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RECRUITING
NCT06732180
PHASE1

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

Sponsor: Gain Therapeutics, Inc.

View on ClinicalTrials.gov

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

Interventions

DRUG

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