Tricaprilin Phase 3 AD Study

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the effects of tricaprilin (20 g twice a day) on cognition, activities of daily living, resource utilisation, safety and tolerability, in subjects with mild to moderately severe probable AD. This is a randomised, double-blind, placebo-controlled, parallel-group, multi-centre design in up to 535 participants.

Official Title
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A Phase III, 26-Week, Double-blind, Randomised, Placebo-controlled, Parallel-group Study to Investigate the Efficacy and Safety of Daily Administration of Tricaprilin in Subjects With Mild to Moderately Severe Probable Alzheimer's Disease

Conditions
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Alzheimer Disease

Intervention / Treatment
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* Drug: Tricaprilin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Mini Mental State Exam (MMSE) score between 14 to 24 Meets diagnostic clinical criteria of probable Alzheimer's dementia according to the NIA-AA criteria Magnetic resonance imaging (MRI) compatible with a diagnosis of probable AD according to central MRI reader. Participants taking the following cholinesterase inhibitors: donepezil, galantamine, or rivastigmine; and/or memantine Key Exclusion Criteria: Current use or use within 3 months of Visit 3 (Baseline), of medium chain triglyceride -containing products. Completed less than 6 years of formal education. Has any medical or neurological condition, other than AD, that could explain the subject's dementia (e.g., structural abnormality, traumatic brain injury, stroke, epilepsy, Parkinson's disease, alcohol-related dementia) Has a modified Hachinski Ischaemia score > 4

Ages Eligible for Study
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Minimum Age: 50 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Tricaprilin<br>Tricaprilin formulation twice daily for 26 weeks, liquid for oral administration | Drug: Tricaprilin<br>* Each dose of IMP will be administered orally 30 minutes after completing a meal. There is 1 g of tricaprilin per 2 ml of AC-OLE-01-VA. Subjects will titrate from 5 g twice a day to 20 g twice a day, 40 g total daily dose of tricaprilin; 80 ml total daily dose of AC-OLE-01-VA, over the course of 3 weeks.<br>|
| Placebo Comparator: Placebo<br>Placebo formulation, twice daily for 26 weeks, liquid for oral administration | Drug: Placebo<br>* Each dose of IMP will be administered orally 30 minutes after completing a meal. Subjects will titrate from 5 g twice a day to 20 g twice a day, 40 g total daily dose of safflower oil; 80 ml total daily dose of placebo, over the course of 3 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog) total score | The ADAS-Cog is a cognitive scale that assesses memory, language, orientation, and praxis with a total score range of 0 (no impairment) to 70 (severe impairment). | 26 weeks | 
| Alzheimer's Disease Cooperative Study - Clinical Global Impression of Change (ADCS-CGIC) | The ADCS-CGIC is a clinician-rated scale of cognition, function, and behavior. Scores range from 1 (marked improvement) to 7 (marked worsening). | 26 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Subjects with Treatment Related Adverse Events | TEAE incidence rate per treatment group | 26 weeks | 
| Change From Baseline in Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL) score | The ADCS-ADL is a 23-item semi-structured interview with a caregiver and assesses 6 basic activities of daily living and 7 instrumental activities of daily living with a total score of 0 to 78. Lower scores indicate greater severity. | 26 weeks |