{"id":"W4231010457","doi":"10.1182/blood.v122.21.2275.2275","title":"Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results","year":2013,"lang":"en","type":"article","venue":"Blood","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"Jewish General Hospital; Mount Sinai Hospital","funders":"","keywords":"Enzyme replacement therapy; Medicine; Clinical endpoint; Placebo; Adverse effect; Internal medicine; Randomized controlled trial; Anemia; Bone marrow; Gastroenterology; Surgery; Disease; Pathology","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":[0.002779644,0.001921543,0.003496241,0.0005708797,0.0007469801,0.001069861,0.001060936,0.002287033,0.00652139],"category_scores_gemma":[0.001494275,0.0007646082,0.001798482,0.0004615832,0.001387254,0.001338839,0.0005432863,0.003067437,0.001052249],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0007868175,"about_ca_system_score_gemma":0.001377487,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006344679,"about_ca_topic_score_gemma":0.00194828,"domain_scores_codex":[0.9991143,0.0004621628,0.00005763622,0.0001511819,0.00008209073,0.0001326396],"domain_scores_gemma":[0.9987655,0.0002622387,0.0003210924,0.00009003734,0.0001089705,0.0004522501],"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":[0.9674708,0.01594703,0.001073125,0.0005747915,0.0007256123,0.00005253549,0.00005548944,0.0002219224,0.005008934,0.00006459116,0.0007587813,0.008046402],"study_design_scores_gemma":[0.9118966,0.08539944,0.001662346,0.00001421227,0.0002601437,0.00002506928,0.00001340669,0.0001322819,0.0002493496,0.00005626385,0.0002806531,0.00001018954],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9801943,0.003535699,0.0009372003,0.0007585526,0.0002926336,0.01025397,0.001911887,0.0002768644,0.001838824],"genre_scores_gemma":[0.9735961,0.001826789,0.002490503,0.002248151,0.0005193772,0.01458117,0.001789985,0.00004164247,0.002906346],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00652139,"threshold_uncertainty_score":0.02181625,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02996136580230417,"score_gpt":0.3169617012495453,"score_spread":0.2870003354472411,"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."}}