{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.002430243,0.002044638,0.002725602,0.0004776929,0.0006938765,0.001153516,0.001218864,0.002146022,0.005346643],"category_scores_gemma":[0.001496963,0.0007918417,0.001857594,0.0003642569,0.001438269,0.001445761,0.0004933423,0.004721546,0.001172657],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00117881,"about_ca_system_score_gemma":0.00264949,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001409141,"about_ca_topic_score_gemma":0.003724626,"domain_scores_codex":[0.9991167,0.0003853194,0.00003962517,0.000151626,0.0001033109,0.0002034354],"domain_scores_gemma":[0.9986224,0.0002199129,0.0003036103,0.0001011533,0.0001126924,0.0006402306],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.8485236,0.09167103,0.003966115,0.0005254367,0.0006809289,0.0002761651,0.0002207017,0.001177833,0.02404563,0.0002562984,0.002912115,0.02574415],"study_design_scores_gemma":[0.6307495,0.3582261,0.006929693,0.00002869238,0.0001601027,0.0001451971,0.00004787436,0.000650257,0.001590198,0.0001274835,0.001321958,0.00002283208],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9847758,0.001674067,0.0007674698,0.0005451487,0.0002223141,0.00799689,0.002109859,0.0001696753,0.001738729],"genre_scores_gemma":[0.9736614,0.001435756,0.003636499,0.002031502,0.0004022994,0.01161879,0.003466952,0.00005548315,0.003691392],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.005346643,"threshold_uncertainty_score":0.01788634,"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."}}