{"artifact":{"id":"experiment-exp-wiki-experiments-4r-tau-targeting-psp-cbs","artifact_type":"experiment","entity_ids":null,"title":"4R-Tau Targeting Therapies for PSP and CBS","quality_score":0.675,"created_by":"crosslink-v2","provenance_chain":null,"content_hash":null,"metadata":{"source":{"type":"manual","source_name":"wiki","extracted_by":"backfill_v1","extraction_date":"2026-04-16T01:00:16.899795Z"},"summary":"# 4R-Tau Targeting Therapies for PSP and CBS\n## Background and Rationale\nThis Phase II randomized, double-blind, placebo-controlled clinical trial investigates isoform-selective 4R-tau targeting therapies for Progressive Supranuclear Palsy (PSP) and Corticobasal Syndrome (CBS). These primary 4R-tauopathies represent distinct pathological entities from Alzheimer's disease, characterized by selective aggregation of four-repeat tau isoforms in neurons and glia. The pathological hallmark involves mi","entities":{"genes":["PSP"],"diseases":["Alzheimer's Disease"]},"model_system":"human","_schema_version":1,"experiment_type":"clinical","primary_outcome":"Validate 4R-Tau Targeting Therapies for PSP and CBS","methodology_notes":"Phase 1 (Weeks 0-4): Screen 300 participants with probable PSP (MDS-PSP criteria) or CBS (Armstrong criteria) aged 40-80. Conduct comprehensive baseline assessments including neuropsychological batteries, movement disorder scales, volumetric brain MRI, and lumbar puncture for CSF biomarkers. Randomize 180 eligible participants 2:1 to mAb-4RT (15 mg/kg IV monthly) versus placebo. Phase 2 (Weeks 4-52): Administer monthly infusions with safety monitoring including vital signs, laboratory assessments, and immunogenicity panels. Conduct clinical evaluations every 8 weeks using PSP Rating Scale, CBS Functional Rating Scale, and Schwab-England Activities of Daily Living. Perform CSF sampling at weeks 12, 24, and 48 for 4R-tau quantification via liquid chromatography-tandem mass spectrometry. Phase 3 (Weeks 24, 48): Execute neuroimaging assessments including structural MRI for volumetric analysis of midbrain, superior cerebellar peduncle, and cortical regions using FreeSurfer processing. 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