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, ManuelNeth, OlafDouglass, Jo AKrivan, GergelyKobbe, RobinBernatowska, EwaGrigoriadou, SofiaBethune, ClaireChandra, AnitaHorneff, GerdBorte, MichaelSonnenschein, AnjaKralickova, PavlinaSanchez-Ramon, SilviaLangguth, DamanGonzalez-Granado, Luis IgnacioAlsina, LaiaQuerolt, MontseGriffin, RhondaHames, CarrieMondou, ElsaPrice, JeffreySanz, AnaLin, Jiang2023-05-032023-05-032022-04Santamaria 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.http://hdl.handle.net/10668/20596The 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.enAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/20% immunoglobulinGTI1503Primary immunodeficiencyimmunoglobulin replacement therapysubcutaneousAdolescentAdultChildHumansImmunoglobulin GImmunoglobulins, IntravenousImmunologic Deficiency SyndromesImmunologic FactorsInfusions, SubcutaneousA Multi‑Center, Open‑Label, Single‑Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency.research article34973143open accessInmunoglobulinasDosificaciónInmunoglobulina GCromatografíaInfecciones bacterianasFarmacocinéticaCaprilatosTerapéuticaPacientes10.1007/s10875-021-01181-61573-2592PMC9016006https://link.springer.com/content/pdf/10.1007/s10875-021-01181-6.pdfhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC9016006/pdf