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
Standard
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. / Santamaria, Manuel; Neth, Olaf; Douglass, Jo A; Krivan, Gergely; Kobbe, Robin; Bernatowska, Ewa; Grigoriadou, Sofia; Bethune, Claire; Chandra, Anita; Horneff, Gerd; Borte, Michael; Sonnenschein, Anja; Kralickova, Pavlina; Ramón, Silvia Sánchez; Langguth, Daman; Gonzalez-Granado, Luis Ignacio; Alsina, Laia; Querolt, Montse; Griffin, Rhonda; Hames, Carrie; Mondou, Elsa; Price, Jeffrey; Sanz, Ana; Lin, Jiang.
in: J CLIN IMMUNOL, Jahrgang 42, Nr. 3, 04.2022, S. 500-511.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
Harvard
APA
Vancouver
Bibtex
}
RIS
TY - JOUR
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
AU - Santamaria, Manuel
AU - Neth, Olaf
AU - Douglass, Jo A
AU - Krivan, Gergely
AU - Kobbe, Robin
AU - Bernatowska, Ewa
AU - Grigoriadou, Sofia
AU - Bethune, Claire
AU - Chandra, Anita
AU - Horneff, Gerd
AU - Borte, Michael
AU - Sonnenschein, Anja
AU - Kralickova, Pavlina
AU - Ramón, Silvia Sánchez
AU - Langguth, Daman
AU - Gonzalez-Granado, Luis Ignacio
AU - Alsina, Laia
AU - Querolt, Montse
AU - Griffin, Rhonda
AU - Hames, Carrie
AU - Mondou, Elsa
AU - Price, Jeffrey
AU - Sanz, Ana
AU - Lin, Jiang
N1 - © 2021. The Author(s).
PY - 2022/4
Y1 - 2022/4
N2 - PURPOSE: 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).METHODS: 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 < 1]). IgG subclasses and specific pathogen antibody levels were also measured.RESULTS: 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 (< 1), and the null hypothesis was rejected. The rate of hospitalization due to infection per person per year was 0.017 (2-sided 95% confidence interval: 0.008-0.033) overall. The mean trough total IgG concentrations were comparable to the previous IgG replacement regimen. The average of the individual mean trough ratios (IGSC 20%:previous regimen) was 1.078 (range: 0.83-1.54). The average steady-state mean trough IgG concentrations were 947.64 and 891.37 mg/dL, respectively. Seven subjects had serious treatment-emergent adverse events (TEAEs); none was drug-related. The rate of all TEAEs, including local infusion site reactions, during 3045 IGSC 20% infusions was 0.135. Most TEAEs were mild or moderate.CONCLUSIONS: IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.
AB - PURPOSE: 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).METHODS: 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 < 1]). IgG subclasses and specific pathogen antibody levels were also measured.RESULTS: 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 (< 1), and the null hypothesis was rejected. The rate of hospitalization due to infection per person per year was 0.017 (2-sided 95% confidence interval: 0.008-0.033) overall. The mean trough total IgG concentrations were comparable to the previous IgG replacement regimen. The average of the individual mean trough ratios (IGSC 20%:previous regimen) was 1.078 (range: 0.83-1.54). The average steady-state mean trough IgG concentrations were 947.64 and 891.37 mg/dL, respectively. Seven subjects had serious treatment-emergent adverse events (TEAEs); none was drug-related. The rate of all TEAEs, including local infusion site reactions, during 3045 IGSC 20% infusions was 0.135. Most TEAEs were mild or moderate.CONCLUSIONS: IGSC 20% demonstrated efficacy and good safety and tolerability in subjects with PI.
U2 - 10.1007/s10875-021-01181-6
DO - 10.1007/s10875-021-01181-6
M3 - SCORING: Journal article
C2 - 34973143
VL - 42
SP - 500
EP - 511
JO - J CLIN IMMUNOL
JF - J CLIN IMMUNOL
SN - 0271-9142
IS - 3
ER -