biostudies-literatureEijsvogel PMichael J. Fox Foundation for Parkinson's Research (Michael J. Fox Foundation)Michael Fund International Foundation for Genetics ResearchMichael J. Fox Foundation for Parkinson’s Research2631-2640https://www.ebi.ac.uk/biostudies/studies/S-EPMC11405261biostudies-literatureUnknown30(9)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 primNature medicineTarget engagement and immunogenicity of an active immunotherapeutic targeting pathological α-synuclein: a phase 1 placebo-controlled trial.PMC11405261MJFF-020184Misra PConcha-Marambio LRadanovic IMirski DGroeneveld GJMa YSun YSKremer PVroom MMSinger WDodart JCDing SFedor LFarris CMHsieh YTde Kam MLShareghi GEijsvogel PShahnawaz MYu HJBoyd JDVissers MFJMfalseTarget engagement and immunogenicity of an active immunotherapeutic targeting pathological α-synuclein: a phase 1 placebo-controlled trial.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 prim2024-01-01T00:00:00Z2024 Sep2026-06-01T07:01:12.464Z2025-04-04T01:38:16.986ZS-EPMC114052613890254610.1038/s41591-024-03101-8