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Also known as Low Dose GT-02287
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...
Low Dose GT-02287 is an investigational small molecule being developed for the treatment of Parkinson Disease. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities. The drug is being studied for its safety, tolerability, and potential efficacy in patients with this condition.
GT-02287 targets GCase, a lysosomal enzyme. By modulating this enzyme, the drug aims to address a key pathway implicated in Parkinson Disease. This mechanism is being evaluated in clinical trials to determine its therapeutic potential for patients with the condition.
GT-02287 is being developed by Gain Therapeutics, Inc., a biopharmaceutical company traded 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 Disease.
GT-02287 is in Phase 2 clinical development for Parkinson Disease. It is an investigational drug, meaning it has not yet received regulatory approval. The Phase 2 trial is designed to assess efficacy, safety, tolerability, and biomarker effects in patients with early Parkinson Disease.
GT-02287 is being studied in two clinical trials. The first, NCT06732180, is a Phase 1 study in Australia evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics in 20 patients with Parkinson Disease. The second, NCT07280299, is a Phase 2 trial in 111 patients with early Parkinson Disease, assessing efficacy, safety, tolerability, and biomarker effects.
Yes, Low Dose GT-02287 is the same drug as GT-02287. The name 'Low Dose' refers to a specific dosage form or regimen being studied, but the active pharmaceutical ingredient is identical. Both names refer to the same investigational compound developed by Gain Therapeutics.