{"id":"W4387302584","doi":"10.1016/j.nmd.2023.07.027","title":"P46 ENVOL, a Phase 2, open-label trial evaluating the safety and expression of delandistrogene moxeparvovec in Duchenne muscular dystrophy: study design","year":2023,"lang":"en","type":"article","venue":"Neuromuscular Disorders","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Roche (Canada)","funders":"","keywords":"Duchenne muscular dystrophy; Open label; Medicine; Muscular dystrophy; Physical medicine and rehabilitation; Internal medicine; Clinical trial; Oncology; Physical therapy","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":[0.001781464,0.002343359,0.002279065,0.0003373187,0.0003640715,0.001279897,0.0007628254,0.001648692,0.007487357],"category_scores_gemma":[0.001222403,0.0005836413,0.001298161,0.0003116505,0.001277682,0.00102448,0.0003195446,0.002900941,0.001281306],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0007732221,"about_ca_system_score_gemma":0.001474835,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006730584,"about_ca_topic_score_gemma":0.001534753,"domain_scores_codex":[0.9993006,0.0003313196,0.00002346084,0.0001293825,0.00004144377,0.00017376],"domain_scores_gemma":[0.9993729,0.0001533428,0.0001167543,0.00004199773,0.0000307843,0.0002843073],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.9564751,0.009528696,0.0009229519,0.0005960628,0.0006931238,0.0001238966,0.00007054891,0.001024599,0.008657618,0.0004348173,0.001584281,0.01988828],"study_design_scores_gemma":[0.8374332,0.1567784,0.001626969,0.00002986558,0.0003366578,0.00007345421,0.00003858584,0.0006086155,0.001159136,0.0003347786,0.001554714,0.00002556122],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"protocol","genre_scores_codex":[0.9769784,0.002753041,0.002112481,0.0008089769,0.0004777912,0.008916828,0.002793798,0.0002662572,0.004892434],"genre_scores_gemma":[0.9688776,0.002328503,0.002990936,0.001800501,0.0004186121,0.01064671,0.001968125,0.00008010191,0.01088888],"genre_candidate":"protocol","genre_consensus":null,"teacher_disagreement_score":0.007487357,"threshold_uncertainty_score":0.02504772,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06171342177315503,"score_gpt":0.3690074144959761,"score_spread":0.307293992722821,"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."}}