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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:
Participants will:
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| single-arm. All participants receive the active molecule (once daily GT-02287 administration) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GT-02287 | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence, nature, relationship to investigational product (IP), and severity of adverse events (AEs) | 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) | From first dose to Day 105 |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Plasma Concentration [Cmax] | Pharmacokinetics | Day 1 and Day 90 |
| Area under the curve [AUC] | Pharmacokinetics | Day 1 and Day 90 |
| Measure | Description | Time Frame |
|---|---|---|
| Gcase activity (µmol/L/h) measurement in dried blood spots (DBS) | Pharmacodynamics (Exploratory outcome): mean change from baseline in Gcase activity in DBS | From first dose to Day 90 |
| Concentration of sphingolipids in cerebrospinal fluid (CSF ) |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Gain Therapeutics Clinical Operations | Contact | +41919211131 | info@gaintherapeutics.com |
| Name | Affiliation | Role |
|---|---|---|
| Chief Medical Officer | Gain Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| St Vincent's Hospital Sydney | Recruiting | Darlinghurst | New South Wales | Australia |
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| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| Time to reach Cmax [Tmax] | Pharmacokinetics | Day 1 and Day 90 |
| Elimination half life [T1/2] | Pharmacokinetics | Day 90- Day 93 |
| Apparent Clearance (CL/F) | Pharmacokinetics | Day 90- Day 93 |
| Apparent Volume of Distribution (Vz/F) | Pharmacokinetics | Day1 and Day 90- Day 93 |
Pharmacodynamics (Exploratory outcome): mean change from baseline in sphingolipids in CSF
| From first dose to Day 90 |
| Southern Neurology | Recruiting | Kogarah | New South Wales | Australia |
|
| Westmead Hospital | Recruiting | Westmead | New South Wales | Australia |
|
| Princess Alexandra Hospital | Recruiting | Woolloongabba | Queensland | Australia |
|
| CMAX | Recruiting | Adelaide | South Australia | Australia |
|
| Alfred Health | Recruiting | Melbourne | Victoria | Australia |
|
| Royal Melbourne Hospital | Recruiting | Parkville | Victoria | Australia |
|
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |