Active clinical trials for "Primary Immunodeficiency Diseases"

Study Design: This is a 2 arm non-interventional trial that will compare the occurrence of adverse drug reactions between Octagam 5% and other marketed IVIG infusion treatments.

Background: - People with primary immune deficiency diseases (PIDD) have weak immune systems. This makes it hard for their bodies to fight infection. The Immune Deficiency Foundation has a network to collect data about people with PIDD. It is called the United States Immunodeficiency Network. It will help doctors and scientists better understand these disorders. The goal is to get medical data for everyone with these disorders in the U.S. and Canada. Data will be stored in a registry. Researchers can use it to study if these disorders are increasing. They can also learn how the disorders are diagnosed and treated. Objectives: - To collect data on people with primary immune deficiency disorders. Eligibility: - People who have a PIDD. Design: Data can be added with no record of personal identity. Data can be added with identity kept separate. This data will be linked to the registry by a code number. Data for the registry includes: Family history Disease treatment Disease characteristics Medical history Laboratory data

Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs). Patients who experience AEs on any 10% IVIG solution will be changed to octagam 5% for six infusions to evaluate the potential benefit for reduction of AEs on a lower concentration IVIG product.

The management of patients with primary immune deficiency is increasingly codified, however contraception and pregnancy have not yet extensively studied or codified, and the medical monitoring and the prevention of infectious complications thus remains at the discretion of the practitioner. The aim the research is to study the obstetric features and outcome of patients with primary immune defects.

The purpose of this study is to evaluate patients with primary immunodeficiency disorders to identify patients with mutations of the genes for the following proteins: Jak3, STAT1, STAT4, interleukin-7, interleukin-7 receptor, interleukin-12 receptor subunits, and others. Patients will undergo screening history, physical examination, and clinical laboratory evaluation at referring institutions and tissue samples, or cell lines will be sent to the NIH. We will establish cell lines if necessary, prepare DNA and RNA for molecular genetic analysis and study cytokine signal transduction in patient cell lines.

Some Primary Immune Deficiencies can be associated with an inflammatory bowel disease, mimicking Crohn disease : the Chronic Granulomatous Disease (CGD), the XIAP deficiency, and the TTC7A deficiency. This inflammatory bowel disease is frequent but inconstant, raising questions about other factors contributing to the disease. The aim of our study is to analyze, describe and compare the gut microbiota of patients with those primary immune deficiency, with or without intestinal disease. The investigators can expect, in the long term, to compare on a same patient, the gut microbiota evolution, and to assess the role of gut microbiota modifications on the onset of an inflammatory bowel disease.

This study is a retrospective chart review of the use of subcutaneous IgG (SCIG) in infants less than two years old.

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

The purpose of this observational, multi-center, retrospective cohort study is to assess treatment patterns of Cuvitru (SCIG) 20 percent (%) and HyQvia (fSCIG) in polish pediatric participants with PID. The study will collect pediatric patient data. These data are gathered and collected during routine clinical care. As this is a non-interventional/observational study, no treatment/pharmacotherapy is provided as part of the study.

This study will provide insights on the infusion parameters, dosing, and experience of participants transitioning to CUVITRU in a real-world setting.