Research ExplorerPublicationsA Multi‑Center, Open‑Label, Single‑Arm Trial to Evaluate the...

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, Vol. 42, No. 3, 04.2022, p. 500-511.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Santamaria, M, Neth, O, Douglass, JA, Krivan, G, Kobbe, R, Bernatowska, E, Grigoriadou, S, Bethune, C, Chandra, A, Horneff, G, Borte, M, Sonnenschein, A, Kralickova, P, Ramón, SS, Langguth, D, Gonzalez-Granado, LI, Alsina, L, Querolt, M, Griffin, R, Hames, C, Mondou, E, Price, J, Sanz, A & Lin, J 2022, '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, vol. 42, no. 3, pp. 500-511. https://doi.org/10.1007/s10875-021-01181-6

APA

Santamaria, M., Neth, O., Douglass, J. A., Krivan, G., Kobbe, R., Bernatowska, E., Grigoriadou, S., Bethune, C., Chandra, A., Horneff, G., Borte, M., Sonnenschein, A., Kralickova, P., Ramón, S. S., Langguth, D., Gonzalez-Granado, L. I., Alsina, L., Querolt, M., Griffin, R., ... Lin, J. (2022). 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, 42(3), 500-511. https://doi.org/10.1007/s10875-021-01181-6

Vancouver

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. https://doi.org/10.1007/s10875-021-01181-6

Bibtex

@article{57ee5d040e9242c8a5401d89916f1049,
title = "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",
abstract = "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.",
author = "Manuel Santamaria and Olaf Neth and Douglass, {Jo A} and Gergely Krivan and Robin Kobbe and Ewa Bernatowska and Sofia Grigoriadou and Claire Bethune and Anita Chandra and Gerd Horneff and Michael Borte and Anja Sonnenschein and Pavlina Kralickova and Ram{\'o}n, {Silvia S{\'a}nchez} and Daman Langguth and Gonzalez-Granado, {Luis Ignacio} and Laia Alsina and Montse Querolt and Rhonda Griffin and Carrie Hames and Elsa Mondou and Jeffrey Price and Ana Sanz and Jiang Lin",
note = "{\textcopyright} 2021. The Author(s).",
year = "2022",
month = apr,
doi = "10.1007/s10875-021-01181-6",
language = "English",
volume = "42",
pages = "500--511",
journal = "J CLIN IMMUNOL",
issn = "0271-9142",
publisher = "Springer New York",
number = "3",

}

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 -