15 Clinical Trials for Various Conditions
A total of 40 subjects will be recruited for participation in this study. 20 subjects (10 males and 10 females) will be randomized to the active group (those receiving re-infusion of autologous blood) and 20 subjects (10 males and 10 females) will be randomized to the placebo group (receiving NS infusion).
The objective of this study is to assess effects of the storage of PRBC on endothelial function, inflammation and platelet activation in healthy volunteers
The purpose of this study is to determine whether storage time affects how human body responds to autologous blood transfusion. An autologous blood transfusion is when a person donates blood and then receives that same blood back in the transfusion. We also want to find out if in this situation inhaled nitric oxide can help to prevent the potential reduction of vasodilation capacity. Vasodilation capacity is the ability of the blood vessel to widen when needed.
The Purpose of the study is to test the hypothesis that administration of an S-nitrosylating (SNO) agent can improve tissue oxygenation during transfusion of packed red blood cells (RBCs).
The purpose of this study is to test the safety and effectiveness of a cord blood infusion in children who have motor disability due to cerebral palsy (CP). The subjects will be children whose parents have saved their infant's cord blood, who have non-progressive motor disability, and whose parents intend to have a cord blood infusion.
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections. Researchers want to see if a new procedure can help a person s cells kill germs for a short time. It uses messenger RNA (mRNA) to deliver correct instructions for the gene mutation to the cells. Objective: To test a procedure in which mRNA is added to a person s blood cells. Eligibility: Males aged 18-75 with CGD with a mutation in the gene that makes the protein gp91phox. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Swab to test for strep throat Some screening tests will be repeated during the study. Participants will be admitted to the NIH Clinical Center hospital for at least 7 days. They will have apheresis. For this, a medicine is injected under their skin to prepare their white blood cells for collection. An IV line is placed into an arm vein. Blood goes through the IV line into a machine that divides whole blood into red blood cells, plasma, and white blood cells. The white blood cells are removed, and the rest of the blood is returned to the participant through an IV line in their other arm. The next day, they will get their mRNA-corrected cells via IV. They will be monitored for 3 more days. After discharge, participants will keep a symptom diary. They will be contacted weekly for one month, and then once a month. They will have a follow-up visit 3 months after the infusion.
The purpose of the study is to determine whether transfusion is associated with mild pulmonary changes.
Autologous blood transfused at the end of cardiopulmonary bypass will reduce total blood loss 24 hours after surgery and improve mitochondrial oxygen delivery measured by plasma succinate levels. The study design is a prospective randomized interventional trial of transfusion of fresh autologous whole blood versus standard of care expectant management of bleeding during elective cardiac surgery.
Objectives: * Determine the corrected count increment of autologous transfused platelets that had been stored by cryopreservation with ThromboSol. * Determine the ability of autologous platelets that had been stored by cryopreservation with ThromboSol to correct thrombocytopenia.
This protocol represents a continuation of a series of prospective studies to define the incidence and etiology of transfusion-associated hepatitis (TAH) and to examine the impact on TAH of various modifications in the selection of blood donors. The primary goal of the study will be to determine TAH incidence after the institution of a variety of interventive measures to exclude hepatitis and AIDS virus carriers: including surrogate assays (ALT, anti-HBc), a specific assay for the hepatitis C virus (HCV), a specific assay for the human immunodeficiency virus (HIV) and intensified donor questioning for high-risk behavior patterns. There is high probability that the exclusion of donors at high risk for AIDS transmission will also exclude donors at high risk for hepatitis transmission. Incidence data obtained in the study will be enhanced by the simultaneous follow-up of a control population undergoing identical surgical procedures, but receiving no blood or only autologous blood. This control population, made possible by the recent dramatic increase in the amount of autologous blood utilized, will allow for a clear distinction between transfusion-associated hepatitis and that due to nosocomial transmission or other background causes of hepatocellular inflammation in cardiac surgery patients.
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
The overall aim is to reduce overall allogeneic transfusion requirements during cardiac surgery when compared to standard management. To evaluate this the investigators will test the hypothesis that intraoperative, autologous platelet apheresis will primarily avoid allogeneic platelet transfusion. Following induction of anesthesia and intravascular line insertion, the patient will be randomized to control or treatment arms by sealed envelope technique where computer generated, randomization numbers are assigned prior to enrollment based on study patient number which is never reused. The control arm will have central venous access "sham" connected to the apheresis machine Trima® (Terumo BCT, Denver CO); the treatment arm will be connected and undergo pheresis. The clinical team will be blinded by a sterile sheet acting as a curtain and a recorded playback of the typical sounds of the operation of the apheresis machine. At the end of the pheresis, the platelet units will be disguised with opaque coverings and agitated at room temperature in compliance with the American Association of Blood Banks (AABB) recommendations for platelet storage. On separation from CPB, the blinded administration of autologous platelets or allogeneic (blood bank) platelets will occur after protamine administration, if the surgeon requests platelet transfusion (this is typically the case for these operations). The surgeon will be blinded and he will order subsequent transfusions based on clinical evidence of microvascular bleeding in accordance with standard guidelines, as is the investigators practice for these operations.
A single-site, single-blind study of ascending doses of Lyophilized Plasma in normal healthy volunteers.
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.