MedPath

Tricaprilin in Mild to Moderate Alzheimer's Disease

Phase 2
Completed
Conditions
Alzheimer's Disease
Interventions
Other: Placebo
Registration Number
NCT00142805
Lead Sponsor
Cerecin
Brief Summary

The purpose of this study is to evaluate the safety, tolerability and effectiveness of tricaprilin administered once a day for ninety days in subjects with mild to moderate, probable Alzheimer's disease.

Detailed Description

Substantial scientific evidence has shown that defects in glucose metabolism occur in Alzheimer's disease. Attempts to compensate for the reduced cerebral metabolic rates in AD have met with some success. Treatment of AD patients with high doses of glucose and insulin will raise cognitive scores. However, this effect is slight, and high doses of insulin can have adverse consequences. Administration of ketone bodies or their metabolic precursors such as medium chain triglycerides (MCTs) presents an attractive alternative to glucose and insulin. In a preliminary study, tricaprilin, an MCT, demonstrated pharmacological activity and statistically significant efficacy in improving short-term memory and attention performance after a single dose.

Participants will be randomized to receive either tricaprilin or a matching placebo, administered once a day by mixing powder in a glass of liquid. The treatment period will last 90 days, followed by a 2-week washout period. Each patient will be seen 5 times: at screening, baseline, and post-baseline days 45, 90, and 104. The visits will include physical and/or neuropsychological examinations, electrocardiograms (ECGs) and laboratory tests.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
152
Inclusion Criteria
  • Informed Consent Form signed by patient and caregiver
  • Diagnosis of probably Alzheimer's disease of mild to moderate severity
  • Age 50 or older
  • If female, 2 years postmenopausal or surgically sterile
  • Hearing, vision, and physical abilities adequate to perform assessments (corrective aids allowed)
  • Caregiver to attend all visits, perform assessments, and supervise administration of study medication
  • CT or MRI within 24 months prior to screening compatible with a diagnosis of probably Alzheimer's disease
  • Modified Hachinski Ischemia Scale score of 4 or less
  • ADAS-Cog score between 15 and 35 inclusive at screening
  • MMSE score between 14 and 24 inclusive at screening
  • Stable medical condition for 3 consecutive months immediately prior to baseline
  • No clinically significant abnormal findings on the physical examination, medical history, or clinical laboratory results during screening
Exclusion Criteria
  • Any condition that would, in the opinion of the Principal Investigator, render the patient or the caregiver unsuitable for the study, or place them at substantial risk of adverse outcome
  • Unwillingness or inability of the patient and/or caregiver to fulfill the requirements of the study
  • Resident in a skilled nursing facility
  • Any significant neurological disease other than probable AD (e.g. Parkinson's disease, Huntington's disease, brain tumor, normal pressure hydrocephalus, progressive supranuclear palsy, seizure disorder, subdural hematoma, multiple sclerosis, history of stroke, or history of head injury requiring hospitalization)
  • An alternate cause for dementia other than AD as determined by a required CT or MRI scan within 24 months prior to screening
  • Current history of major psychiatric disorder
  • Major depression as determined by a Cornell Scale for Depression in Dementia
  • Clinically significant hypothyroidism
  • Clinically significant B12 deficiency
  • Unstable or clinically significant cardiovascular disease
  • Diabetes of any type
  • History of tertiary syphilis
  • Cancer within 3 years prior to baseline, with the exception of squamous and basal cell carcinoma
  • Vital sign abnormalities
  • Clinically significant renal disease or insufficiency
  • Clinically significant hepatic disease or insufficiency
  • Alcohol consumption greater than 2 oz of spirits per day or 14 oz per week (1 oz of spirits is equal to 6 oz of wine or 12 oz of beer)
  • Current history of alcohol abuse or other substance abuse within 24 months prior to baseline
  • Known HIV infection
  • Use of any investigational compound within 30 days prior to screening
  • Use of prohibited medications (contact site for details)
  • Prior or current use of medium-chain triglycerides (MCTs) for medical purposes
  • Known allergies to coconut oil

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Matching Placebo to AC-1202PlaceboPlacebo formulation, once daily. Administered orally
AC-1202TricaprilinTricaprilin formulation, once daily. Administered orally
Primary Outcome Measures
NameTimeMethod
Number of subjects with treatment related adverse events104 days

AE incidence rate per treatment group

Secondary Outcome Measures
NameTimeMethod
Pharmacokinetics (PK) profile of tricaprilinBaseline, Day 45, Day 90

Correlations between the Cmax serum BHB level on Day 90 and the change from baseline total score for the three efficacy scales was determined by the Pearson correlation statistics

Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog)90 days

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)

Clinical Global Impression of Change90 days

Clinician's global impression rated with Alzheimer's Disease cooperative Study - Clinical Global Impression of Change (ADCS-CGIC). The rating is from marked improvement to marked worsening.

Mini-Mental State Exam (MMSE)90 days

Change in MMSE

Trial Locations

Locations (14)

Comprehensive NeuroScience

🇺🇸

Saint Petersburg, Florida, United States

Baumel-Eisner Neuromedical Institute

🇺🇸

Miami Beach, Florida, United States

Sunrise Clinical Research

🇺🇸

Hollywood, Florida, United States

Grayline Clinical Drug Trials

🇺🇸

Wichita Falls, Texas, United States

Research Across America

🇺🇸

Dallas, Texas, United States

Pharmacology Research Institute

🇺🇸

Riverside, California, United States

Multi-Specialty Research Associates of North Carolina

🇺🇸

Raleigh, North Carolina, United States

The Southwest Institute for Clinical Research

🇺🇸

Rancho Mirage, California, United States

21st Century Neurology, a division of Xenoscience Inc.

🇺🇸

Phoenix, Arizona, United States

Meridien Research

🇺🇸

Tampa, Florida, United States

Baumel-Eisner Neuromedical Institute, Inc.

🇺🇸

Fort Lauderdale, Florida, United States

Anchor Research Center

🇺🇸

Naples, Florida, United States

Renstar Medical Research

🇺🇸

Ocala, Florida, United States

Radiant Research

🇺🇸

San Antonio, Texas, United States

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