93 Clinical Trials for Various Conditions
This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE * To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES * To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). * To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts * To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects
Objective: To collect information on how often a solid tumor cancer might lose the Human Leukocyte Antigen (HLA) by next generation sequencing and perform apheresis to collect and store an eligible participant's own T cells for future use to make CAR T-Cell therapy for their disease treatment. Design: This is a non-interventional, observational study to evaluate participants with solid tumors with a high risk of relapse for incurable disease. No interventional therapy will be administered on this study. Some of the information regarding the participant's tumor analysis may be beneficial to management of their disease. Participants that meet all criteria may be enrolled and leukapheresed (blood cells collected). The participant's cells will be processed and stored for potential manufacture of CAR T-cell therapy upon relapse of their cancer.
Purpose: To describe a novel configuration of venous access for the performance of intermittent apheresis. Participants: 20 participants at UNC who were referred for change from a vortex port to a powerflow port. Procedures (methods): Placement of one of two configurations of the powerflow port and follow up visits between January 1, 2019 and December 31, 2023.
This is a prospective, multi-center, controlled, randomized, non-inferiority study to evaluate the clinical effectiveness of Conventional versus Mirasol-treated apheresis platelets in subjects with hypoproliferative thrombocytopenia who are expected to have platelet count(s) ≤ 10,000/μL requiring ≥ 2 platelet transfusions.
This is a platelet transfusion study. The purpose of this study is to measure the life span and quality of platelets stored in a refrigerator. Participants will give platelets by apheresis. Platelets will be stored for 3 -20 days. A small portion of the subject's own stored platelets will be tagged with a radioactive isotope and infused back into the participant. This will enable us to track how many transfused platelets survive after storage in the refrigerator.
A longitudinal, randomized, controlled, single-center Phase IV clinical trial will be performed to assess changes in bone mineral density (BMD) among voluntary apheresis blood donors. The primary outcome measure will be clinically significant decline in BMD at the lumbar spine assessed by dual-energy x-ray absorptiometry (DXA).
This study will collect blood plasma and white blood cells from individuals using a procedure called apheresis. Apheresis is a method of collecting larger quantities of certain blood components than can safely be collected through a simple blood draw or blood donation process. The blood components will be used in laboratory research studies to investigate aspects of infectious and immunologic allergic diseases. Patients 7 years of age and older who are currently enrolled in a NIH clinical research protocol may participate in this study. (Children between the ages of 2 and 6 may participate if they will benefit clinically from undergoing apheresis.) Family members of patients and normal healthy volunteers will also be enrolled. * For all adults and children weighing 55 pounds or more. Blood is drawn through a needle placed in an arm vein and circulated through a cell separator machine. The plasma and white cells are extracted, and the red cells are returned to the donor through a needle in the other arm. The procedure takes from 1 to 2 hours. * For children weighing less than 55 pounds. One unit (1 pint) of blood is drawn through a needle placed in an arm vein, similar to donating a pint of whole blood. The red blood cells are separated from the rest of the blood and returned to the donor through the same needle. This procedure requires only one needlestick and takes about 30 to 45 minutes to complete. In some circumstances, the procedure must be repeated one or more times in order to obtain large enough quantities of plasma or cells for study.
The primary objective of the study is to demonstrate that the performance of the modified Plasmat® Futura H.E.L.P. Apheresis System is non-inferior to the current FDA approved Plasmat® Secura H.E.L.P Apheresis System for use under the approved indication of the acute reduction of LDL-cholesterol from the plasma in populations for whom diet has been ineffective and maximum drug therapy has either been ineffective or not tolerated.
This study will collect blood plasma and white blood cells from individuals using a procedure called apheresis. Apheresis is a method of collecting larger quantities of certain blood components that can safely be collected through a simple blood draw. The blood components will be used in laboratory research studies. Patients 18 years of age and older who are currently enrolled in a NIH clinical research protocol may participate in this study. Relatives of patients and normal healthy volunteers will also be enrolled. Individuals will undergo one of the following two apheresis procedures: * Automated pheresis Blood is drawn through a needle placed in an arm vein and circulated through a cell separator machine. The plasma (liquid part of the blood) and white cells are extracted, and the red cells are re-infused into the donor through the same needle or a needle in the other arm. An anticoagulant (medication to prevent blood from clotting) is usually added to the blood while in the machine to prevent it from clotting during processing. * Manual pheresis One unit (1 pint) of blood is drawn through a needle placed in an arm vein, similar to donating a pint of whole blood. The red blood cells, with or without plasma, are separated from the rest of the blood and returned to the donor through the same needle. Manual pheresis will be done only when a person s estimated total blood volume or red cell count is too low to safely permit removal of blood through a pheresis machine. An adult small in size or markedly anemic, for example, may fall into this category. Some of the blood collected through apheresis may be stored for future studies of HIV disease and immune function and for HLA testing, a genetic test of markers of the immune system. Some of the blood may be used to screen for different types of viral liver infections, such as hepatitis A, B, C, D, E, F, or G.
The objective of this study is to assess the post-infusion recovery and survival of platelets in 100% Plasma treated with INTERCEPT Blood System for Platelets with LED Illuminator and stored for 5 days after apheresis collection. The post-infusion recovery and survival of autologous radiolabeled 5-day INTERCEPT platelets (Test) stored in 100% plasma will be measured in comparison to fresh autologous radiolabeled platelets (Control).
This study includes extended CD34+ profiling on the apheresis product of multiple myeloma patients undergoing standard-of-care quad-induction followed by motixafortide + G-CSF mobilization, and in addition, assesses the pharmacodynamics (PD) of motixafortide following "standard" (\~12 hours) vs "early" (\~16 hours) dosing. The investigators hypothesize that quad-induction may alter the stem cell subsets within the mobilized graft. The investigators further hypothesize that standard and early dosing strategies will result in comparable mobilization and stem cell collection rates.
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
This is phase 1 single cohort study evaluating the effects of N-803 administration on mononuclear cells (MNC) collected from healthy donors undergoing a procedure called apheresis.
This trial is being performed to evaluate the feasibility of the study protocol and to test the efficacy and safety of platelets stored at cold conditions (1-6°C) in 100% plasma for 10-14 days (CSP) in cardiac surgery patients who are actively bleeding and require platelet transfusion.
The study is a prospective, single-arm, non-randomized, non-blinded, non-controlled, non-significant risk, single center study enrolling up to 200 healthy adult subjects consented to undergo an apheresis donation procedure. Subjects will be connected to the Zynex Cardiac Monitor, Model 1500 (CM-1500) to characterize changes in the relative index during an apheresis donation procedure.
This is a randomized pilot study of therapeutic apheresis procedures (TAP) using the current standard of care catheter (SOC) vs the BD Nexiva Diffusics Catheter. It is a single blinded, randomized study. Data will be used to refine and power a full randomized control trial. For this study, a sample of 33 encounters in each group (total of 66 encounters). The specific aim is to test the hypothesis that the 20-gauge BD Nexiva Diffusics Catheter provides the same efficacy and lower pain level with no increase in adverse events for patients undergoing apheresis treatments.
Focal segmental glomerulosclerosis (FSGS) is the most common cause of end-stage renal disease (ESRD) in adolescents. The refractory nature of FSGS and a more than 30% recurrence rate after kidney transplantation renders treatment of FSGS one of the most difficult challenges in pediatric nephrology. A significant knowledge gap in understanding the mechanism of FSGS treatment resistance and progression hampers development of successful treatment strategies. Beneficial effect of removal of low-density lipoproteins by LDL-apheresis indicates that lipids contribute to progression in FSGS. The investigators will test the hypothesis that removal of Lp-PLA2 and lipid metabolites by LDL-apheresis ameliorates proteinuria and cardiovascular comorbidities. Patients with FSGS and FSGS recurrence after kidney transplantation receiving LDL-apheresis as part of standard of care will be enrolled to the study. Pre-and post serum and effluent concentrations of LPC, free FA, Lp-PLA2, oxidized LDL, fasting lipid profile, interleukin (IL)-6, tumor necrosis factor (TNF)-α, and IL-1β will be monitored in patients undergoing LDL-apheresis. Investigators will also study the impact of LDL-apheresis on cardiovascular and clinical comorbidities by monitoring degree of proteinuria, blood pressures and arterial stiffness index.
The principle objective of this study is to evaluate the hypothesis that INTERCEPT Platelets in 100% plasma stored for 5 or more days (up to 7 days) after apheresis collection retain sufficient viability for therapeutic transfusion efficacy. The post-infusion recovery and survival of autologous radiolabeled 7 day INTERCEPT platelets (Test) stored in 100% plasma will be measured in comparison to "fresh" autologous radiolabeled platelets (Control) according to FDA guidance for platelet testing (FDA 1999) in Stage 2 of this study protocol. A secondary objective is to compare the recovery and survival results for Test platelets prepared for radiolabeling using the procedures outlined by the Biomedical Excellence for Safer Transfusion Collaboration (BEST) or a variation of the BEST procedure (referred to as Variant 1) in Stage 1 of this study protocol. Cerus has demonstrated that the Variant 1 method, which does not incorporate an initial soft spin in the presence of ACD A, results in improved in vitro platelet recovery and quality during preparation for radiolabeling compared to the BEST procedure. This comparison will evaluate the hypothesis that preparation methods prior to radiolabeling may influence in vitro quality of the radiolabeled platelets and post-infusion viability outcomes.
To evaluate the efficacy of subcutaneous (SC) evolocumab, compared to regularly scheduled low-density lipoprotein cholesterol (LDL-C) apheresis, on reducing the need for future apheresis.
Severe hypercholesterolemia produced by conditions such as heterozygous familial hypercholesterolemia is associated with multiple complications including premature atherosclerotic disease. There is evidence that microvascular perfusion, particularly flow reserve, in critical organs is limited due to abnormalities in plasma viscosity, abnormal RBC deformability, and an imbalance between vasodilators and vasoconstrictors. There is little is currently known about acute changes in microvascular blood flow and microvascular rheology that occur in response to plasmapharesis which is used in some patients to lower critically elevated cholesterol levels. Our research group has pioneered CEU methods for assessing myocardial and skeletal muscle perfusion, and has previously demonstrated in pre-clinical models that acute hyperlipidemia produces a reduction in microvascular RBC transit rate. In this study, the investigators will assess acute changes in microvascular perfusion in patients undergoing clinically-indicated plasmapharesis.
The primary objective of the study is to evaluate the effect of alirocumab 150 mg every 2 weeks (Q2W) in comparison with placebo on the frequency of low-density lipoprotein (LDL) apheresis treatments in participants with heterozygous familial hypercholesterolemia (HeFH) undergoing weekly or bi-weekly LDL apheresis therapy.
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.
The purpose of this protocol is to characterize the performance of CaridianBCT's Spectra Optia Apheresis System, when used to collect mononuclear cells (MNCs) and cluster of differentiation 34 (CD34) positive cells from healthy nonmobilized blood donors and healthy G-CSF (granulocyte colony stimulating factor) mobilized blood donors, respectively.
Background: - Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection. Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease. Researchers are interested in collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the cells to develop potential gene therapy treatments. Objectives: - To collect blood stem cells from patients with immunodeficiency diseases tto test our ability to correct the defects of these cells in the test tube. Eligibility: * Individuals between 18 and 40 years of age with immunodeficiency diseases. * Individuals with human immunodeficiency virus (HIV) will not be able to participate in this study. Design: * Participants will provide an initial blood sample for disease screening (such as hepatitis B and C, syphilis, or viruses like the Epstein-Barr virus, herpes simplex virus, or toxoplasmosis) and to check kidney and liver function. * Starting 5 days before blood donation, participants will receive daily injections of a drug called G-CSF (granulocyte colony stimulating factor, or filgrastim), which pushes stem cells out of the bone marrow and into the bloodstream. Participants will receive the injections at the National Institutes of Health Clinical Center. * On day 5, participants will have a single leukapheresis procedure to collect the stem cells from the blood. * No additional treatment will be provided as part of this protocol. The cells that are collected will be used fore experiments in the lab and will not be used to treat individuals with these diseases.
The primary objective of this study is to measure how LDL apheresis affects levels of inflammatory and cholesterol markers in human beings. The investigators will address this question by drawing pre- and post-LDL apheresis blood from patients who are undergoing this procedure. A secondary objective of this study is to learn how specific inflammatory markers behave in our blood in terms of time to rebound back to normal levels. The investigators will address this question by drawing post-LDL apheresis blood at predetermined time intervals.
The purpose of the study is to evaluate the safety and efficacy of dosing with mipomersen for 26 weeks in treating severely hypercholesterolemic patients who are on a maximally tolerated lipid-lowering regimen and who are not on apheresis.
This study will examine the effects of repeated apheresis procedures on bone density and calcium balance. Apheresis is a procedure for collecting large numbers of a specific blood component, such as white cells (leukapheresis) or platelets (plateletpheresis). For the procedure, whole blood is collected through a needle in an arm vein and is directed through a machine that separates it into its components by spinning. The desired cells are removed and the rest of the blood is returned to the donor, either through the same needle or through a needle in the other arm. A blood thinning medicine called citrate is added to the cell-separating machine. Citrate reduces the ionized calcium levels in the blood, which prevents the blood from clotting. When the blood is returned to the donor, the donor also receives the citrate. This lowers the donor's ionized calcium levels which may irritate nerve and muscle cells, causing tingling around the mouth, hands, and feet during the procedure. The reduced ionized calcium levels result in increased parathyroid hormone levels in the donor, can effect bone calcium stores. In addition, some of the citrate that is returned to the donor is excreted in the urine along with calcium, which causes further loss of calcium from the body. It is not known if the calcium loss during apheresis in people who undergo this procedure repeatedly has any long-term effects on body calcium balance and bone calcium stores. This study will measure bone density and calcium balance in long-term platelet and white cell donors and compare the findings with those of whole blood donors, who do not receive citrate. Healthy people between 18 and 80 years of age who weigh between 110 and 300 pounds, do not have a metal prosthesis, and are not pregnant may be eligible for this study. Participants undergo the following procedures: Whole blood donors * Blood sample collection 2 weeks before blood donation. * I removed undergo standard whole blood donation * Urine sample collection. * DEXA scan to assess bone density by measuring bone calcium stores. For this procedure, the subject lies still on a table while the spine, hip, and whole body are scanned using a small amount of radiation. The forearm is also scanned while the subject is seated. The scan may be repeated after 2 years. Plateletpheresis and leukapheresis donors * Standard platelet or white cell donation. * Blood sample collections immediately prior to and after donation, and on the first, fourth, and fourteenth days after donation. * Urine sample collections at the beginning and at the end of the apheresis procedure and on the first, fourth, and fourteenth days after the donation. * DEXA scan at the beginning of the study (no earlier than 2 weeks after their latest apheresis donation). The scan may be repeated after 2 years. * Some apheresis donors may be asked to have a second procedure in which they take calcium according to standard guidelines for plateletpheresis and leukapheresis. During the second procedure, platelet donors will take oral calcium tablets before starting plateletpheresis. White cell donors will receive calcium intravenously (through a vein) during the second leukapheresis. For this second procedure, the donors provide additional blood and urine samples as described above.
This study collects specimens from volunteers for use in studies by NIAID's Vaccine Research Center. A number of different types of specimens or samples can be collected, including blood, urine, body fluids or secretions, skin swabs, or skin biopsies. The samples are used for medical research, including the study of HIV, hepatitis, and other diseases; immune system responses, such as responses to vaccinations or infections; and for research on vaccine development. Blood samples may be collected either by ordinary blood drawing (phlebotomy) or by apheresis, a procedure for collecting a larger quantity of blood cells or plasma than would be possible through simple blood drawing. For this procedure, the participant lies on a recliner or couch. Blood is removed through a needle in the vein of one arm and spun in a machine that separates out the desired component (plasma or white blood cells). The remainder of the blood is returned either through the same needle or through a needle in the other arm. The procedure takes about 1 to 3 hours. Volunteers who are 18 years of age and older, including participants in other NIH research protocols, may be eligible. Individuals who have a condition that the research staff considers a reason not to make a sample donation will be excluded from the study. Participants may have only one sample collected or may be asked to undergo repetitive sample collection procedures, depending upon the requirements of the particular research project for which the samples are being collected. Each individual's enrollment is for a 1-year period, which can be extended.
This study will determine whether magnesium replacement during apheresis can decrease side effects that donors commonly experience. Apheresis is a method of collecting large numbers of certain blood cells, such as white cells, stem cells, or platelets. In this procedure, whole blood is collected through a needle in an arm vein, similar to donating blood. The blood is separated into its components by centrifugation (spinning), the required cells are extracted, and the rest of the blood is returned to the body, either through the same needle or through another needle in the other arm. When healthy people donate cells by apheresis, a blood thinner called citrate is added to prevent the blood from clotting in the apheresis machine. Citrate works by reducing calcium in the blood. When the blood is returned to the donor, citrate from the machine is also returned, lowering the donor's calcium levels. As a result, donors often feel tingling around the mouth, hands, and feet. Some of these symptoms can be prevented by giving calcium intravenously (through a vein) during the procedure. Even with the added calcium, however, some donors still have symptoms. Magnesium levels are also lowered by citrate, but it is not known if this causes symptoms. This study will examine whether the decrease in magnesium levels also contributes to the side effects of apheresis and whether magnesium replacement can reduce these symptoms. Healthy apheresis donors 18 years of age or older who are enrolled in NIH protocols may participate in this study. Donors will undergo the apheresis procedures required by the NIH protocol they are enrolled in. Throughout the procedure, they will receive an intravenous infusion of a salt solution that may or may not contain magnesium. Blood samples of 5 milliliters (1 teaspoon) each will be taken from the apheresis machine at the beginning and end of the procedure and at 30- to 60-minute intervals during the procedure. No more than 50 ml (3 tablespoons) will be taken during any single apheresis. The last sample will be drawn 60 minutes after completion of the apheresis. In addition, donors will: * Provide a urine sample at the beginning and end of each apheresis procedure. * Donate an additional urine sample and an additional 5 ml blood sample the morning after apheresis * Describe any symptoms experienced during apheresis to the apheresis nurse
The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that will be used in the laboratory and/or in the clinic to develop new cell therapies for patients with inherited or acquired disorders of immunity or blood cells. Development of novel cellular therapies requires access to HSC, Mononuclear Cells and/or granulocytes as the essential starting materials for the pre-clinical laboratory development of gene therapies and other engineered cell products. HSC or blood cells from healthy adult volunteers serve both as necessary experimental controls and also as surrogates for patient cells for clinical scale-up development. HSC or blood cells from patients serve both as the necessary experimental substrate for novel gene therapy and cellular engineering development for specific disorders and as pre-clinical scale up of cellular therapies. Collection of cells from adult patients collected in the NIH Department of Transfusion Medicine (DTM) under conditions conforming to accepted blood banking clinical practice may also be used directly in or cryopreserved for future use in other NIH protocols that have all required regulatory approvals allowing such use. In summary, the research goal of this protocol is the collection of HSC or blood cells that may be used for both laboratory research and/or for clinical treatment in other approved protocols.