{"id":"W2525072188","doi":"10.1182/blood.v124.21.2732.2732","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: Results after 18 Months","year":2014,"lang":"en","type":"article","venue":"Blood","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":5,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University; Jewish General Hospital; Mount Sinai Hospital","funders":"","keywords":"Medicine; Clinical endpoint; Placebo; Adverse effect; Randomized controlled trial; Double blind; Surgery; Internal medicine","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":[0.002377148,0.0016838,0.002468741,0.0004142291,0.0006394202,0.0008876616,0.0008412171,0.001788698,0.005315827],"category_scores_gemma":[0.00127025,0.0005287054,0.001385228,0.0003754982,0.001099893,0.0009859035,0.0004668786,0.002653352,0.0009072277],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006255291,"about_ca_system_score_gemma":0.00136155,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005166905,"about_ca_topic_score_gemma":0.001538356,"domain_scores_codex":[0.9993018,0.0003669456,0.00004496248,0.0001201411,0.00007439932,0.00009172031],"domain_scores_gemma":[0.9987755,0.0002399267,0.0003636591,0.0001011293,0.0001014138,0.0004183769],"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":[0.9466265,0.02245109,0.002727345,0.0004810061,0.0008948591,0.00007379564,0.00009017003,0.0002894434,0.00896843,0.00006809163,0.0009875575,0.01634177],"study_design_scores_gemma":[0.8310969,0.1614566,0.005393054,0.00001595652,0.0003471517,0.00006284034,0.00002057391,0.0002002468,0.0007494466,0.00006418311,0.0005781887,0.00001489429],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9889756,0.001896856,0.0006125759,0.0003477152,0.00009952095,0.004666373,0.001707772,0.0001636368,0.001530001],"genre_scores_gemma":[0.9828866,0.001008461,0.001930859,0.001116029,0.0003076502,0.008134164,0.002531806,0.00003919336,0.00204518],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.005315827,"threshold_uncertainty_score":0.01778328,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02728809472378711,"score_gpt":0.3171527097417952,"score_spread":0.2898646150180081,"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."}}