Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
Bone Marrow Failure SyndromeThalassemia14 moreIn this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy
Common Variable ImmunodeficiencyX-linked Agammaglobulinemia1 moreThe objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
"Prime Boost" Vaccination Strategy Combining Conjugated Anti- Pneumococcal Vaccine (s0) and Polysaccharide...
Common Variable ImmunodeficiencyThe main objective of this study is to evaluate and to compare the specific antibody response to a " prime-boost " vaccine strategy combining the seven valence pneumococcal conjugate vaccine (PnCj) prime at W0 followed by the administration of the pneumococcal capsular polysaccharide vaccine (PPS) boost at W4, to the administration of the pneumococcal capsular polysaccharide vaccine (PPS) alone at W4 in patients with common variable immunodeficiency.
Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients
Immunologic Deficiency SyndromeAgammaglobulinemia3 moreThe objective of this study is to determine if the safety and tolerability of Immune Globulin Intravenous (Human), 10% caprylate/chromatography (IGIV-C)purified is similar when infused at two different infusion rates. The primary objective is to compare the incidence and severity of all infusion related adverse events when IGIV-C, 10% is administered at a rate of 0.14 mL/kg/min compared to a rate of 0.08 mL/kg/min after a single daily infusion.
Assessment of Immunogenicity of Zostavax® in Patients With Antibody Deficiency 60 Years of Age and...
Common Variable Immune DeficiencySpecific Antibody Deficiency1 moreRecommendations concerning the administration of Zostavax® in patients with antibody deficiency are unclear. The investigators plan to assess the immunogenicity and safety of Zostavax® in patients with antibody deficiency as compared with healthy volunteers.
Study of Efficacy of CDZ173 in Patients With APDS/PASLI
Common Variable Immunodeficiency (CVID)APDS / PASLIThis 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.
Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10...
Primary Immune Deficiency DisordersCommon Variable Immunodeficiency2 moreThe 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.
Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin...
Common Variable ImmunodeficiencyAgammaglobulinemiaObjective: Measure serum IgG antibody to Streptococcus pneumoniae serotypes 1, 3, 5, 6B, 9V e 14, Haemophilus influenzae type b and tetanus toxoid in patients with primary antibody deficiencies who were treated with subcutaneous immunoglobulin infusions.
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies...
AgammaglobulinemiaIgG Deficiency1 moreThe purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency. As secondary endpoints the rate of overall infections, the tolerability and safety of IVIG-F10 are studied.
Efficacy and Safety of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies...
AgammaglobulinemiaIgG Deficiency1 moreThe purpose of this study is to demonstrate the effect of IgPro10 on the prevention of serious bacterial infections in patients with primary immunodeficiency. As secondary endpoints the rate of overall infections, the tolerability and safety of IgPro10 are studied. A part of the patients are participating in a pharmacokinetic substudy.