Hypogammaglobulinemia Clinical Trials

The overall objective is to evaluate X4P-001 in the treatment of patients with WHIM Syndrome.

The purpose of the study is to determine whether home subcutaneous immunoglobulin (ScIG) is tolerated as replacement therapy for hypogammaglobulinemia in patients that have undergone allogeneic hematopoietic cell transplantation (alloHCT). Secondary objective is to perform a financial analysis comparing the cost of ScIG with intravenous immunoglobulin (IVIG). The study hypothesis ...

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 ...

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 ...

Background Disorders of T-cell proliferation and/or dysregulation (TCP/D) can lead to T-cell lymphoproliferative disorders, autoimmunity, infection, and aberrant immune activation with resulting organ dysfunction, morbidity, and mortality. Allogeneic hematopoietic cell transplantation (HCT) has the potential to cure disorders of TCP/D. Subjects with TCP/D may be at higher risk for graft ...

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 ...

Mozobil (TM) (plerixafor injection, Genzyme Corp.) is a Food and Drug Administration approved medication to mobilize CD34+ hematopoietic stem cells prior to apheresis and use in autologous transplantation in non-Hodgkin lymphoma and multiple myeloma when used in conjunction with granulocyte-colony stimulating factor (G-CSF). The drug s mechanism of action is ...

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 ...

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 ...