10 Clinical Trials for Various Conditions
To determine the maximum tolerated dose (MTD) and toxicity of sargramostim (recombinant granulocyte-macrophage colony-stimulating factor; GM-CSF) given by continuous intravenous infusion (CIV) in patients with leukopenia in association with AIDS virus infection. In addition, single dose and steady state pharmacokinetics will also be determined.
To determine the safety, immunogenicity, biological activity, ad pharmacokinetics of sargramostim ( recombinant granulocyte-macrophage colony-stimulating factor; GM-CSF ) human granulocyte-macrophage colony-stimulating factor ( GM-CSF ), given by subcutaneous ( SC ) injection to patients with leukopenia in association with HIV infection.
To evaluate the safety of repeated courses of sargramostim ( recombinant granulocyte-macrophage colony-stimulating factor; GM-CSF ) administered subcutaneously to patients with HIV infection and leukopenia. To determine if administration of GM-CSF will prevent some or all of the hematologic toxicity associated with zidovudine ( AZT ) treatment in patients with pre-existing leukopenia. To assess any clinical and/or virologic benefits from administering alternating weeks of GM-CSF and AZT to patients with symptomatic HIV infection who have a history of cytologically confirmed Pneumocystis carinii pneumonia ( PCP ) or a circulating absolute CD4 lymphocyte count less than 200 cells/mm3.
To assess the safety and efficacy of subcutaneously administered sargramostim ( granulocyte-macrophage colony-stimulating factor; GM-CSF ) in increasing and maintaining the neutrophil count in HIV-infected adults who have developed neutropenia as a result of receiving the antiretroviral agent, zidovudine ( AZT ). To assess the safety and efficacy of subcutaneously administered GM-CSF in increasing and maintaining the neutrophil count in HIV-infected adults with pre-existing neutropenia who are at high risk of developing hematologic intolerance while receiving the antiretroviral agent, AZT, for the first time. To assess the potential therapeutic benefit of concomitant GM-CSF and AZT on the natural history of HIV infection and associated infectious complications.
This study will evaluate the feasibility of using capillary blood samples collected with the Tasso device for analysis of CBC in diseased patients with leukemia, lymphoma, and/or other blood cell disorders. This investigation will include a minimum of 40 sample sets from unique patients.
Background: * WHIMS (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis Syndrome) is caused by various genetic changes that increase the activity of the chemokine receptor, CXCR4. Excessive function of this receptor causes mature neutrophils (part of the white blood cells) to be retained within the bone marrow rather than being released to the blood and is one of the causes of severe inherited neutropenia (low white blood counts). In neutropenia, the body is less able to fight off infection. Patients with WHIMS usually are at risk for skin, soft tissue, sinus, and lung infections, which can result in loss of hearing, teeth, and lung function. * Current treatment for WHIMS consists of regular injections of a white blood cell growth stimulating medication called granulocyte colony stimulating factor (G-CSF), and supplemental immunoglobulin (antibody). These therapies are expensive, nonspecific, have significant side effects and toxicities, and do not fully correct all problems, especially warts and cancers related to human papillomavirus (HPV). * A drug called Mozobil has been approved for use in combination with G-CSF to increase the number of stem cells that can be collected prior to bone marrow transplantation. Mozobil may offer a specific and well-tolerated new treatment for WHIMS and other syndromes characterized by neutropenia. Objectives: * To evaluate whether Mozobil is safe and effective to treat neutropenia (low white blood cell count) in patients with WHIMS. * To determine an appropriate treatment dose of Mozobil, within currently approved dosage levels. Eligibility: - Individuals between 18 and 75 years of age who have been diagnosed with WHIMS and have a history of severe infections. Design: * Potential participants will undergo a screening with a medical history, physical examination, questionnaire, heart and lung function scans, and blood and urine samples. Tests will also be done for hepatitis B and C virus, and human immunodeficiency virus (HIV) that causes acquired immunodeficiency syndrome (AIDS), as well as to check neutrophil function. * Patients who are being treated with G-CSF will stop injections for 2 days before being admitted to the National Institutes of Health (NIH) Clinical Center. * Patients may participate in a Dose Escalation study and receive increasing doses of Mozobil over 5 days of treatment until their white blood cell count improves sufficiently or the maximum approved dose is reached. Blood samples will be taken regularly throughout the treatment process. Patients will then receive an additional dose of Mozobil at the maximum approved dose or the dose sufficient to cause improvement, before restarting the G-CSF injections. * Patients may also participate in a long-term Chronic Dosing study and receive Mozobil once or twice a day for up to a maximum of 60 months.
The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). The main questions the study seeks to answer include: * How many patients experience improvements in their low blood counts (red cells, platelets, or white cells) within 24 weeks, based on specific criteria for blood conditions like myelodysplastic syndromes (MDS)? * How long these improvements last before the condition worsens or changes. * The percentage of participants showing improvements at 12, 24, and 48 weeks. * How long it takes for the condition to progress to more severe diseases like myeloid disorders. * How long red blood cell responses last and how quickly these responses are seen. * The average change in hemoglobin levels over 24 weeks. * How many patients need blood transfusions during the study and how soon transfusions are required. * Changes in participants' well-being and energy levels based on a standardized questionnaire. * Monitoring for any side effects, including progression to MDS or leukemia, heart-related issues, or sudden increases in hemoglobin. Participants will: * Receive luspatercept as an injection every three weeks. * Visit the clinic every three weeks for treatment and monitoring.
In recent decades, hematologists have noticed that persons of African descent sometimes have lower white blood cell counts of a certain type, called granulocytes. These cells help to fight infections. The lower number of granulocytes in this situation does not appear to lead to more infections, and these individuals do not have any symptoms. This condition is called benign ethnic neutropenia (BEN), and is observed in a small percentage of individuals of African descent. This study will investigate the condition by studying people with and without BEN. The goals of this study are to: 1. identify individuals of African descent with BEN. 2. determine the effects of two drugs, G-CSF and dexamethasone, on granulocyte production and movement. 3. determine whether there are differences in those with and without BEN in the way genes are stimulated after the administration of G-CSF and dexamethasone. Study participants will be asked to interview with the research team, undergo physical exams, donate a blood sample, and receive G-CSF by injection, followed by dexamethasone (orally) about three weeks later. They also will be required to undergo apheresis three times, a procedure in which blood is drawn from a donor and separated into its components. Some components are retained for research analyses, such as granulocytes, and small amount of blood; the remainder is returned by transfusion to the donor. This procedure will be required of participants before they receive G-CSF, the day after they receive G-CSF, and the day after they receive dexamethasone. Gene messages (mRNA will be isolated from granulocytes, and analyzed to better understand granulocyte growth and movement.
Severe aplastic anemia, characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation. However, approximately 1/3 of patients will not show blood count improvement after ATG/CsA. General experience and small pilot studies have suggested that such patients may benefit from further immunosuppression. Furthermore, analysis of our own clinical data suggest that patients with poor blood count responses to a single course of ATG, even when transfusion-independence is achieved, have a markedly worse prognosis than patients with robust hematologic improvement. The management of such cases is uncertain. This study will enroll patients who are either refractory to h-ATG (continued severe pancytopenia) or who have only modest improvement in blood counts (weak hematologic responders) to receive a further immunosuppressive therapy, delivered either as rabbit ATG (Thymoglobulin, r-ATG) or a humanized monoclonal antibody to T-cells, alemtuzumab (Campath-1H ). Primary endpoint will be response rate at 3 months defined as no longer meeting criteria for severe aplastic anemia. Relapse, robustness of hematopoietic recovery at 3 months, survival and clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia and acute leukemia will be the secondary endpoints.
To determine the safety, tolerability and maximum tolerated dose of SDZ ILE 964 administered by daily subcutaneous injections in patients infected with human immunodeficiency virus (HIV) who have cytopenias (low blood cell counts). To obtain information about the biologic effects of SDZ ILE 964 administration in improving blood counts in HIV-infected patients. To obtain information about the effects of SDZ ILE-964 administration on both parameters of HIV replication and on residual immunologic function.