Tests the safety of GT-02287 in people with Parkinson's disease
Tests GT-02287, a daily drug, in adults with Parkinson's disease to check safety.
Plain-language title generated from the registry record
Registry title: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
Key facts
- Where
- Adelaide, Darlinghurst, Kogarah, Melbourne, Parkville, Westmead, and Woolloongabba, AustraliaSee the 7 sites
- Phase
- Phase 1
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with Parkinson's disease, within 7 years of diagnosis, with or without a GBA1 gene change. Everyone takes GT-02287 once a day for 90 days.
- Age
- 30 to 85 years
- Sex
- All sexes can join.
- Disease stage
- Parkinson's disease diagnosed by MDS criteria, within 7 years of diagnosis, Hoehn & Yahr stage 1 to 3, no severe motor fluctuations or disabling dyskinesias.
- Earlier treatment
- Never treated for Parkinson's disease, or on stable Parkinson's medication for at least 3 months, with the same dose for at least 4 weeks before screening.
- Biomarker or test result
- Willing to give a blood sample for Parkinson's-related genetic testing. If a GBA1 mutation is known, it must be one linked to a higher risk of Parkinson's disease.
Main reasons for not taking part
- Other brain or nerve diseases, such as Alzheimer's disease or multiple system atrophy
- Gaucher disease, or GBA1 changes linked to Gaucher disease
- Known LRRK2 change or other Parkinson's-related gene changes besides GBA1
- Dementia or moderate memory and thinking problems (score 17 or higher on the Montreal Cognitive Assessment)
- Antipsychotic or anticholinergic medicines, or being pregnant or breastfeeding
Read by machine on 9 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 30 and 85 years old?
Source: NCT06732180, who can take part
Where in Australia
- CMAXAdelaide, AustraliaTaking people
- St Vincent's Hospital SydneyDarlinghurst, AustraliaTaking people
- Southern NeurologyKogarah, AustraliaTaking people
- Alfred HealthMelbourne, AustraliaTaking people
- Royal Melbourne HospitalParkville, AustraliaTaking people
- Westmead HospitalWestmead, AustraliaTaking people
- Princess Alexandra HospitalWoolloongabba, AustraliaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Gain Therapeutics, Inc.
- Trial code
- NCT06732180
- Record updated
- 6 May 2025
- Original description
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:Read full original descriptionShow less
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
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).
Inclusion: Able and willing to provide written informed consent and be willing to comply with the requirements and restrictions of the study; Any sex, ≥30 and ≤85 years of age; Diagnosis of PD based on MDS criteria; Within 7 years of PD diagnosis; Body mass index of ≥18 and ≤40 kg/m2, and a body weight ≥45 kg and ≤120 kg; Willing to provide a blood sample for PD-related genetic testing; Hoehn \& Yahr 1-3, inclusive; No severe motor fluctuations or disabling dyskinesias based on the investigator's clinical assessment; Naïve to pharmacological treatment for PD or on stable PD medication for ≥3 months prior to Screening, including ≥4 weeks at the same dose(s) immediately before Screening; Not pregnant or breastfeeding; If participant is either of childbearing potential or produces potentially viable sperm, participant must agree to use 2 forms of contraception (barrier method and a second highly effective form of birth control/contraception, as defined in the protocol) if engaging in potentially reproductive intercourse (with a partner who produces potentially viable sperm or is of childbearing potential, respectively); Agreeing to not participate in another investigational study while taking part in this study; For participants with known GBA1 mutations, presence of a GBA1 mutation that has been associated with an increased risk of PD.
Exclusion: Concomitant medications metabolized primarily by cytochrome P450 3A4 (CYP3A4) that have a narrow therapeutic window, concomitant medications that are substrates of breast cancer resistance protein and/or P-glycoprotein and that have a narrow therapeutic window, concomitant medications that are potent inhibitors or inducers of CYP3A4; Use of dopamine antagonists (antipsychotics) or anticholinergic medications; Concomitant disease including, but not limited to cardiovascular conditions, diabetes, autoimmune disease, cancer, active infectious disease, psychotic disorders and symptoms, depressive symptoms, drug and/or alcohol misuse as defined in the protocol; participation in any interventional clinical study within 3 months or 5 half-lives, whichever is longer, prior to Screening; Other neurological disorders, including but not limited to Alzheimer's disease, amyotrophic lateral sclerosis, frontotemporal dementia, progressive supranuclear palsy, corticobasal syndrome, Huntington's disease, multiple system atrophy, dementia with Lewy bodies, secondary (e.g. drug-induced) parkinsonism, multiple sclerosis, or epilepsy; A history of Gaucher disease or homozygous for a GBA1 pathogenic variant known to be associated with GD or compound heterozygous for 2 alleles that are known to be associated with GD; Dementia or a moderate cognitive impairment (score ≥17 on the Montreal Cognitive Assessment); Hypersensitivity to GT-02287 or any of its excipients; Malabsorption or relevant disorder which may impact the absorption of GT-02287; Clinically significant abnormalities in laboratory test; Known PD-associated LRRK2 pathogenic variant or other PD-associated genetic mutations other than GBA1; Contraindications to lumbar puncture (LP); Blood donation >500 mL within 3 months; Unable to comply with restrictions on food products, smoking, and /or alcohol use as defined in protocol.