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

NCT07280299 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: GANX-001-V103
• Study Type: interventional
• Target: 111
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (1)

• 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 [Efficacy]

  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.

  From baseline to Week 48

Secondary Outcomes (8)

• Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]

  From baseline to Week 48

• Change from Baseline to Week 48 in the MDS-UPDRS Part II score [Effect on motor function]

  From baseline to Week 48

• Change from Baseline to Week 48 in the MDS-UPDRS Part III score [Effect on motor function]

  From baseline to Week 48

• Change from Baseline to Week 48 in the Timed Up and Go (TUG) test [Effect on motor function]

  From baseline to Week 48

• Change from Baseline to Week 48 in the Parkinson's Disease Questionnaire 39 (PDQ-39) score [Effect on quality of life]

  From baseline to Week 48

Other Outcomes (12)

• Change from Baseline to Week 48 in the MDS-UPDRS Part I score [Effect on non-motor symptoms of PD]

  From baseline to Week 48

• Change from Baseline to Week 48 in the Cognitive Summary Score (CSS) [Effect on non-motor symptoms of PD]

  From baseline to Week 48

• Change from Baseline to Week 48 in the Purdue Pegboard Test (PPT) total score

  From baseline to Week 48

Study Arms (3)

Low Dose GT-02287

ACTIVE COMPARATOR

GT-02287 400 or 600 mg/day

Drug: Low Dose GT-02287

High Dose GT-02287

ACTIVE COMPARATOR

GT-02287 800 or 1000 mg/day

Drug: High Dose GT-02287

Placebo

PLACEBO COMPARATOR

Magnesium aluminometasilicate (MAS)

Drug: Placebo

Interventions

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.

Also known as: GT-02287 400 or 600 mg/day

Low Dose GT-02287

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.

Also known as: GT-02287 800 or 1000 mg/day

High Dose GT-02287

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.

Also known as: Magnesium aluminometasilicate (MAS)

Placebo

Eligibility Criteria

• Age: 30 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 weight of at ≥55 kg and \<120 kg at Screening
• Diagnosis of PD based on MDS criteria
• Within 5 years of PD diagnosis
• Positive SAA in CSF at Baseline
• Hoehn \& Yahr 1-2.5, inclusive
• Naïve to pharmacological treatment for PD with no initiation of dopaminergic treatment expected during the first 9 months of the study or on stable PD medication for ≥3 months prior to Screening, including ≥4 weeks at the same dose(s) immediately before Screening with no changes to dose(s), or medication(s) expected during the first 9 months of the study
• 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 not to participate in another investigational study while taking part in this study

You may not qualify if:

• 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
• PD-associated LRRK2 pathogenic variant or other PD-associated genetic mutations other than GBA1
• Severe motor fluctuations and/or disabling dyskinesias based on the investigator's clinical assessment
• Deep-brain stimulation
• Hallucinations, delusions, or other psychotic symptoms requiring antipsychotic medication Use of dopamine antagonists (antipsychotics) or anticholinergic medications
• Dementia by clinical diagnosis and/or a MoCA score of ≤20 and/or a history of behavioral impairment
• Hypersensitivity to GT 02287 or any of its excipients
• Concomitant medications including drugs metabolized primarily by CYP3A4 that have a narrow therapeutic window, substrates of BCRP that have a narrow therapeutic window, strong or moderate inhibitors or strong inducers of CYP3A4 that could affect the metabolism and plasma levels of GT 02287, including herbal supplements and certain foods.
• 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
• Clinically significant abnormalities in laboratory test
• Contraindications to lumbar puncture (LP) including current treatment with anticoagulants or any other contraindications that might preclude safe completion of the LP
• Blood donation \>500 mL within 3 months
• Malabsorption or relevant disorder which may impact the absorption of GT-02287
• Participation in any interventional clinical study within 3 months or 5 half-lives, whichever is longer, prior to Screening

Sponsors & Collaborators

• Gain Therapeutics, Inc.: lead

MeSH Terms

Conditions

Parkinson Disease

Interventions

licorice extract - magnesium aluminometasilicate

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Jonas Hannestad, CMO

  Gain Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

Gain Therapeutics Clinical Operations

CONTACT

+41919211131; info@gaintherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: December 12, 2025
• Study Start: May 30, 2026
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): June 30, 2028
• Last Updated: December 12, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share