This is a prospective, single-arm, single-dose clinical study designed to evaluate the safety, tolerability, efficacy, immunogenicity, pharmacodynamic (PD) and pharmacokinetic (PK) profiles of LY-N001 Injection in patients with moderate-to-advanced Parkinson's disease carrying GBA1 mutations. The study consists of a main study phase and a long-term follow-up phase.
Aged 30 to 70 years inclusive; both males and females are eligible.
The participant fully understands the purpose, nature, procedures of the study and potential adverse events, voluntarily agrees to take part in the study and signs the informed consent form (ICF).
Participants with clinically diagnosed idiopathic Parkinson's disease (PD) meeting the 2015 Movement Disorder Society (MDS) diagnostic criteria for idiopathic PD.
Participants carrying at least one pathogenic GBA1 variant.
Atypical or secondary parkinsonism (including Parkinson-plus syndromes, hereditary parkinsonism, drug-induced parkinsonism, etc.).
Patients with clinically confirmed LRRK2 mutation.
Previous history of pallidotomy, Deep Brain Stimulation (DBS) surgery, striatal surgery, extrapyramidal surgery, stereotactic brain surgery or other brain surgeries; or any other surgical history judged by the investigators to interfere with the subject's participation in this study.
Participants with previous head Computed Tomography (CT) / Magnetic Resonance Imaging (MRI) showing neurological diseases such as brain trauma, vascular malformations, hydrocephalus, brain tumors; subjects with hyperintense white matter signals on MRI indicating risk of intracranial hemorrhage; subjects with cerebrovascular lesions or microbleeds on SWI (Susceptibility-Weighted Imaging); subjects with vulnerable plaques or intraplaque hemorrhage of craniocervical arteries on HR-MRI (High-Resolution Magnetic Resonance Imaging); or subjects with imaging abnormalities in the striatum or other brain regions that substantially increase surgical risks.
Sequential
LY-N001 Injection
GeneticLY-N001 Injection shall be administered as a single intracerebroventricular (ICV) injection, with one administration only
Incidence of dose-limiting toxicity (DLT) events occurring within at least 28 days following a single intracranial administration of LY-N001
DLT events will be assessed per CTCAE Version 6.0.
Incidence of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) occurring during the treatment period
All AEs and SAEs will be graded per CTCAE Version 6.0 and adjudicated in accordance with SAE criteria.
Change from baseline in Movement Disorder Society Unified Parkinson's Disease Rating Scale Part III (MDS-UPDRS-III) motor examination scores during OFF and ON medication states
Efficacy was assessed via the change from baseline in the MDS-UPDRS Part III total score. The maximum possible total score of the scale is 132, and higher scores correspond to greater disease severity.
Parkinson's disease medication use: changes in daily oral levodopa (L-DOPA) dose or levodopa equivalent dose (LED)
Assessment was based on the change from baseline in daily Parkinson's disease medication usage.
Motor fluctuations assessed via patient diaries: changes in "ON" time without dyskinesia or without troublesome dyskinesia, and changes in "OFF" time
Assessment was conducted based on changes in ON/OFF time recorded in patient diaries.
Changes in scores of the MDS Unified Parkinson's Disease Rating Scale (MDS-UPDRS Part I, MDS-UPDRS Part II and MDS-UPDRS Part IV)
Assessment was performed based on the scores of MDS-UPDRS Part I, MDS-UPDRS Part II and MDS-UPDRS Part IV.The maximum total scores of MDS-UPDRS Part I, Part II and Part IV are 52, 52 and 24 respectively. Higher scores indicate more severe disease.
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