{"id":"W4212774905","doi":"10.1016/j.jcf.2022.02.003","title":"A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant","year":2022,"lang":"en","type":"article","venue":"Journal of Cystic Fibrosis","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":13,"is_retracted":false,"has_abstract":false,"ca_institutions":"BC Children's Hospital","funders":"Engineering and Physical Sciences Research Council; National Institute for Health and Care Research; NIHR Imperial Biomedical Research Centre; Vertex Pharmaceuticals; AbbVie; Cystic Fibrosis Trust; Cystic Fibrosis Foundation","keywords":"Ivacaftor; Tolerability; Medicine; Cystic fibrosis; Discontinuation; Adverse effect; Internal medicine; Clinical endpoint; 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":[],"consensus_categories":[],"category_scores_codex":[0.003006235,0.0002441859,0.001192546,0.0004113398,0.0002107026,0.00007696493,0.0004724123,0.00006719628,0.00003874064],"category_scores_gemma":[0.004091898,0.000177176,0.0001306984,0.0005042345,0.0001743896,0.0001917205,0.0005280045,0.0003817993,2.474395e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001816169,"about_ca_system_score_gemma":0.0004219677,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001339981,"about_ca_topic_score_gemma":0.00001586072,"domain_scores_codex":[0.9963507,0.0003912545,0.001564672,0.0004147567,0.0008845043,0.0003941179],"domain_scores_gemma":[0.9952688,0.003067443,0.0006975279,0.0004194582,0.0002967125,0.000250106],"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":[0.8961614,0.01909191,0.001512932,0.0003991677,0.0009413814,0.0001517745,0.001415472,0.0002849603,0.02900674,0.00003425922,0.001406905,0.04959304],"study_design_scores_gemma":[0.5231333,0.3475643,0.1180628,0.0005518394,0.001734872,0.001566282,0.002898589,0.0008450875,0.0006563939,0.0008080592,0.001730165,0.0004483279],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9883812,0.000623233,0.002269353,0.0003311009,0.0001059075,0.007742215,0.0005359189,0.000007292642,0.000003750154],"genre_scores_gemma":[0.9846653,0.00002643407,0.01452733,0.00007385455,0.0002224314,0.0003551698,0.00002031008,0.00004565708,0.00006345813],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3730282,"threshold_uncertainty_score":0.7225028,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05493126610839461,"score_gpt":0.3762085119645562,"score_spread":0.3212772458561616,"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."}}