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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
Sponsor: Gain Therapeutics, Inc.
Summary
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).
Official title: An Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Participants With Parkinson's Disease With or Without a Pathogenic GBA1 Mutation
Key Details
Gender
All
Age Range
30 Years - 85 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2025-02-21
Completion Date
2025-11-30
Last Updated
2025-05-06
Healthy Volunteers
No
Conditions
Interventions
GT-02287
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
Locations (7)
St Vincent's Hospital Sydney
Darlinghurst, New South Wales, Australia
Southern Neurology
Kogarah, New South Wales, Australia
Westmead Hospital
Westmead, New South Wales, Australia
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
CMAX
Adelaide, South Australia, Australia
Alfred Health
Melbourne, Victoria, Australia
Royal Melbourne Hospital
Parkville, Victoria, Australia