RT Journal Article
T1 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.
A1 Santamaria, Manuel
A1 Neth, Olaf
A1 Douglass, Jo A
A1 Krivan, Gergely
A1 Kobbe, Robin
A1 Bernatowska, Ewa
A1 Grigoriadou, Sofia
A1 Bethune, Claire
A1 Chandra, Anita
A1 Horneff, Gerd
A1 Borte, Michael
A1 Sonnenschein, Anja
A1 Kralickova, Pavlina
A1 Sanchez-Ramon, Silvia
A1 Langguth, Daman
A1 Gonzalez-Granado, Luis Ignacio
A1 Alsina, Laia
A1 Querolt, Montse
A1 Griffin, Rhonda
A1 Hames, Carrie
A1 Mondou, Elsa
A1 Price, Jeffrey
A1 Sanz, Ana
A1 Lin, Jiang
K1 20% immunoglobulin
K1 GTI1503
K1 Primary immunodeficiency
K1 immunoglobulin replacement therapy
K1 subcutaneous
AB 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.
PB Springer New York LLC
YR 2022
FD 2022-04
LK http://hdl.handle.net/10668/20596
UL http://hdl.handle.net/10668/20596
LA en
NO Santamaria M, Neth O, Douglass JA, Krivan G, Kobbe R, Bernatowska E, et al. 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. J Clin Immunol. 2022 Apr;42(3):500-511.
DS RISalud
RD Apr 10, 2025