Common Variable Immunodeficiency (CVID) Clinical Trials

The purpose of this protocol is to carry out laboratory studies concerning the immunopathogenesis of Common Variable Immunodeficiency (CVI) and related primary humoral immunodeficiency diseases. Additionally, we aim to document and track the progression of known complications of this primary immunodeficiency. Complications associated with CVID include recurrent respiratory, and gastrointestinal ...

This study is designed to explore CDZ173, a selective PI3K inhibitor, in patients with genetically activated PI3K, i.e., patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/ p110-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)I. The study consists of two parts. Part I is the open label part designed to ...

This protocol is designed as an adjunct to other NIAID IRB approved protocols that allow for genetic testing, which may include those that are screening protocols for patients with rare primary immunodeficiency or immunodysregulation disorders. Patients deemed of sufficient research interest after review of outside medical records, clinical evaluation, and ...

Bronchiectasis, or abnormal dilation of the airways, is a condition typically characterized by chronic and recurring respiratory tract infections. Frequently, depending on the underlying cause, these infections involve the entire respiratory tract resulting in sinus, ear, and lung disease. This condition used to be more common in children prior to ...

Based on diagnosis and clinical history, a determination of the most appropriate regimen will be made based on the following prep plans: Arm A: Fully Myeloablative Preparative Regimen, Arm B: Reduced Toxicity Ablative Preparative Regimen, Arm C: Reduced Intensity Conditioning, Arm D: No Preparative Regimen

An increased susceptibility to bacterial and viral infections is the hallmark primary immunodeficiencies (PIDs). The most common PIDs requiring treatment with Ig replacement (SCIg or IVIg) is Common Variable Immune Deficiency (CVID), which is diagnosed by the presence of hypogammaglobulinemia plus defective responses to vaccine antigens. Prior to diagnosis, CVID ...

Background Primary immunodeficiency diseases (PIDs) are conditions associated with major quantitative or qualitative immunologic abnormalities that are, in most cases, due to defects in cells of hematopoietic origin Patients with PID can have life-threatening complications including malignancy, recurrent infection, and autoimmunity/immune dysregulation Allogeneic blood or marrow transplantation (allo BMT) has ...

The purpose of this study is to assess the safety/tolerability and efficacy of using ustekinumab in subjects with common variable immunodeficiency CVID or selective IgG subclass deficiency (functional agammaglobulinemia) who have associated symptomatic gastrointestinal inflammation (CVID enteropathy). Ustekinumab (a Food and Drug Administration [FDA] approved drug) is a monoclonal antibody ...

Hematopoietic stem cell transplantation is the only curative choice for a number of inherited bone marrow failure syndromes, hemoglobinopathies, metabolic disorders and primary immune deficiencies. While survival of these patients is typically better than survival of patients with malignancies, toxicities of conditioning regimens and failure of engraftment remain challenges. Most ...

This is a single-site, pilot study conducted in the US to determine the benefit of human C1-esterase inhibitor [recombinant] (C1-INH-R) therapy to ameliorate ADRs in subjects receiving IVIG therapy for CVID for experience ADRs post-infusion. CVID subjects who are currently receiving IVIG every 3 to 4 weeks for immunodeficiency and ...