New drug slows Parkinson progression
- Trial ID
- NCT06732180
- 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
- Goal
- New drug slows Parkinson progression
- Phase
- PHASE1
- Status
- RECRUITING
- Sponsor
- Gain Therapeutics, Inc.
- Study Type
- INTERVENTIONAL
- Enrollment
- 20 participants
- Conditions
- Parkinson Disease
- Interventions
- GT-02287
Plain-Language Summary
The goal is to check safety, tolerability, and how GT-02287 behaves in the body while looking for signs it can change disease-related biology tied to GBA1. GT-02287 is an experimental oral small molecule designed to act on the GBA1-related pathway, with the aim of improving glucocerebrosidase function and lysosomal clearance so cells handle alpha-synuclein better; researchers will measure drug levels and biological effects in blood and spinal fluid. People can stay on stable Parkinson's meds including levodopa, since GT-02287 is being tested alongside existing treatments rather than as a replacement. The trial is enrolling adults 30 to 85 years old, within seven years of diagnosis, with early to moderate PD (Hoehn and Yahr 1,3) who are willing to have GBA1 genetic testing and procedures like blood draws and lumbar puncture.
Locations
- 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
Frequently Asked Questions
- What is this trial testing?
- This trial is studying GT-02287. The goal is to check safety, tolerability, and how GT-02287 behaves in the body while looking for signs it can change disease-related biology tied to GBA1. GT-02287 is an experimental oral small molecule designed to act on the GBA1-related pathway, with the aim of improving glucocerebrosidase function and lysosomal clearance so cells handle alpha-synuclein better; researchers will measure drug levels and biological effects in blood and spinal fluid. People can stay on stable Parkinson's meds including levodopa, since GT-02287 is being tested alongside existing treatments rather than as a replacement. The trial is enrolling adults 30 to 85 years old, within seven years of diagnosis, with early to moderate PD (Hoehn and Yahr 1,3) who are willing to have GBA1 genetic testing and procedures like blood draws and lumbar puncture.
- Who can participate?
- Participants must be between 30 Years and 85 Years.
- Where is this trial located?
- This trial is recruiting at 7 locations.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This Phase 1 trial is estimated to last approximately 9 months.