Efficacy, Safety, Tolerability, and Biomarker Effects of GT-02287 in Early Parkinson's Disease

This is a 48-week, double-blind, randomized, placebo-controlled Phase 2a study testing two doses of oral GT-02287 in people with early Parkinson's disease (PD), both treated and untreated.

The study has three parts:

* Screening Period lasting up to 45 days * Treatment Period lasting about 341 days * Follow-up Period lasting up to 33 days Participants will have 7 onsite visits for efficacy, safety, tolerability, and biomarker assessments (Week 4, Week 8, Week 12, Week 24, Week 36, Week 48, and a Follow-Up Visit at Week 52). In addition, there will be 5 additional visits for laboratory blood tests (Weeks 2, 16, 20, 30, and 42).

Approximately 111 participants will be randomized into three groups (high dose, low dose, placebo).

Participants can have idiopathic PD or be heterozygous for a pathogenic variant in the GBA1 gene. Participants who have other PD-associated genetic variants (e.g., leucine rich repeat kinase 2 \[LRRK2\]) are ineligible. All participants will be genotyped to determine their PD-associated genetic status before enrollment.

At the start, participants undergo screening and baseline tests, including motor assessments, quality of life, sensor measurements, and collection of fluid and blood samples for biomarkers. Some baseline tests may be done just before dosing.

The study will measure efficacy through various motor, quality of life, disease progression, non-motor and other symptoms of PD, and cognitive tests, along with wearable sensor data.

Safety will be assessed by recording adverse events, lab tests, vital signs, body weight, heart monitoring, and questionnaires.

The study will also analyze GT-02287 levels in blood and spinal fluid, and its effects on biomarkers.