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Also known as GT-02287
Low Dose GT-02287 · 2 trials · 1 indication
Change from Baseline to Week 48 in the sum of the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II score and Part III score assessed in the practically defined OFF state. MDS-UPDRS is a multimodal scale assessing impairment and disability consisting of four parts. Part II assesses motor experiences of daily living (Range 0-52). It contains 13 questions which are to be rated by the patient and/or caregiver. Part III assesses the motor signs of PD and is rated by the investigator (Range 0-132). Part III contains 33 scores based on 18 items. For each question a numeric score is assigned between 0-4, where 0 = Normal, 1 = Slight, 2 = Mild, 3 = Moderate, 4 = Severe. A higher score indicates more severe symptoms of PD.
| Arm | Type | Description |
|---|---|---|
| Low Dose GT-02287 | ACTIVE_COMPARATOR | GT-02287 400 or 600 mg/day |
| High Dose GT-02287 | ACTIVE_COMPARATOR | GT-02287 800 or 1000 mg/day |
| Placebo | PLACEBO_COMPARATOR | Magnesium aluminometasilicate (MAS) |
| single-arm. All participants receive the active molecule (once daily GT-02287 administration) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Low Dose GT-02287 | DRUG | Sachets containing 400 mg/day or 600 mg/day of active (GT-02287), depending on the participant's body weight. Each daily dose will be prepared as an oral suspension by mixing the content of 1 sachet with the vehicle (supplied in an amber glass bottle) and the requisite volume of tap water. |
| High Dose GT-02287 | DRUG | Sachets containing 800 mg/day or 1000 mg/day of active (GT-02287), depending on the participant's body weight. Each daily dose will be prepared as an oral suspension by mixing the content of 1 sachet with the vehicle (supplied in an amber glass bottle) and the requisite volume of tap water. |
| Placebo | DRUG | Sachets containing 420 to 1200 mg of MAS indistinguishable from the active sachets. Each daily dose will be prepared as an oral suspension by mixing the content of 1 sachet with the vehicle (supplied in an amber glass bottle) and the requisite volume of tap water. |
| 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 |
Inclusion Criteria: * Able and willing to provide written informed consent and willing to comply with the requirements and restrictions of the study * Willing to undergo PD-related genetic testing and analysis * Any sex, ≥30 and ≤85 years of age * Body mass index of ≥18 and ≤40 kg/m2 and a body wei...
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Low Dose GT-02287 is an investigational small molecule being developed for the treatment of Parkinson's disease. It is currently in clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with Parkinson's disease to evaluate its safety, tolerability, pharmacokinetics, pharmacodynamics, efficacy, and biomarker effects.
GT-02287 targets GCase, a lysosomal enzyme. By modulating this enzyme, the drug aims to address a potential underlying mechanism in Parkinson's disease. The therapeutic approach focuses on enhancing lysosomal function, which may be relevant to the disease pathology. This mechanism is being investigated in clinical trials for its potential benefit in Parkinson's disease.
GT-02287 is being developed by Gain Therapeutics, Inc., a biopharmaceutical company listed on the stock exchange under the ticker symbol GANX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in Parkinson's disease. Gain Therapeutics is responsible for the drug's development program and regulatory strategy.
GT-02287 is in Phase 2 clinical development for Parkinson's disease. The drug is currently being studied in a Phase 1 trial and a Phase 2 trial. The Phase 2 trial is designed to assess efficacy, safety, tolerability, and biomarker effects in early Parkinson's disease. The drug remains investigational and is not yet approved.
GT-02287 is being evaluated in two clinical trials. The first is NCT06732180, a Phase 1 study in Australia assessing safety, tolerability, pharmacokinetics, and pharmacodynamics in Parkinson's disease, with 20 participants. The second is NCT07280299, a Phase 2 trial in early Parkinson's disease evaluating efficacy, safety, tolerability, and biomarkers, with 111 participants.
Yes, Low Dose GT-02287 is the same drug as GT-02287. The name "Low Dose GT-02287" refers to the same investigational compound, GT-02287, developed by Gain Therapeutics. The term "Low Dose" may indicate a specific dosage form or strength being studied, but the active pharmaceutical ingredient is identical.