{"id":"W4393870177","doi":"10.1038/s41586-024-07266-7","title":"Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia","year":2024,"lang":"en","type":"article","venue":"Nature","topic":"Metabolism and Genetic Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":118,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"Moderna","keywords":"Adverse effect; Interim; Decompensation; Medicine; Propionic acidemia; Limiting; Pharmacology; Internal medicine; Interim analysis; Endocrinology; Clinical trial","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":[0.005582979,0.001158773,0.001628568,0.0002678598,0.0004492514,0.0008716202,0.000463371,0.001013632,0.004605437],"category_scores_gemma":[0.00456998,0.000364602,0.001497955,0.0002383196,0.0004957219,0.0008552664,0.000339377,0.002521337,0.0006574312],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004968517,"about_ca_system_score_gemma":0.0010657,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002826424,"about_ca_topic_score_gemma":0.0005916596,"domain_scores_codex":[0.9985878,0.0009059263,0.00005984932,0.0001195373,0.0001189241,0.0002079073],"domain_scores_gemma":[0.9976012,0.001011645,0.0005310164,0.0003057093,0.0002039085,0.0003464917],"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":[0.8201193,0.0175181,0.01006178,0.0008910093,0.002187096,0.0005325559,0.0003085824,0.005385505,0.03934074,0.0008569721,0.0115558,0.09124247],"study_design_scores_gemma":[0.3740072,0.5583847,0.02622205,0.0001046566,0.002064235,0.0005964966,0.0001387187,0.00346138,0.01553195,0.002097494,0.0173259,0.00006532182],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9782789,0.002608013,0.004108697,0.001118075,0.0003852648,0.005256849,0.003715882,0.0002766061,0.004251632],"genre_scores_gemma":[0.9724773,0.001135972,0.005773607,0.001680714,0.0002569173,0.00791689,0.005053237,0.00009490678,0.005610359],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.005582979,"threshold_uncertainty_score":0.02952594,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02314809631765712,"score_gpt":0.3893751206396006,"score_spread":0.3662270243219434,"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."}}