A Phase III Multi Regional Clinical Trial (MRCT) of Tricaprilin in Mild to Moderately Severe Probable Alzheimer's Disease With Optional Open Label Extension

NCT04187547 | Withdrawn Phase 3 DMC FDA Drug

• 1 other identifier: AC-19-020
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is Phase 3 study, multi-centre, double-blind, placebo controlled, parallel group to evaluate the effects of AC-SD-03 on the efficacy and safety among participants with mild to moderate Alzheimer's Disease.

Conditions

Alzheimer's Disease

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Alzheimer's Disease Assessment Scale-cognitive Subscale (ADAS-cog11). Total Score up to 20-week Treatment

  Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-cog 11) is an 11- item cognitive subscale that objectively measures memory, language, orientation, and praxis with a total score range of 0 (no impairment) to 70 (severe impairment).

  20 weeks

Secondary Outcomes (5)

• Number of subjects with treatment related adverse events

  20 weeks

• Clinical Global Impression of Change

  20 weeks

• Change From Baseline in Dependence Scale.Total Score up to 20-week Treatment

  20 weeks

• Change From Baseline in RUD-Lite

  20 weeks

• Change From Baseline in Disability Assessment for Dementia

  20 weeks

Study Arms (2)

AC-SD-03

ACTIVE COMPARATOR

Tricaprilin SD formulation, twice daily. Administered orally

Drug: Tricaprilin

AC-SD-03P

PLACEBO COMPARATOR

Placebo formulation, twice daily. Administered orally

Drug: Placebo

Interventions

Tricaprilin DRUG

Powder formulation will be mixed with 240 mL water and shaken until fully dispersed. Each dosing unit of AC-SD-03 contains 20 g of the active ingredients (tricaprilin)

Also known as: AC-SD-03

AC-SD-03

Placebo DRUG

Matching placebo to AC-SD-03 will be mixed with 240 mL water and shaken until fully dispersed.

Also known as: AC-SD-03P

AC-SD-03P

Eligibility Criteria

• Age: 50 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects between the ages of 50 and 85 (inclusive) at the time of Screening Visit 1. Subjects 86 to 90 years of age (inclusive) can be enrolled, provided that the Medical Monitor reviews the subject's medical condition during the screening process and approves enrolment.
• Dementia of mild to moderate severity, as classified by MMSE score between 14 to 26, inclusive, at Screening Visit 1.
• Meets diagnostic clinical criteria of probable Alzheimer's dementia according to the National Institute on Aging and the Alzheimer's Association (NIA-AA) \[25\].
• Magnetic resonance imaging prior to Visit 3 (Baseline) compatible with a diagnosis of probable AD.
• Fluorodeoxyglucose Positron Emission Tomography (FDG-PET) brain scan showing a pattern typical for AD according to central PET reader (with areas of glucose hypometabolism in posterior cingulate, temporal, parietal, and/or prefrontal cortices, as per criteria in imaging manual).
• Confirmed APOE negative genotype result obtained by the central laboratory prior to Screening Visit 2 or documented prior to screening.
• Subjects taking the following cholinesterase inhibitors (ChEI): donepezil, galantamine, or rivastigmine; and/or GV-971, and/or memantine and/or other agents which may impact cognition (e.g., Souvenaid®, NeuroAidTM, Cerefolin®, Gingko biloba, etc) are eligible for enrolment:
• If the subject has been taking such medication(s)/products for 3 months or more at Screening Visit 1.
• If the current dosage regimen is within the approved dose range.
• The daily dose has remained unchanged for at least 6 weeks prior to screening.
• If the dose is not expected to change during trial participation.
• Tacrine use is not allowed.
• In the opinion of the Investigator, the subject's medical condition, except for dementia, has been stable for at least 3 consecutive months prior to Screening Visit 1.
• Subjects do not have active suicidal thoughts (answers 'yes' to questions 4 or 5, on the Columbia-Suicide Severity Rating Scale \[C-SSRS\]) within 6 months preceding Screening Visit 2 or Visit 3 (Baseline). Additionally, subjects have no active history of suicide attempt in the previous 2 years or more than 1 lifetime suicide attempt, nor are they at serious suicidal risk, in the opinion of the Investigator.
• The subject is able to comply with protocol testing and procedures for the entire length of the trial.

You may not qualify if:

• Current use or use within 3 months of Visit 3 (Baseline), of Axona® or other MCT-containing products. Use of coconut oil up to 15 mL (1 tablespoon) per day is allowed. Use of MCT-containing products is not allowed at any time during trial participation.
• Use of any other investigational agent within 60 days prior to Screening Visit 1.
• Has a known allergy or hypersensitivity to triglycerides.
• In the opinion of the Investigator, has presence or history of an advanced, severe, progressive, or unstable disease of any type that could interfere with efficacy and safety assessments, or put the subject at risk.
• 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, etc.)
• Has a modified Hachinski Ischemia score greater than (\>) 4 at Screening Visit 2.
• Has a history or clinical laboratory evidence of cerebrovascular disease (stroke, transient ischemic attack, haemorrhage), or diagnosis of possible, probable, or definite vascular dementia at Screening Visit 1 in accordance with National Institute of Neurological Disorders and Stroke criteria.
• Brain MRI performed at Screening (prior to Visit 3) (per centrally read MRI) that shows evidence of any of the following:
• Acute, sub-acute or chronic haemorrhage.
• Cortical infarct (defined as \> 1.5 cm in diameter).
• \> 1 lacunar infarct (defined as \< 1.5 cm in diameter).
• Superficial siderosis.
• History of diffuse white matter disease as defined by a score of 3 on the age-related white matter changes scale \[26\].
• Any finding that, in the opinion of the Investigator, might be a contributing cause of subject's dementia, might pose a risk to the subject, or might prevent a satisfactory MRI assessment for safety monitoring.
• Has a history or clinical laboratory evidence of moderate congestive heart failure defined by the New York Heart Association Criteria (Class I to IV).

Sponsors & Collaborators

• Cerecin: lead

MeSH Terms

Conditions

Alzheimer Disease

Interventions

tricaprylin

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 3
• 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 29, 2019
• First Posted: December 5, 2019
• Study Start: June 30, 2022
• Primary Completion: December 31, 2023
• Study Completion: February 28, 2024
• Last Updated: April 14, 2023

Record last verified: 2023-04