) monotherapy treatment. Neither…
We are conducting a survey to gather feedback about lung cancer patients experience with treatment and the challenges you faced. Your insights can help shape better care for future patients and explore how technology can be of help! The survey takes 30-40 minutes, and eligible participants who complete it will receive a gift card. Interested in…
We are doing this study because we want to find out if MRI brain scans alone is better or worse than MRI brain scans plus preventative brain radiation for patients with small lung cancer. The usual approach for patients with small cell lung cancer is prophylactic cranial irradiation. If you decide to take part in this study, you will either receive PCI for…
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 purpose of this study is to determine whether the tumor cells of lung cancer patients contain changes to the DNA sequence of two proteins found on the cells surface- ALK and EGFR. If the tumor contains changes to the DNA of one of these proteins, the patient may be invited to participate on a trial that will look at drugs which may target tumors that…
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 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…