--- title: New drug slows Parkinson progression nct_id: NCT06732180 phase: PHASE1 status: RECRUITING sponsor: Gain Therapeutics, Inc. study_type: INTERVENTIONAL canonical_url: "https://parkinsonspathways.com/trial/NCT06732180" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT06732180" last_fetched: "2026-05-10T14:05:07.116Z" source: "Parkinson's Pathways (curated)" --- # New drug slows Parkinson progression **Goal (in five words):** New drug slows Parkinson progression **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 **Trial ID:** [NCT06732180](https://clinicaltrials.gov/study/NCT06732180) ## Key Facts - **Phase:** PHASE1 - **Status:** RECRUITING - **Study Type:** INTERVENTIONAL - **Sponsor:** Gain Therapeutics, Inc. - **Target Enrollment:** 20 participants - **Start Date:** 2025-02-21 - **Completion Date:** 2025-11-30 - **Conditions:** Parkinson Disease - **Interventions:** GT-02287 - **Intervention Types:** DRUG ## Summary For Families The goal is to check safety, tolerability, and how GT-02287 behaves in the body while looking for signs it can change disease-related biology tied to GBA1. GT-02287 is an experimental oral small molecule designed to act on the GBA1-related pathway, with the aim of improving glucocerebrosidase function and lysosomal clearance so cells handle alpha-synuclein better; researchers will measure drug levels and biological effects in blood and spinal fluid. People can stay on stable Parkinson's meds including levodopa, since GT-02287 is being tested alongside existing treatments rather than as a replacement. The trial is enrolling adults 30 to 85 years old, within seven years of diagnosis, with early to moderate PD (Hoehn and Yahr 1,3) who are willing to have GBA1 genetic testing and procedures like blood draws and lumbar puncture. ## Eligibility - **Minimum age:** 30 Years - **Maximum age:** 85 Years - **Sex:** ALL ### Full 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 ``` ## Locations (7) - St Vincent's Hospital Sydney, Darlinghurst, New South Wales, Australia _(-33.8794, 151.2192)_ - Umesh Tulachan — (CONTACT) — umesh.tulachan@svha.org.au - Valerie Bramah — (CONTACT) — + 61 2 8382 4977 — valerie.bramah@svha.org.au - Southern Neurology, Kogarah, New South Wales, Australia _(-33.9681, 151.1356)_ - Elena Simon — (CONTACT) — +61 408455183 — elena.simon@southern-neurology.com.au - Westmead Hospital, Westmead, New South Wales, Australia _(-33.8038, 150.9877)_ - Sarah Bray — (CONTACT) — + 61 28890 6793 — sarah.bray@health.nsw.gov.au - Cassie Chen — (CONTACT) — + 61 438 678 797 — xi.chen2@health.nsw.gov.au - Princess Alexandra Hospital, Woolloongabba, Queensland, Australia _(-27.4886, 153.0366)_ - Schible Kurian — (CONTACT) — +61 436447386 — schible.kurian@health.qld.gov.au - CMAX, Adelaide, South Australia, Australia _(-34.9287, 138.5986)_ - CMAX — (CONTACT) — 1800 150 433 — PET@cmax.com.au - Alfred Health, Melbourne, Victoria, Australia _(-37.8140, 144.9633)_ - Susan Rose — (CONTACT) — +61 3 9903 9402 — susan.rose@alfred.org.au - Beth Sutherland — (CONTACT) — + 61 3 9903 9402 — b.sutherland@alfred.org.au - Royal Melbourne Hospital, Parkville, Victoria, Australia _(-37.7833, 144.9500)_ - Reena Chopra — (CONTACT) — + 61 3 9342 8182 — reena.chopra@mh.org.au - Rebecca Ravenhill — (CONTACT) — + 61 3 9342 8182 — Rebecca.Ravenhill@mh.org.au ## Central Contacts - Gain Therapeutics Clinical Operations — (CONTACT) — +41919211131 — info@gaintherapeutics.com --- *Canonical: https://parkinsonspathways.com/trial/NCT06732180* *HTML version: https://parkinsonspathways.com/trial/NCT06732180* *Source data: https://clinicaltrials.gov/study/NCT06732180*