NCT07280299

Efficacy, Safety, Tolerability, and Biomarker Effects of GT-02287 in Early Parkinson's Disease

A Randomized, Placebo-controlled, Double-blind, Phase 2a Study to Evaluate the Clinical Efficacy, Safety, Tolerability, and Biomarker Effects of 2 Dose Levels of GT-02287 in Participants With Early Parkinson's Disease

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 111 (estimated)
• Sponsor: Gain Therapeutics, Inc. · Industry
• Sex: All
• Age: 30 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the efficacy, the safety, and the effect on biomarkers of 2 dose levels of oral GT-02287 over placebo after 48 weeks of treatment in treated and untreated participants with early PD.

Detailed description

This is a 48-week, double-blind, randomized, placebo-controlled Phase 2a study testing two doses of oral GT-02287 in people with early Parkinson's disease (PD), both treated and untreated. The study has three parts: * Screening Period lasting up to 45 days * Treatment Period lasting about 341 days * Follow-up Period lasting up to 33 days Participants will have 7 onsite visits for efficacy, safety, tolerability, and biomarker assessments (Week 4, Week 8, Week 12, Week 24, Week 36, Week 48, and a Follow-Up Visit at Week 52). In addition, there will be 5 additional visits for laboratory blood tests (Weeks 2, 16, 20, 30, and 42). Approximately 111 participants will be randomized into three groups (high dose, low dose, placebo). Participants can have idiopathic PD or be heterozygous for a pathogenic variant in the GBA1 gene. Participants who have other PD-associated genetic variants (e.g., leucine rich repeat kinase 2 \[LRRK2\]) are ineligible. All participants will be genotyped to determine their PD-associated genetic status before enrollment. At the start, participants undergo screening and baseline tests, including motor assessments, quality of life, sensor measurements, and collection of fluid and blood samples for biomarkers. Some baseline tests may be done just before dosing. The study will measure efficacy through various motor, quality of life, disease progression, non-motor and other symptoms of PD, and cognitive tests, along with wearable sensor data. Safety will be assessed by recording adverse events, lab tests, vital signs, body weight, heart monitoring, and questionnaires. The study will also analyze GT-02287 levels in blood and spinal fluid, and its effects on biomarkers.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DRUG	Low Dose GT-02287	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.
DRUG	High Dose GT-02287	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.
DRUG	Placebo	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.

Timeline

• Start date: 2026-05-30
• Primary completion: 2028-06-30
• Completion: 2028-06-30
• First posted: 2025-12-12
• Last updated: 2025-12-12

Regulatory

• FDA-regulated drug study