Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease

Exclusion Criteria

• •Other neurological disorders, including but not limited to Alzheimer's disease, amyotrophic lateral sclerosis, frontotemporal dementia, progressive supranuclear palsy, corticobasal syndrome, Huntington's disease, multiple system atrophy, dementia with Lewy bodies, secondary (e.g. drug-induced) parkinsonism, multiple sclerosis, or epilepsy
• •A history of Gaucher disease or homozygous for a GBA1 pathogenic variant known to be associated with GD or compound heterozygous for 2 alleles that are known to be associated with GD.
• •Known PD-associated LRRK2 pathogenic variant or other PD-associated genetic mutations other than GBA1
• •Dementia or a moderate cognitive impairment (score ≥17 on the Montreal Cognitive Assessment)
• •Hypersensitivity to GT-02287 or any of its excipients
• •Concomitant medications metabolized primarily by cytochrome P450 3A4 (CYP3A4) that have a narrow therapeutic window, concomitant medications that are substrates of breast cancer resistance protein and/or P-glycoprotein and that have a narrow therapeutic window, concomitant medications that are potent inhibitors or inducers of CYP3A4
• •Use of dopamine antagonists (antipsychotics) or anticholinergic medications
• •Concomitant disease including, but not limited to cardiovascular conditions, diabetes, autoimmune disease, cancer, active infectious disease, psychotic disorders and symptoms, depressive symptoms, drug and/or alcohol misuse as defined in the protocol
• •Malabsorption or relevant disorder which may impact the absorption of GT-02287
• •Clinically significant abnormalities in laboratory test
• •Contraindications to lumbar puncture (LP)
• •Blood donation \>500 mL within 3 months
• •Unable to comply with restrictions on food products, smoking, and /or alcohol use as defined in protocol
• •participation in any interventional clinical study within 3 months or 5 half-lives, whichever is longer, prior to Screening