The purpose of this study is to study the safety and feasibility of giving intestinal transplant patients CD34+ stemcells (the cells that make all the types of blood cells) obtained from their organ donor's bone marrow as an IV…
The purpose of this study is to see whether or not the medicine, Abatacept, when given after hematopoietic stemcell transplantation (HSCT) is effective in treating GVHD in children and adolescents with sickle cell disease (SCD). We are also planning to gather…
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…
The purpose of this study is to test a new medicine to prevent liver venoocclusive disease (VOD) in patients undergoing hematopoietic stemcell 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…
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 stemcell transplant in which someone receives bone marrow tissue or…
Are you undergoing a stemcell 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 stemcell…
This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stemcells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent…
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…
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…
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…
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stemcell transplant from a family member or unrelated adult donor for a non-malignant condition.