{"id":"W4324324977","doi":"10.1164/rccm.202301-0084oc","title":"Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2–5 Years with Cystic Fibrosis and at Least One <i>F508del</i> Allele","year":2023,"lang":"en","type":"article","venue":"American Journal of Respiratory and Critical Care Medicine","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":129,"is_retracted":false,"has_abstract":true,"ca_institutions":"St. Michael's Hospital; University of Toronto; BC Children's Hospital; University of British Columbia","funders":"National Institute of Diabetes and Digestive and Kidney Diseases; Vertex Pharmaceuticals","keywords":"Ivacaftor; Medicine; Tolerability; Cystic fibrosis; Adverse effect; Pharmacokinetics; Clinical endpoint; Pharmacodynamics; Internal medicine; Clinical trial; Cystic fibrosis transmembrane conductance regulator","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["sts"],"consensus_categories":[],"category_scores_codex":[0.001853691,0.0002576345,0.001554976,0.0004134473,0.00008756991,0.00002564176,0.000286137,0.00009362299,0.00007010636],"category_scores_gemma":[0.004126768,0.0001909968,0.00007628281,0.0007946952,0.003180058,0.0002090661,0.0002330506,0.0007432561,0.000003336738],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00009523634,"about_ca_system_score_gemma":0.0003294614,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00006152796,"about_ca_topic_score_gemma":0.000005183635,"domain_scores_codex":[0.9962245,0.000539099,0.001278269,0.0004371288,0.001034313,0.0004867023],"domain_scores_gemma":[0.9964095,0.001627123,0.0003725531,0.0003247733,0.0004133282,0.0008527444],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.3790643,0.003625074,0.2774169,0.0008242285,0.0005819681,0.001827922,0.0004275598,0.000003690592,0.03341625,0.000160863,0.004282104,0.2983691],"study_design_scores_gemma":[0.3293808,0.2604677,0.3932312,0.002754595,0.0008778665,0.0006913698,0.005199682,0.00002496583,0.0006908745,0.00008434409,0.00609906,0.0004976374],"study_design_candidate":"randomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9948485,0.00374186,0.00005177296,0.0005568611,0.00002969809,0.0006375465,0.00004661237,0.00001639495,0.00007076617],"genre_scores_gemma":[0.9963927,0.000522731,0.001728267,0.0006787255,0.0005743367,0.00001871366,0.00001699902,0.00004035397,0.00002723319],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2978715,"threshold_uncertainty_score":0.9995327,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0618414887940985,"score_gpt":0.4241833803070962,"score_spread":0.3623418915129977,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}