{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Eijsvogel P"],"funding":["Michael J. Fox Foundation for Parkinson's Research (Michael J. Fox Foundation)","Michael Fund International Foundation for Genetics Research","Michael J. Fox Foundation for Parkinson’s Research"],"pagination":["2631-2640"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11405261"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["30(9)"],"pubmed_abstract":["Investigational therapeutics that target toxic species of α-synuclein (αSyn) aim to slow down or halt disease progression in patients with Parkinson's disease (PD). Here this 44-week, randomized, placebo-controlled, double-blind, single-center phase 1 study investigated safety, tolerability and immunogenicity of UB-312, an active immunotherapeutic targeting pathological αSyn, in patients with PD. The primary outcome measures were adverse event frequency and change in anti-αSyn antibody titers in blood and cerebrospinal fluid (CSF). Exploratory outcomes were changes in clinical scales and biomarker-based target engagement as measured by seed amplification assays. Twenty patients were randomized 7:3 (UB-312:placebo) into 300/100/100 μg or 300/300/300 μg (weeks 1, 5 and 13) intramuscular prim"],"journal":["Nature medicine"],"pubmed_title":["Target engagement and immunogenicity of an active immunotherapeutic targeting pathological α-synuclein: a phase 1 placebo-controlled trial."],"pmcid":["PMC11405261"],"funding_grant_id":["MJFF-020184"],"pubmed_authors":["Misra P","Concha-Marambio L","Radanovic I","Mirski D","Groeneveld GJ","Ma Y","Sun YS","Kremer P","Vroom MM","Singer W","Dodart JC","Ding S","Fedor L","Farris CM","Hsieh YT","de Kam ML","Shareghi G","Eijsvogel P","Shahnawaz M","Yu HJ","Boyd JD","Vissers MFJM"],"additional_accession":[]},"is_claimable":false,"name":"Target engagement and immunogenicity of an active immunotherapeutic targeting pathological α-synuclein: a phase 1 placebo-controlled trial.","description":"Investigational therapeutics that target toxic species of α-synuclein (αSyn) aim to slow down or halt disease progression in patients with Parkinson's disease (PD). Here this 44-week, randomized, placebo-controlled, double-blind, single-center phase 1 study investigated safety, tolerability and immunogenicity of UB-312, an active immunotherapeutic targeting pathological αSyn, in patients with PD. The primary outcome measures were adverse event frequency and change in anti-αSyn antibody titers in blood and cerebrospinal fluid (CSF). Exploratory outcomes were changes in clinical scales and biomarker-based target engagement as measured by seed amplification assays. Twenty patients were randomized 7:3 (UB-312:placebo) into 300/100/100 μg or 300/300/300 μg (weeks 1, 5 and 13) intramuscular prim","dates":{"release":"2024-01-01T00:00:00Z","publication":"2024 Sep","modification":"2026-06-01T07:01:12.464Z","creation":"2025-04-04T01:38:16.986Z"},"accession":"S-EPMC11405261","cross_references":{"pubmed":["38902546"],"doi":["10.1038/s41591-024-03101-8"]}}