View clinical trials related to Common Variable Immunodeficiency.
Filter by:This is an observational, case--control study with a single blood draw among two cohorts, patients with CVID and healthy controls. Samples will be analyzed by cytometry (CyTOF) to simultaneously examine the major signaling pathways of all circulating innate and adaptive immune cell types as well as whole exome sequencing. The goal is to improve our general understanding of the human immune response to infections and the diagnosis of CVID.
The study will investigate the efficacy and safety of subcutaneous interferon alpha -2a to eradicate rhinovirus in patients with primary hypogammaglobulinemia. Patients with hypogammaglobulinemia have persistent rhinovirus infections. Rhinovirus may worsen pulmonary complications. Pegylated interferon alpha with ribavirin appear to effectively clear persistent rhinovirus infections in hypogammaglobulinemia patients. Patients with primary hypogammaglobulinemia and confirmed respiratory rhinovirus infection will be randomly assigned in a double-blind fashion to receive either - Group 1: subcutaneous pIFNα2a - Group 2: subcutaneous placebo Subjects will have scheduled study visits at 1-week and at 2-month after entry to study. In addition, possible bacterial infections will be treated with antibiotics. Each patient will be followed with weekly nasal surveillance samples for 2 months and a symptom diary. Blood draws take place at study entry, 1-week and 2-month time-points.
Background: Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies. Eligibility: Donors: Healthy people ages 4 or older Recipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant Design: Participants will be screened with medical history, physical exam, and blood tests. Participants will have urine tests, EKG, and chest x-ray. Donors will have: Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone. OR Blood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm. Possible vein assessment or pre-anesthesia evaluation Recipients will have: Lung test, heart tests, radiology scans, CT scans, and dental exam Possible tissue biopsies or lumbar puncture Bone marrow and a small piece of bone removed by needle in the hipbone. Chemotherapy 1-2 weeks before transplant day Donor stem cell donation through a catheter put into a vein in the chest or neck Several-week hospital stay. They will take medications and may need blood transfusions and additional procedures. After discharge, recipients will: Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission. Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.
This research is being done to construct a survey questionnaire, a quality of life tool, to determine the health, well-being, ability to perform daily activities, and physical, social and emotional functioning in participants with primary immunodeficiency disorders (PIDD). This quality of life (QOL) tool will help physicians understand the effects of PIDD on a person's health. These quality of life surveys are important tools for physicians to measure outcomes for satisfaction and effects of treatment.
This study was designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency (APDS/PASLI). The study consisted of two parts: Part I was the open label part designed to establish the safety and pharmacokinetics of CDZ173 in the target population, as well as to select the optimal dose to be tested in Part II. Part II was designed to assess efficacy and safety of CDZ173 in the target population.
The primary objective was to determine the efficacy of Human Normal Immunoglobulin (Subgam®) given subcutaneously by weekly infusion to patients with primary antibody deficiency. The secondary objective was to determine the safety of Subgam® given subcutaneously by weekly infusion to patients with primary antibody deficiency.
Background: - Some people with Common Variable Immunodeficiency Disease (CVID) have gastrointestinal inflammation. This can cause diarrhea, weight loss, and not being able to absorb nutrition from food. Researchers want to see if the drug ustekinumab can help these problems. This drug blocks some proteins that cause inflammation. Objective: - To test the safety and efficacy of the drug ustekinumab for people with CVID with gastrointestinal inflammation. Eligibility: - Adults ages 18-75 with CVID. They must have chronic diarrhea, have unintentionally lost weight in the last year, and/or need to use nutritional supplements to maintain their weight. Design: Participants will undergo the following screening studies to make sure that this study is a good fit for your medical situation, and to make sure it is safe for you to receive the study medications tests, including tests for HIV and hepatitis . This will be done as an inpatient at the NIH Clinical Center and takes about 5-6 days: - Participants will be screened with: - Medical history - Physical exam - Blood tests, including tests for HIV and hepatitis. - Stool tests, including a timed 48 hour collection for fat malabsorption and a 24 hour collection for protein malabsorption - Urine tests, including a pregnancy test for any women with the ability to have a child - Chest CT scan to look for infection - D-xylose testing, which involves drinking a sugary solution and then having a blood sample drawn to test carbohydrate (sugar) malabsorption - Hydrogen breath testing for test for small intestinal bacterial overgrowth (SIBO) this test also involves drinking a sugary solution and then collecting breath samples - Upper endoscopy (EGD) and/or colonoscopy to look at the lining of the GI tract and take biopsies for testing. This will be done under sedation by a qualified gastroenterologist. Participants who complete screening and meet all criteria will then return to the NIH Clinical Center for the following visits: - First Treatment Visit (1 clinic day): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. A pregnancy test for women of childbearing potential. A nurse will give you three shots of 90 mg ustekinumab (270 mg total dose) by very small needles injected under the skin, and then observe you for 1 hour. - Week 8 Treatment Visit (1 clinic day): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. - Week 16 Treatment Visit (1 clinic day): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. - Week 24 Treatment and Mid-point Evaluation Visit (4-6 inpatient days): Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected, including repeating the d-xylose carbohydrate malabsorption testing, the 24 hour stool collection for protein malabsorption and the 48 hour stool collection for fat malabsorption. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. - Week 32 Treatment Visit: Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. - Week 40 Treatment Visit: Medical history, physical exam, measurement of vital signs and weight, review of medications, and an assessment of number and consistency of stools each day. Blood, urine and stool samples will be collected. A pregnancy test for women of childbearing potential. A nurse will give you one 90 mg dose of ustekinumab by a very small needle injected under the skin, and then observe you for 1 hour. - Week 48 ...
The immune system is an intricate system comprised of specialized cells, proteins, tissues and organs. Proper functioning is critical to the body's ability to defend itself against harmful pathogens. Immunological disorders and deficiencies are defects in the immune system that lead to abnormal immune responses. Abnormal immune responses could be derived from immune deficiencies, dysregulations or hypersensitivities. The overall goal of this research study is to identify the mechanisms of primary immune deficiencies and immune disorders at the genetic, cellular and molecular level, using novel analytic techniques to be performed on immune cells derived from blood samples. The knowledge gained from the aims of this study could lead to better diagnostics and identify novel targets for therapeutic interventions.
The primary objective is to demonstrate the bioequivalence of Gammaplex® 10 intravenous immunoglobulin (IGIV) and Gammaplex® 5% IGIV with respect to area under the curve within a 28-day dosing interval (AUC0-28) in a cohort of adult subjects. The secondary objectives are to demonstrate the bioequivalence of Gammaplex® 10 IGIV and Gammaplex® 5% IGIV with respect to area under the curve within a 21-day dosing interval (AUC0-21) in adult subjects; to assess the pharmacokinetics of Gammaplex 10 IGIV and Gammaplex 5% IGIV including Immunoglobulin G (IgG) trough levels and to investigate the safety and tolerability of Gammaplex 10 IGIV and Gammaplex 5% IGIV in adults subjects; to assess the pharmacokinetics of Gammaplex 10 IGIV including IgG trough levels and to investigate the safety and tolerability of Gammaplex 10 IGIV in pediatric subjects.
Patients with Common variable immunodeficiency (CVID) have various forms of autoimmune and auto inflammatory disorders. The study will investigate if intervention with Rifaximin modifies the gut microbiota with a subsequent alteration in markers of systemic immune activation and inflammation in patients with CVID. The investigators hypothesize that the gut microbiota of CVID patients, at least partly through interaction with the innate immune system within the intestine, contribute to a low-grade systemic inflammation in these patients, and that an intervention with the non-absorbable antibiotic Rifaximin attenuates systemic inflammation through modulation of the gut microbiota. The study may lead to increased understanding of the interaction between microbiota and the immune system. The study could give new insight into important disease processes in relation to the interaction between the microbiota, the intestine and the systemic compartment, and potentially be the basis of new therapeutic strategies in these patients to prevent and down-regulate the auto-inflammatory and autoimmune complications seen in CVID. The findings could also be of relevance for other disorders where the interaction between microbiota and intestinal and systemic inflammation is involved such as various cardiovascular and metabolic disorders. The investigators hypothesize that the gut microbiota of CVID patients, at least partly through interaction with the innate immune system within the intestine, contribute to a low-grade systemic inflammation in these patients, and that an intervention with the non-absorbable antibiotic Rifaximin attenuates systemic inflammation through modulation of the gut microbiota.