{"id":"W4322623206","doi":"10.1016/j.ymgme.2023.107437","title":"DESIGN OF A PHASE 3 STUDY OF AAV-MEDIATED GENE TRANSFER OF ORNITHINE TRANSCARBAMYLASE (OTC) IN PATIENTS WITH LATE-ONSET OTC DEFICIENCY","year":2023,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Metabolism and Genetic Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":false,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"","keywords":"Medicine; Hypoglycemia; Insulin; Intensive care medicine; Internal medicine; Bioinformatics; Endocrinology; Pediatrics; Biology","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":[0.003212319,0.002307571,0.002135684,0.0006999646,0.001193664,0.001372068,0.001210127,0.002290966,0.003920021],"category_scores_gemma":[0.001312927,0.0008207886,0.00164454,0.0005163087,0.002337008,0.001061055,0.0006099769,0.003453304,0.0009544012],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001871387,"about_ca_system_score_gemma":0.004224896,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002282979,"about_ca_topic_score_gemma":0.002744468,"domain_scores_codex":[0.9984158,0.0006116831,0.00008006558,0.0003202462,0.0001229614,0.0004491727],"domain_scores_gemma":[0.9991543,0.0001772962,0.00009997584,0.0001230268,0.00007299949,0.000372366],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"not_applicable","study_design_scores_codex":[0.3760808,0.1650019,0.01330679,0.0007631528,0.001027578,0.002485777,0.001367404,0.008713271,0.3539082,0.003089007,0.003402373,0.07085378],"study_design_scores_gemma":[0.2414355,0.7104686,0.006058549,0.00006328071,0.000539261,0.0009151116,0.0002766961,0.00261471,0.02904933,0.0008891997,0.007612633,0.00007712151],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"protocol","genre_scores_codex":[0.9685147,0.0008108785,0.006971453,0.0004979178,0.000302009,0.01980438,0.001122768,0.0001437884,0.001832077],"genre_scores_gemma":[0.9484468,0.0007140047,0.01588261,0.001023202,0.0002391082,0.02959434,0.001417443,0.00007876724,0.002603576],"genre_candidate":"protocol","genre_consensus":null,"teacher_disagreement_score":0.003920021,"threshold_uncertainty_score":0.01698858,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.008385522277556418,"score_gpt":0.2293500238029689,"score_spread":0.2209645015254124,"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."}}