Evaluating the safety and effects 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

This study is testing a new drug called GT-02287 to see if it is safe and helps people with Parkinson's Disease feel better.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 30 Years to 85 Years
• Sex: All
• Sponsor: Gain Therapeutics, Inc. (Industry-sponsored)
• Locations: 7 sites (Darlinghurst, New South Wales and 6 other locations)
• Trial ID: NCT06732180 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and tolerability of the drug GT-02287 in individuals diagnosed with Parkinson's Disease. Participants will undergo a 30-day screening period to determine eligibility, followed by a 90-day treatment phase where they will receive daily doses of GT-02287. The study will also investigate how the drug is processed in the body and its potential biological effects in blood and cerebrospinal fluid. Participants will have regular clinic visits to monitor their health and response to the treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 30 to 85 with a diagnosis of Parkinson's Disease within the last 7 years and who meet specific health criteria.

Not a fit: Patients with severe motor fluctuations or those who have been previously treated with Parkinson's medications may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option that improves the management of Parkinson's Disease.

How similar studies have performed: While this approach is novel, similar studies targeting pharmacological interventions in Parkinson's Disease have shown promise in the past.

Eligibility criteria

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

Where this trial is running

Darlinghurst, New South Wales and 6 other locations

• St Vincent's Hospital Sydney — Darlinghurst, New South Wales, Australia (Recruiting)
• Southern Neurology — Kogarah, New South Wales, Australia (Recruiting)
• Westmead Hospital — Westmead, New South Wales, Australia (Recruiting)
• Princess Alexandra Hospital — Woolloongabba, Queensland, Australia (Recruiting)
• Cmax — Adelaide, South Australia, Australia (Recruiting)
• Alfred Health — Melbourne, Victoria, Australia (Recruiting)
• Royal Melbourne Hospital — Parkville, Victoria, Australia (Recruiting)

Study contacts

• Study coordinator: Gain Therapeutics Clinical Operations

Conditions Parkinson Disease