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.
Authors
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
Querolt, Montse
Griffin, Rhonda
Hames, Carrie
Mondou, Elsa
Price, Jeffrey
Sanz, Ana
Lin, Jiang
Publication Date
2022-04Journal Title
J Clin Immunol
ISSN
0271-9142
Publisher
Springer Science and Business Media LLC
Volume
42
Issue
3
Pages
500-511
Language
en
Type
Article
This Version
VoR
Metadata
Show full item recordCitation
Santamaria, M., Neth, O., Douglass, J. A., Krivan, G., Kobbe, R., Bernatowska, E., Grigoriadou, S., et al. (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
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.
Keywords
Original Article, Primary immunodeficiency, immunoglobulin replacement therapy, subcutaneous, 20% immunoglobulin, GTI1503
Identifiers
s10875-021-01181-6, 1181
External DOI: https://doi.org/10.1007/s10875-021-01181-6
This record's URL: https://www.repository.cam.ac.uk/handle/1810/336211
Rights
Licence:
http://creativecommons.org/licenses/by/4.0/
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