{"id":"W2170032136","doi":"10.1136/thoraxjnl-2011-200393","title":"Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the <i>F508del-CFTR</i> mutation","year":2011,"lang":"en","type":"article","venue":"Thorax","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":496,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"National Center for Advancing Translational Sciences; University of Colorado Denver; National Institutes of Health; National Center for Research Resources; Georgia Clinical and Translational Science Alliance; Vertex Pharmaceuticals; University of Washington; Nationwide Children's Hospital; University of Pittsburgh; National Institute of Diabetes and Digestive and Kidney Diseases; Johns Hopkins University; Harvard Catalyst; Case Western Reserve University; Washington University in St. Louis; Harvard University; Hospital for Sick Children; Northwestern University; Cystic Fibrosis Foundation Therapeutics; Children's Hospital of Philadelphia","keywords":"Cystic fibrosis; Medicine; Placebo; Cystic fibrosis transmembrane conductance regulator; Tolerability; Pharmacodynamics; Adverse effect; Pharmacokinetics; Sweat test; Internal medicine; Discontinuation; Gastroenterology; Sweat gland; Pharmacology; Pathology; SWEAT","routes":{"ca_aff":true,"ca_fund":true,"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":[0.0007179359,0.0001782774,0.0004341716,0.0002100538,0.00008048582,0.00001307386,0.0002062759,0.00004837988,0.00002165334],"category_scores_gemma":[0.001090658,0.0001196176,0.00004788959,0.0005928997,0.0003425453,0.0001774177,0.00003935177,0.0001667176,0.00000263925],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00006942386,"about_ca_system_score_gemma":0.0002867057,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001336374,"about_ca_topic_score_gemma":0.002012148,"domain_scores_codex":[0.9979962,0.0001922834,0.000579582,0.0003275102,0.0006249191,0.0002795077],"domain_scores_gemma":[0.9975125,0.001137605,0.0002920731,0.0004760248,0.0004339542,0.0001478866],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.2382745,0.0333672,0.06127907,0.002857286,0.001138877,0.0006300591,0.105223,0.003947994,0.5179898,0.0007332786,0.002254117,0.03230483],"study_design_scores_gemma":[0.1260287,0.1305902,0.4499571,0.002538083,0.00153173,0.0005470493,0.0414894,0.05235224,0.1905021,0.002657298,0.0006049949,0.001201109],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9947561,0.0001616441,0.002236913,0.00007891504,0.00006910067,0.002321986,0.0002222848,0.00002826331,0.0001247792],"genre_scores_gemma":[0.9880978,0.000004894025,0.01136297,0.00002852421,0.00005022899,0.0002115135,0.0001537884,0.00002899241,0.00006125976],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.388678,"threshold_uncertainty_score":0.4877864,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06009892135269617,"score_gpt":0.3495034684645923,"score_spread":0.2894045471118961,"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."}}