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Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial.

dc.contributor.authorGriese, Matthias
dc.contributor.authorCosta, Stefano
dc.contributor.authorLinnemann, Rachel W
dc.contributor.authorMall, Marcus A
dc.contributor.authorMcKone, Edward F
dc.contributor.authorPolineni, Deepika
dc.contributor.authorQuon, Bradley S
dc.contributor.authorRingshausen, Felix C
dc.contributor.authorTaylor-Cousar, Jennifer L
dc.contributor.authorWithers, Nicholas J
dc.contributor.authorMoskowitz, Samuel M
dc.contributor.authorDaines, Cori L
dc.date.accessioned2024-12-05T16:21:09Z
dc.date.available2024-12-05T16:21:09Z
dc.identifier.pmid32969708
dc.identifier.doi10.1164/rccm.202008-3176LE
dc.identifier.urihttp://hdl.handle.net/10147/643456
dc.descriptionSafety results from this interim analysis were consistent with the initial 24-week placebo-controlled F/MF pivotal study, with similar or lower exposure-adjusted event rates observed in the OLE (Table 1) (5). ELX/TEZ/IVA was generally safe and well tolerated. Most AEs were consistent with common manifestations of CF and were not treatment limiting (3, 10). In participants who received ELX/TEZ/IVA in parent studies, improvements in efficacy and PD measures, including ppFEV1, SwCl concentration, BMI, CFQ-R RD score, and PEx event rate, were maintained or continued to improve further over 24 weeks (F/MF genotypes) or 36 weeks (F/F genotype) of additional treatment. These results validate the durability of ELX/TEZ/IVA efficacy responses, with no emerging safety concerns. Among participants who had received placebo or TEZ/IVA in the respective parent studies, initiation of ELX/TEZ/IVA rapidly led to marked improvements in these efficacy measures that were consistent with the results seen in the ELX/TEZ/IVA arms of those parent studies. Thus, the results of this combined-group interim analysis demonstrate the safety and sustained efficacy of long-term ELX/TEZ/IVA treatment in pwCF 12 years old or older with one or more F508del alleles.en_US
dc.language.isoenen_US
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International*
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/*
dc.subjectCYSTIC FIBROSISen_US
dc.subjectDRUG THERAPYen_US
dc.titleSafety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor for 24 Weeks or Longer in People with Cystic Fibrosis and One or More Alleles: Interim Results of an Open-Label Phase 3 Clinical Trial.en_US
dc.typeArticleen_US
dc.identifier.eissn1535-4970
dc.identifier.journalAmerican journal of respiratory and critical care medicineen_US
dc.source.journaltitleAmerican journal of respiratory and critical care medicine
dc.source.volume203
dc.source.issue3
dc.source.beginpage381
dc.source.endpage385
refterms.dateFOA2024-12-05T16:21:10Z
dc.source.countryUnited States
dc.source.countryUnited States


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Attribution-NonCommercial-NoDerivatives 4.0 International
Except where otherwise noted, this item's license is described as Attribution-NonCommercial-NoDerivatives 4.0 International