GT-02287 for Parkinson's (NCT06732180)
Test safety of new medication
- 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
- Test safety of new medication
- Phase
- PHASE1
- Status
- RECRUITING
- Sponsor
- Gain Therapeutics, Inc.
- Study Type
- INTERVENTIONAL
- Enrollment
- 20 participants
- Conditions
- Parkinson Disease
- Interventions
- GT-02287
Summary For Families
This study is testing the safety and tolerability of a new oral medication, and will also look at how the body handles it and whether it causes any biological changes in blood and spinal fluid that might be helpful for people with Parkinson's. Participants will take the medication once a day for 90 days at a weight-based dose of about 13.5 milligrams per kilogram per day, with regular clinic visits, blood tests, and a spinal fluid sample via lumbar puncture to check drug levels and effects. Adults 30 to 85 years old who were diagnosed within the last 7 years and are in early to mid-stage Parkinson's (stages 1 to 3) may be eligible, if they do not have dementia, are not taking certain excluded medications, and are either new to Parkinson's drugs or have been on stable doses for at least 3 months. About 20 people will be enrolled, and the study welcomes people with or without certain GBA1 genetic changes but excludes those with Gaucher disease or specific GBA1 variants tied to that condition.
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. This study is testing the safety and tolerability of a new oral medication, and will also look at how the body handles it and whether it causes any biological changes in blood and spinal fluid that might be helpful for people with Parkinson's. Participants will take the medication once a day for 90 days at a weight-based dose of about 13.5 milligrams per kilogram per day, with regular clinic visits, blood tests, and a spinal fluid sample via lumbar puncture to check drug levels and effects. Adults 30 to 85 years old who were diagnosed within the last 7 years and are in early to mid-stage Parkinson's (stages 1 to 3) may be eligible, if they do not have dementia, are not taking certain excluded medications, and are either new to Parkinson's drugs or have been on stable doses for at least 3 months. About 20 people will be enrolled, and the study welcomes people with or without certain GBA1 genetic changes but excludes those with Gaucher disease or specific GBA1 variants tied to that condition.
- 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?
- You are never charged a fee to join a legitimate clinical trial, and the study treatment and study-specific tests are provided by the trial sponsor at no charge. In the US, routine care you would receive anyway can still be billed to you or your insurance; coverage rules differ by country, and trials in the UK and Canada usually run through public health systems. Travel, parking, and time are real costs, and some trials reimburse them while others do not. Before enrolling, ask the study coordinator to explain in writing what is and is not covered.
- How long does the trial last?
- This Phase 1 trial is estimated to last approximately 9 months.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.
- Will I get a placebo in this trial?
- Everyone in this trial receives the experimental treatment. There is no placebo group.
- How many visits does this trial involve?
- You will have clinic visits during a 30-day Screening Period, during a 90-day Treatment Period (with visits on the first day, after 2 weeks, and every month), and a 14-day Follow-Up Period.
- What procedures are involved in this trial?
- Based on the protocol, this trial involves: Lumbar puncture (spinal tap) (invasive). Confirm the full schedule with the study coordinator.