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…
Are you undergoing a stem cell transplant? You may be at high risk for infections after the transplant. This study is looking at the safety and effectiveness of an investigational medication for those who have undergone a stem cell transplant. Recovery after a stem cell transplant can already be challenging without the added worry of life-threatening viral…
This study is testing a drug called GDC-8264, which is being developed to treat acute graft-versus-host-disease (acute GVHD). GDC-8264 is an experimental drug, which means health authorities have not approved GDC-8264 in combination with standard medications for the treatment of acute GVHD. The purpose of this study is to evaluate the effects, good or bad,…
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 test the good and bad effects of the combined treatment, using the drugs cetuximab and avelumab as well as avelumab by itself. Avelumab is a drug that works by triggering the immune system so it can try to attack the cancer again. Avelumab works in a similar way to cemiplimab, which is the usual drug for treatment of your…
This study is for patients with melanoma, non-small cell lung cancer (NSCLC), or squamous cell cancer of the head and neck (SCCHN) that has a protein called B7-H3 on the tumor cells or blood vessels in thetumor. The purpose of this study is to test the safety, tolerability, and most effective dose ofMGA271 in combination with ipilimumab (also called Yervoy…
The purpose of this study is to see which study drug is the better treatment for CMV infection. The study also wants to see how safe the study drugs are. The study drugs are Maribavir and Valganciclovir. Those who participate in the study will not know which drug they are taking. The study team will not know either.
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 patients with recurrent or metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN). This study will test the safety of an experimental anti-cancer drug (MEDI4736). MEDI4736 is an antibody (a protein produced by the defense system of the body immune system) that has been made in the laboratory. MEDI4736 boosts the ability of your…
In this trial, we will evaluate whether partially-HLA matched allogeneic multivirus-specific VSTs, will be safe and produce anti-viral effects in immunodeficient recipients infected with one of more of the targeted viruses that are persistent despite conventional anti-viral therapy. This will be in patients who have received a hematopoietic stem cell…
The purpose of this study is to determine the effectiveness and safety of study drug MEDI4736 monotherapy, tremelimumab monotherapy, and MEDI4736 and tremelimumab combination therapy in the treatment of patients with recurrent or metastatic PD-L1-negative squamous cell carcinoma of the head and neck who have progressed during or after treatment with…
The primary objectives of this study are to measure the safety and effectiveness of bone marrow transplant (BMT) using specific chemotherapy in children with less severe SCD and to understand the long term effects of bone marrow transplant in children with SCD, by evaluating ovarian reserve, sickle cell related brain problems, sickle cell related kidney…
The purpose of this study is to see whether or not the medicine, Abatacept, when given after hematopoietic stem cell transplantation (HSCT) is effective in treating GVHD in children and adolescents with sickle cell disease (SCD). We are also planning to gather additional information about the safety of treating sickle cell disease with a hematopoietic stem…
This is a research study of a new drug for the treatment of new onset chronic graft versus host disease (cGVHD). Chronic GVHD is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient's body. The current way to treat cGVHD is to use prednisone (a type of…
The main purpose of the study is to find out about whether the study drug is safe in humans and aneffective treatment in pediatric patients with acute Graft versus Host Disease (GVHD). GVHD mayoccur after a bone marrow or stem cell transplant in which someone receives bone marrow tissue orcells from a donor (called an allogeneic transplant). The new,…
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…
The purpose of this study is to test a new medicine to prevent liver venoocclusive disease (VOD) in patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or very high risk for liver VOD following HSCT. Liver VOD can be a complication of bone marrow and/or stem cell transplant,which may be life threatening. The condition is a…
This study is for adult subjects with head and neck cancer (oral cavity, oropharynx middle part of the throat, hypopharynx bottom part of the throat, or larynx part of throat between the base of the tongue and the windpipe) that has either returned after treatment (recurrent) or is newly diagnosed. If newly diagnosed, the cancer must be metastatic,…