The main goal of this clinical trial is to learn about the safety and tolerability of GT-02287. The questions it aims to answer are: * What medical problems do participants have when taking GT-02287? * How is GT-02287 absorbed, distributed, and removed from the body of participants over time (pharmacokinetics)? * Are there any biological effects of GT-02287 in blood and in cerebrospinal fluid that could be beneficial for people with Parkinson's disease? Participants will: * visit the clinic to assess if they qualify for the study (30-day Screening Period) * if eligible, receive GT-02287 once a day every day for 90 days (90-day Open Label Treatment period) * visit the clinic the first day of treatment, after the first 2 weeks of treatment, and every month during the 90-day Treatment Period. * visit the clinic to assess how they are doing 14 days after the end of GT-02287 treatment (14-day Follow-Up Period).
This Phase 1b, 3-month, multicenter, open-label study of GT-02287 in participants with Parkinson's Disease with or without GBA1 mutations. The primary objective of the study is to evaluate the safety and tolerability of GT-02287. The study will also investigate pharmacokinetics (PK) and explore pharmacodynamic parameters in blood and CSF and effect of GT-02287 on scores from selected clinical scales. Approximately 20 participants will be enrolled to result in approximately 15 evaluable participants. All participants will receive an oral dose of GT-02287 of 13.5 mg/kg/day (plus/minus 2 mg/kg/day) once a day for 90 days.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Dose of 13.5 mg/kg/day (plus/minus 2 mg/kg/day based on body weight) to be administered orally once a day for 90 days. Dosage form: powder in sachet (200 mg of GT-02287 per sachet) for reconstitution with a suspending agent
St Vincent's Hospital Sydney
Darlinghurst, New South Wales, Australia
RECRUITINGSouthern Neurology
Kogarah, New South Wales, Australia
RECRUITINGWestmead Hospital
Westmead, New South Wales, Australia
RECRUITINGPrincess Alexandra Hospital
Woolloongabba, Queensland, Australia
RECRUITINGCMAX
Adelaide, South Australia, Australia
RECRUITINGAlfred Health
Melbourne, Victoria, Australia
RECRUITINGRoyal Melbourne Hospital
Parkville, Victoria, Australia
RECRUITINGIncidence, nature, relationship to investigational product (IP), and severity of adverse events (AEs)
Time frame: From first dose to Day 105
Incidence of clinically significant findings for clinical laboratory evaluations, physical and neurological examinations, body weight , vital signs measurements, 12-lead o 12-lead electrocardiograms (ECGs), Columbia Suicide Severity Rating Scale (C-SSRS)
Time frame: From first dose to Day 105
Maximum Plasma Concentration [Cmax]
Pharmacokinetics
Time frame: Day 1 and Day 90
Area under the curve [AUC]
Pharmacokinetics
Time frame: Day 1 and Day 90
Time to reach Cmax [Tmax]
Pharmacokinetics
Time frame: Day 1 and Day 90
Elimination half life [T1/2]
Pharmacokinetics
Time frame: Day 90- Day 93
Apparent Clearance (CL/F)
Pharmacokinetics
Time frame: Day 90- Day 93
Apparent Volume of Distribution (Vz/F)
Pharmacokinetics
Time frame: Day1 and Day 90- Day 93
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