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dc.contributor.authorSantamaria, Manuel
dc.contributor.authorNeth, Olaf
dc.contributor.authorDouglass, Jo A
dc.contributor.authorKrivan, Gergely
dc.contributor.authorKobbe, Robin
dc.contributor.authorBernatowska, Ewa
dc.contributor.authorGrigoriadou, Sofia
dc.contributor.authorBethune, Claire
dc.contributor.authorChandra, Anita
dc.contributor.authorHorneff, Gerd
dc.contributor.authorBorte, Michael
dc.contributor.authorSonnenschein, Anja
dc.contributor.authorKralickova, Pavlina
dc.contributor.authorRamón, Silvia Sánchez
dc.contributor.authorLangguth, Daman
dc.contributor.authorGonzalez-Granado, Luis Ignacio
dc.contributor.authorAlsina, Laia
dc.contributor.authorQuerolt, Montse
dc.contributor.authorGriffin, Rhonda
dc.contributor.authorHames, Carrie
dc.contributor.authorMondou, Elsa
dc.contributor.authorPrice, Jeffrey
dc.contributor.authorSanz, Ana
dc.contributor.authorLin, Jiang
dc.date.accessioned2022-04-19T15:12:10Z
dc.date.available2022-04-19T15:12:10Z
dc.date.issued2022-04
dc.date.submitted2021-03-09
dc.identifier.issn0271-9142
dc.identifier.others10875-021-01181-6
dc.identifier.other1181
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/336211
dc.description.abstractPURPOSE: 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.
dc.languageen
dc.publisherSpringer Science and Business Media LLC
dc.subjectOriginal Article
dc.subjectPrimary immunodeficiency
dc.subjectimmunoglobulin replacement therapy
dc.subjectsubcutaneous
dc.subject20% immunoglobulin
dc.subjectGTI1503
dc.titleA Multi‑Center, Open‑Label, Single‑Arm Trial to Evaluate the Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency.
dc.typeArticle
dc.date.updated2022-04-19T15:12:10Z
prism.endingPage511
prism.issueIdentifier3
prism.publicationNameJ Clin Immunol
prism.startingPage500
prism.volume42
dc.identifier.doi10.17863/CAM.83631
dcterms.dateAccepted2021-11-16
rioxxterms.versionofrecord10.1007/s10875-021-01181-6
rioxxterms.versionVoR
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by/4.0/
dc.contributor.orcidGonzalez-Granado, Luis Ignacio [0000-0001-6917-8980]
dc.contributor.orcidAlsina, Laia [0000-0002-3559-0018]
dc.identifier.eissn1573-2592
cam.issuedOnline2022-01


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