Actively Recruiting
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
Led by Gain Therapeutics, Inc. · Updated on 2025-05-06
20
Participants Needed
7
Research Sites
40 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able and willing to provide written informed consent and comply with study requirements
- Any sex, aged 30 to 85 years
- Diagnosis of Parkinson's Disease based on MDS criteria
- Parkinson's Disease diagnosed within the past 7 years
- Body mass index between 18 and 40 kg/m2
- Body weight between 45 kg and 120 kg
- Willing to provide blood sample for Parkinson's Disease-related genetic testing
- Hoehn & Yahr stage 1 to 3 inclusive
- No severe motor fluctuations or disabling dyskinesias per investigator
- Either untreated for Parkinson's Disease or on stable medication for at least 3 months before screening
- Not pregnant or breastfeeding
- Agree to use two forms of contraception if of childbearing potential or producing viable sperm
- Agree not to participate in another investigational study during this study
- For those with known GBA1 mutations, presence of a mutation associated with increased Parkinson's Disease risk
You will not qualify if you...
- Other neurological disorders such as Alzheimer's disease, amyotrophic lateral sclerosis, frontotemporal dementia, progressive supranuclear palsy, corticobasal syndrome, Huntington's disease, multiple system atrophy, dementia with Lewy bodies, secondary parkinsonism, multiple sclerosis, or epilepsy
- History of Gaucher disease or homozygous/compound heterozygous GBA1 variants linked to Gaucher disease
- Known Parkinson's Disease-associated LRRK2 or other genetic mutations aside from GBA1
- Dementia or moderate cognitive impairment (Montreal Cognitive Assessment score ≤17)
- Hypersensitivity to GT-02287 or its ingredients
- Use of medications primarily metabolized by CYP3A4 with narrow therapeutic window or inhibitors/inducers of CYP3A4
- Use of dopamine antagonists or anticholinergic medications
- Concomitant diseases including cardiovascular conditions, diabetes, autoimmune disease, cancer, active infections, psychotic or depressive symptoms, or substance misuse
- Malabsorption or disorders affecting GT-02287 absorption
- Clinically significant laboratory abnormalities
- Contraindications to lumbar puncture
- Blood donation over 500 mL within 3 months
- Unable to follow restrictions on food, smoking, or alcohol use per protocol
- Participation in another interventional clinical study within 3 months or 5 half-lives before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
St Vincent's Hospital Sydney
Darlinghurst, New South Wales, Australia
Actively Recruiting
2
Southern Neurology
Kogarah, New South Wales, Australia
Actively Recruiting
3
Westmead Hospital
Westmead, New South Wales, Australia
Actively Recruiting
4
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
Actively Recruiting
5
CMAX
Adelaide, South Australia, Australia
Actively Recruiting
6
Alfred Health
Melbourne, Victoria, Australia
Actively Recruiting
7
Royal Melbourne Hospital
Parkville, Victoria, Australia
Actively Recruiting
Research Team
G
Gain Therapeutics Clinical Operations
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here