Parkinson DiseasePhase 1NCT06732180
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
A Phase 1 study testing GT-02287 in people with Parkinson Disease, run by Gain Therapeutics, Inc. at 7 sites in Australia.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 30 to 85 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- GT-02287
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 7 sitesAustralia
- Study dates
- 21 Feb 2025to 30 Nov 2025
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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 cannot join
- 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
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- 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
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
7 sites in 1 country
Australia7 sites, 7 recruiting
- Recruiting
CMAX
Adelaide, South Australia
CMAX, 1800 150 433, PET@cmax.com.au
- Recruiting
St Vincent's Hospital Sydney
Darlinghurst, New South Wales
Umesh Tulachan, umesh.tulachan@svha.org.au
Valerie Bramah, + 61 2 8382 4977, valerie.bramah@svha.org.au
- Recruiting
Southern Neurology
Kogarah, New South Wales
Elena Simon, +61 408455183, elena.simon@southern-neurology.com.au
- Recruiting
Alfred Health
Melbourne, Victoria
Susan Rose, +61 3 9903 9402, susan.rose@alfred.org.au
Beth Sutherland, + 61 3 9903 9402, b.sutherland@alfred.org.au
- Recruiting
Royal Melbourne Hospital
Parkville, Victoria
Reena Chopra, + 61 3 9342 8182, reena.chopra@mh.org.au
Rebecca Ravenhill, + 61 3 9342 8182, Rebecca.Ravenhill@mh.org.au
- Recruiting
Westmead Hospital
Westmead, New South Wales
Sarah Bray, + 61 28890 6793, sarah.bray@health.nsw.gov.au
Cassie Chen, + 61 438 678 797, xi.chen2@health.nsw.gov.au
- Recruiting
Princess Alexandra Hospital
Woolloongabba, Queensland
Schible Kurian, +61 436447386, schible.kurian@health.qld.gov.au
About this study
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
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?
Read more
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).
Read the detailed description
This Phase 1b, 3-month, multicenter, open-label study of GT-02287 in participants with Parkinson's Disease with or without GBA1 mutations. The primary objective of the study is to evaluate the safety and tolerability of GT-02287. The study will also investigate pharmacokinetics (PK) and explore pharmacodynamic parameters in blood and CSF and effect of GT-02287 on scores from selected clinical scales. Approximately 20 participants will be enrolled to result in approximately 15 evaluable participants.
All participants will receive an oral dose of GT-02287 of 13.5 mg/kg/day (plus/minus 2 mg/kg/day) once a day for 90 days.
- Conditions
- Parkinson Disease
- Being tested
- GT-02287
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GT-02287Drug
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
| Study group | Role | What they receive |
|---|---|---|
| single-arm. All participants receive the active molecule (once daily GT-02287 administration) | Experimental | Not described Receives: Drug: GT-02287 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, nature, relationship to investigational product (IP), and severity of adverse events (AEs)
Measured: From first dose to Day 105
Incidence of clinically significant findings for clinical laboratory evaluations, physical and neurological examinations, body weight , vital signs measurements, 12-lead o 12-lead electrocardiograms (ECGs), Columbia Suicide Severity Rating Scale (C-SSRS)
Measured: From first dose to Day 105
Secondary outcomes (6)
Maximum Plasma Concentration [Cmax]
Pharmacokinetics
Measured: Day 1 and Day 90
Area under the curve [AUC]
Pharmacokinetics
Measured: Day 1 and Day 90
Time to reach Cmax [Tmax]
Pharmacokinetics
Measured: Day 1 and Day 90
Elimination half life [T1/2]
Pharmacokinetics
Measured: Day 90- Day 93
Apparent Clearance (CL/F)
Pharmacokinetics
Measured: Day 90- Day 93
Apparent Volume of Distribution (Vz/F)
Pharmacokinetics
Measured: Day1 and Day 90- Day 93
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2024
- Study start21 Feb 2025
- Primary completion30 Nov 2025 (estimated)
- Study completion30 Nov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gain Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Medical Officer, Study director, Gain Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06732180
- Sponsor's protocol ID
- GANX-001-V102
- Status last verified
- May 2025
- First submitted
- 9 Dec 2024
- Last updated
- 6 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06732180
- Acronym / sponsor ID
- GANX-001-V102
- Conditions
- Parkinson Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Gain Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Feb 2025 (actual)
- Primary completion
- 30 Nov 2025 (estimated)
- Study completion
- 30 Nov 2025 (estimated)
- First posted
- 13 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
This Phase 1b, 3-month, multicenter, open-label study of GT-02287 in participants with Parkinson's Disease with or without GBA1 mutations. The primary objective of the study is to evaluate the safety and tolerability of GT-02287. The study will also investigate pharmacokinetics (PK) and explore pharmacodynamic parameters in blood and CSF and effect of GT-02287 on scores from selected clinical scales. Approximately 20 participants will be enrolled to result in approximately 15 evaluable participants.
All participants will receive an oral dose of GT-02287 of 13.5 mg/kg/day (plus/minus 2 mg/kg/day) once a day for 90 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single-arm. All participants receive the active molecule (once daily GT-02287 administration) | Experimental | Drug: GT-02287 |
Who may qualify
- Ages
- 30 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GT-02287Dose 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 | Drug | single-arm. All participants receive the active molecule (once daily GT-02287 administration) |
Primary outcome
Incidence, nature, relationship to investigational product (IP), and severity of adverse events (AEs)
Time frame: From first dose to Day 105
Incidence of clinically significant findings for clinical laboratory evaluations, physical and neurological examinations, body weight , vital signs measurements, 12-lead o 12-lead electrocardiograms (ECGs), Columbia Suicide Severity Rating Scale (C-SSRS)
Time frame: From first dose to Day 105
6 secondary outcomes
Maximum Plasma Concentration [Cmax]
Time frame: Day 1 and Day 90
Area under the curve [AUC]
Time frame: Day 1 and Day 90
Time to reach Cmax [Tmax]
Time frame: Day 1 and Day 90
Elimination half life [T1/2]
Time frame: Day 90- Day 93
Apparent Clearance (CL/F)
Time frame: Day 90- Day 93
Apparent Volume of Distribution (Vz/F)
Time frame: Day1 and Day 90- Day 93
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chief Medical Officer study director
Gain Therapeutics, Inc.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GT-02287 in people with Parkinson Disease, run by Gain Therapeutics, Inc. at 7 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Gain Therapeutics Clinical Operations (+41919211131, info@gaintherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence, nature, relationship to investigational product (IP), and severity of adverse events (AEs) (time frame: From first dose to Day 105). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2025.