{"id":"W4210650936","doi":"10.1016/j.ymgme.2021.11.204","title":"A phase 1/2 open-label, multicenter, dose ranging and confirmatory study to assess the safety, tolerability and efficacy of PBKR03 administered to pediatric subjects with early infantile Krabbe disease (globoid cell leukodystrophy; GALax-C)","year":2022,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"McGill University","funders":"","keywords":"Tolerability; Medicine; Dose-ranging study; Open label; Pediatrics; Adverse effect; Internal medicine; 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":[0.002128779,0.002263567,0.002588266,0.0005680702,0.0006938567,0.001166637,0.001204542,0.001909054,0.00424573],"category_scores_gemma":[0.001243655,0.0008251415,0.001277408,0.0004914761,0.00174057,0.001368389,0.0003394142,0.004245438,0.001190691],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001083889,"about_ca_system_score_gemma":0.002524255,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001657732,"about_ca_topic_score_gemma":0.003042111,"domain_scores_codex":[0.9993386,0.0003340545,0.00002192318,0.0001267791,0.00004921851,0.0001294364],"domain_scores_gemma":[0.9987414,0.0002289667,0.0002033022,0.0001475765,0.00009727728,0.0005813621],"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":[0.7495667,0.103603,0.005923359,0.0004612635,0.001059481,0.0004565157,0.0005295902,0.002316467,0.06670284,0.0006041495,0.004184101,0.06459262],"study_design_scores_gemma":[0.4389839,0.5435824,0.008905997,0.00002269511,0.000297711,0.0004560783,0.00009121609,0.0009236868,0.004330317,0.0002740982,0.002089536,0.00004240275],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9901686,0.001256702,0.001756468,0.0003673242,0.0001275066,0.003234506,0.001542219,0.0002177491,0.00132894],"genre_scores_gemma":[0.982417,0.001251959,0.004879106,0.001211617,0.0002188203,0.004074287,0.002778942,0.0001136874,0.003054708],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00424573,"threshold_uncertainty_score":0.01420337,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03006397250644244,"score_gpt":0.3293651749891859,"score_spread":0.2993012024827435,"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."}}