View clinical trials related to Immunologic Deficiency Syndromes.
Filter by:- Background/Rationale: Epidemiological data about Primary Immunodeficiency Disorders (PIDD) in Kuwait is needed to better understand peculiarities and to compare them with other regions and ethnicity. - Study hypothesis: PIDD is relatively common in Kuwait compared to populations from different geographic areas. The distribution of PIDD in Kuwait is different from other geographic areas with more severe forms being more frequent. - Brief inclusion and exclusion criteria of study participants: PIDD patients presented at different clinics/hospital in Kuwait. Patients with secondary immunodeficiencies (drug induced, virus induced, and immunodeficiency associated with metabolic disorders... ect), will be excluded - Estimated sample size of the study: All patients who were registered in KNPIDR since 2004 will be included in the study along with the new patients who will be recruited during the study period. - Primary objectives: - Determine the prevalence and frequency of different PIDD in Kuwait - Identify clinical presentation patterns for PIDD in Kuwait - Identify natural history of PIDD in Kuwait - Help to asses epidemiology of PIDD in Kuwait - Determine particularities about PIDD affecting the population in Kuwait - Determine the health impact of PIDD in Kuwait - Development of strategies to improve the care and the quality of life of patients with PIDD
Oral pre-exposure prophylaxis (PrEP) has been proven effective in reducing HIV infection in high-risk men who have sex with men, heterosexually active women and men, and injecting drug users. Despite its 2012 approval by the FDA and the development of Centers for Disease Control and Prevention (CDC) clinical guidelines, PrEP uptake has been limited. Significant impediments to PrEP implementation include: system barriers (lack of a medical "home" and of models for implementing PrEP); provider barriers (difficulty identifying those likely to benefit from PrEP, inexperience with PrEP, and concerns about adherence and risk compensation); and user barriers (lack of awareness of PrEP, inability to access providers comfortable with prescribing PrEP, and concerns about stigma and side effects). Cost is not a barrier in New York State, where PrEP is covered by many insurance plans, including Medicaid. Primary Care for PrEP (PC4PrEP) is a structural, multilevel intervention that will integrate PrEP into primary care practices that care for underserved communities in the Bronx, NY, an epicenter of continuing HIV infection in the US. PC4PrEP will develop an organizational protocol for prescribing PrEP in primary care; identify high-risk individuals in primary care clinics and community HIV testing sites using a new PrEP Eligibility Tool; link them to primary care providers (PCPs) who can provide PrEP; and counsel potential users about PrEP both before they receive a prescription (to enhance receptivity), and after they initiate PrEP (to enhance adherence). In the course of this study, investigator(s) will (1) develop and pilot PC4PrEP; (2) implement and evaluate it in "real-world" settings (Federally Qualified Health Centers; FQHCs) on objective outcomes as well as provider and patient reports; and (3) present a new model, the PrEP Cascade that - as with the HIV Care Cascade for HIV+ populations - may be used to evaluate the impact of PrEP programs in the US and other countries. PC4PrEP is consistent with CDC and New York State Department of Health (NYSDOH) Guidelines and the Affordable Care Act in integrating PrEP into primary care practices and is responsive to recent 2014 NYSDOH recommendations which now position PrEP as a first-line intervention for MSM and transgender women who engage in ongoing anal sex without condoms, HIV- partners in sero-discordant relationships, and high-risk heterosexual women in high seroprevalence areas.There are two Specific Aims: (1) Finalize the PC4PrEP intervention and, in a clinic-randomized Phase 2 futility trial, assess whether it shows promise for increasing PrEP prescription rates in the Bronx, NY; and (2) Identify strengths and limitations of PC4PrEP in two ways: (a) through a mixed-methods process evaluation PrEP-eligible patients and PCPs, counselors and navigators; and (b) by identifying "fall-off" at each step of the PrEP Cascade.
This study will evaluate the anti-retroviral activity of MK-8527 in HIV-1 infected, ART-naïve participants. The primary hypothesis is that MK-8527 has superior anti-retroviral activity compared to placebo, as measured by change from baseline in plasma HIV-1 ribonucleic acid (RNA) at 168 hours postdose.
Background: The immune system helps the body fight infections. Primary immunodeficiency disorders (PIDs) are diseases that make it easier for people to get sick. Many PIDs are inherited. This means parents can pass them on to their children. Knowing what causes a person s PID is important to decide what treatment to give them. Objective: To test samples from people with a PID or people related to someone with a PID to find out what causes PIDs. Eligibility: People ages 99 or younger who have a PID or have a relative with a PID Design: Participants will be screened with a medical history over the phone. They may need to give permission for researchers talk to their doctors about their health. Their relatives may be contacted to see if they want to join the study. Participants will give samples. These could be: Blood: Participants blood will be taken from a vein in an arm, or with a prick on the finger or heel for children. Saliva, urine, or stool: Participants will provide each sample in a special cup. Nose or cheek swab: Participants will rub the skin inside their nose or cheek using a cotton swab. Cord blood: If participants have a baby during the study, blood will be collected from the baby s umbilical cord after it is born. Samples from medical procedures: If, during the study, the participants have a medical procedure that collects samples, the samples may be used for the study.
Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene phosphoramide (TEPA). The goal of this study is to determine what causes some children to have different drug concentrations of thiotepa and TEPA in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and genetic factors cause changes in thiotepa and TEPA drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.
This phase II clinical trial studies the side effects of pomalidomide and how well it works in treating patients with Kaposi sarcoma and human immunodeficiency virus (HIV) infection. Biological therapies, such as pomalidomide, may stimulate the immune system in different ways and stop tumor cells from growing and it may also block the growth of new blood vessels necessary for tumor growth.
Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. SCID arises from a variety of molecular defects which affect lymphocyte development and function. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. SCID-X1 is caused by defects in the common cytokine receptor gamma chain, which was originally identified as a component of the high affinity interleukin-2 receptor (IL2RG). Allogeneic haematopoietic stem cell transplantation (HSCT), which replaces the patient's bone marrow with that of a healthy donor, is the only treatment that definitively restores the normal function of the bone marrow. HSCT is the first choice of treatment for patients with signs of bone marrow failure and a fully-matched related donor. However, patients without a fully-matched related donor have much worse overall outcomes from HSCT. This study will investigate whether patients with SCID-X1 without a fully matched related donor may benefit from gene therapy. To do this the investigators propose to perform a phase I/II clinical trial to evaluate the safety and efficacy (effect) of gene therapy for SCID-X1 patients using a lentivirus delivery system containing the IL2RG gene. Up to 5 eligible SCID-X1 patients will undergo mobilisation and harvest of their haematopoietic stem precursor cells (HPSCs). In the laboratory the disabled lentivirus will be used to insert a normal human IL2RG gene into the patient's harvested HPSCs. Patients will receive chemotherapy conditioning prior to cell infusion, in order to enhance grafting. The genetically corrected stem cells will then be re-infused into the patient. Patients will be followed up for 2 years. This trial will determine whether gene therapy for SCID-X1 using a lentiviral vector is safe, feasible and effective
Infants with severe combined immunodeficiency (SCID) have a profound decrease in number and function of immune cells, and therefore remain highly vulnerable to infection. If not corrected this often leads to death. Hematopoietic cell transplantation (HCT) from matched sibling donor is the standard treatment for these patients, unfortunately though; most SCID patients lack a sibling donor. Building upon experience and existing data, the investigators are proposing a trial the goals of which are: to provide a conditioning regimen that is well tolerated, and provision of immune cells that altogether should establish rapid immune recovery providing protection from life threatening infections without increasing the risk of dangerous Graft-Versus-Host-Disease. Primary Objectives 1. To evaluate the safety of a TCRα/β/CD19-depleted graft with CD45RA-depleted DLI in infants with SCID 2. To estimate overall survival at 1 year post transplantation Exploratory Objectives 1. To evaluate the significant donor T cell reconstitution of a TCRα/β/CD19 depleted graft with CD45RA-depleted DLI at 1 year (+/-2 weeks). 2. To evaluate engraftment at day 30, 100, month 6, and years 1 to 10 post HCT. 3. To evaluate B cell reconstitution at years 1 to 10 post HCT. 4. To evaluate biomarkers of immune reconstitution at day 30, 60 100, month 6 and years 1 to 10; e.g. immunophenotype (including epigenetic profiling) of T, B, and NK cells, and assays to determine their function. 5. To evaluate clinical outcomes, post HCT. 6. To define the incidence and severity of acute (at day 100, month 6), and chronic (month 6, 12, 24) GVHD following HCT.
STRIDE2 is a longitudinal, non-randomized study of individuals living with HIV who are dependent on opioids. This study is funded by the National Institute on Drug Abuse (R01DA030768, Altice, PI; Taxman & Lawson, Co-PIs) and is being conducted by George Mason University, Yale University, and Howard University.
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the differential diagnostic work-up, and document the clinical course under various treatments. Time points: at inclusion, after 6 months, after 12 months, then yearly up to 4 years after inclusion. No intervention, mere observation and documentation. Guided pre-inclusion (differential) diagnostic work-up.