%0 Journal Article
%A Santamaria, Manuel
%A Neth, Olaf
%A Douglass, Jo A
%A Krivan, Gergely
%A Kobbe, Robin
%A Bernatowska, Ewa
%A Grigoriadou, Sofia
%A Bethune, Claire
%A Chandra, Anita
%A Horneff, Gerd
%A Borte, Michael
%A Sonnenschein, Anja
%A Kralickova, Pavlina
%A Sanchez-Ramon, Silvia 
%A Langguth, Daman
%A Gonzalez-Granado, Luis Ignacio
%A Alsina, Laia
%A Querolt, Montse
%A Griffin, Rhonda
%A Hames, Carrie
%A Mondou, Elsa
%A Price, Jeffrey
%A Sanz, Ana
%A Lin, Jiang
%T A Multi‑Center, Open‑Label, Single‑Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency.
%D 2022
%U http://hdl.handle.net/10668/20596
%X The purpose of this phase 3 study was to evaluate the efficacy, pharmacokinetics (PK), and safety of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) in patients with primary immunodeficiency (PI). Immunoglobulin treatment-experienced subjects with PI received 52 weeks of IGSC 20% given weekly at the same dose as the subject's previous IgG regimen (DAF 1:1); the minimum dose was 100 mg/kg/week. The primary endpoint was serious bacterial infections (SBIs [null vs alternative hypothesis: SBI rate per person per year ≥ 1 vs  Sixty-one subjects (19 children [≤ 12 years], 10 adolescents [> 12-16 years], and 32 adults) were enrolled. The rate of SBIs per person per year was 0.017. The 1-sided 99% upper confidence limit was 0.036 ( 12-16 years], and 32 adults) were enrolled. The rate of SBIs per person per year was 0.017. The 1-sided 99% upper confidence limit was 0.036 ( IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.
%K 20% immunoglobulin
%K GTI1503
%K Primary immunodeficiency
%K immunoglobulin replacement therapy
%K subcutaneous
%~