Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 1INTERVENTIONAL

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

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

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease (NCT06732180) is a Phase 1 interventional studying Parkinson Disease, sponsored by Gain Therapeutics, Inc.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

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).

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For Parkinson Disease, a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 20 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - Able and willing to provide written willing to sign a consent form 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 Who Should NOT Join This Trial: - 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * 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 Criteria: * 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. * Known PD-associated LRRK2 pathogenic variant or other PD-associated genetic mutations other than GBA1 * Dementia or a moderate cognitive impairment (score ≥17 on the Montreal Cognitive Assessment) * Hypersensitivity to GT-02287 or any of its excipients * 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 * Malabsorption or relevant disorder which may impact the absorption of GT-02287 * Clinically significant abnormalities in laboratory test * 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 * participation in any interventional clinical study within 3 months or 5 half-lives, whichever is longer, prior to Screening

Treatments Being Tested

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)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06732180), the sponsor (Gain Therapeutics, Inc.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06732180 clinical trial studying?

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 onc… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06732180?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06732180?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06732180. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06732180. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.