This study will develop a data registry of patients diagnosed with uveal melanoma (UM). This registry will provide data that will be used to support the development of novel therapies for this disease. In this study, we are focused on trying to better understand how uveal melanoma behaves and how it is treated at different medical centers around the country.
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…
Our clinic is currently participating in the Brave Trial, a clinical research study for Duchenne muscular dystrophy (DMD). This study is being conducted to learn whether a study drug can improve motor functions in boys with DMD. If your child takes part, he'll receive at no cost: study drug or placebo, study-related procedures, reimbursement for…
The purpose of the treatment part of the study is to find out what effects drug crizotinib has, good and/or bad on people with "high risk" uveal melanoma after surgery or radiation for the eye. It is thought that giving crizotinib to patients with "high risk" uveal melanoma can lower the chance the cancer will come back. Uveal melanoma…
Moving forward in Charcot-Marie-Tooth disease. A clinical research study assessing the safety and effectiveness of an investigational product in people with Charcot-Marie-Tooth (CMT) disease experiencing muscle cramps is now enrolling. Participation is entirely voluntary. Contacting us does not mean that you are required to take part in this study. If you…
The purpose of this study is evaluate the benefits and safety of using the combination of drugs, AEB071 and BYL719 to treat patients with an advanced form of uveal melanoma. The study will have two parts. The first part twill test different dose levels of each drug.Once the highest safe dose level is found, another group of patients will be treated at 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…
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
You are invited to join voluntarily in a clinical research study to find out if the drug LXS196 alone or in combination with HDM201 is safe and has beneficial effects in people who have metastatic uveal melanoma. The purpose of this study is to determine the highest dose of LXS196 that can be given safely without unacceptable side effects as a single drug…
The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the…
This research study is being done to see if the study drug, IMCgp100, works and is safe as treatment for subjects with uveal melanoma who also test positive for HLA-A*0201. In this study IMCgp100 will be compared to three standard treatments: dacarbazine (a chemotherapy drug), ipilimumab (an immunotherapy drug), or pembrolizumab (an immunotherapy drug).…
The purpose of this study is to find out what effects, good and/or bad, intermittent dosing of the medication Selumetinib will have on you and your uveal melanoma. Selumetinib is a medication that blocks (or turns off) MEK, a protein activated in some uveal melanoma cells. Selumetinib is a MEK inhibitor. Blocking MEK may stop your cancer from growing.
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
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 Phase I study of a weekly intra-patient escalation dose regimen with IMCgp100 in patients with metastatic uveal melanoma (mUM). To help decide whether or not patients can take part in a research study looking at a new form of treatment for advanced uveal melanoma, their blood need to first be tested to determine their Human Leukocyte Antigen (HLA…