Recruiting people with early Alzheimer's Disease for a clinical trial evaluating benfotiamine

ATLANTA, GEORGIA— The Goizueta Alzheimer’s Disease Research Center at Emory University Goizueta Brain Health Institute is conducting a new, national Alzheimer’s disease (AD) clinical research study evaluating the potential benefits of benfotiamine, a synthetic version of thiamine or vitamin B1, as a treatment for mild cognitive impairment (MCI) or early AD. The Phase 2 study, called BenfoTeam, will evaluate the effects of benfotiamine on cognitive function and preservation and/or potential improvement in daily routine activities. Emory University is one of nearly 50 sites in the U.S. participating in the BenfoTeam study. The phase 2 study is funded by the National Institute on Aging, part of the National Institutes of Health (NIH), to evaluate the effects of benfotiamine on cognitive function.

Previous research from a 12-month, single-site pilot trial in people with early AD demonstrated that benfotiamine was safe and well tolerated, with encouraging results in how the body processed and responded to the treatment. For example, the increase in the Alzheimer’s Disease Assessment Scale-Cognitive Subscale was 43% lower in the benfotiamine group than in the placebo group, indicating less cognitive decline. The trial also provided preliminary evidence of the efficacy of benfotiamine on cognitive and functional outcomes, which was encouraging data to support the need for this Phase 2 study.

About Thiamine

Thiamine is crucial for brain function and energy production. A lack of thiamine, or vitamin B1, compromises glucose metabolism and is closely linked to thinking, memory and learning problems., The brain tissue in people with AD shows a marked thiamine deficiency despite sufficient levels of thiamine in a person’s blood. BenfoTeam neuroscientists think that the delivery of thiamine via prodrug will increase the amount of thiamine that gets to the brain.

Benfotiamine is a synthetic, or lab-made, version of vitamin B1 (thiamine), a prodrug. Prodrugs are inactive medications that transform into the drug within the body. They are developed to optimize a drug’s performance and to address things like tolerability, inadequate brain penetration, delivery, and absorption. The BenfoTeam trial will determine whether significantly increasing the amount of thiamine in the blood can help thiamine-dependent brain processes and slow down Alzheimer’s-related cognitive decline in people with mild cognitive impairment and early AD.

Clinical Trial Details

The BenfoTeam clinical trial is a randomized double-blind placebo-controlled Phase 2A-Phase 2B trial to evaluate the safety and efficacy of benfotiamine in people with early AD that is taking place at 50 clinical trial sites across the U.S. Participation in the trial will last up to 18 months (plus screening), with a total enrollment of 406 participants. The trial is recruiting people aged 50-89 who have mild memory problems due to AD or who have mild dementia because of AD. The study’s primary objectives are to determine the highest safe and well-tolerated dose of benfotiamine and to assess the efficacy of benfotiamine on global function and cognition over 72 weeks.

To enroll, participants must be between the ages 50-89 and have mild memory problems or mild dementia due to AD. For more information about participating in the study at Emory University, call 877-807-1290 or visit benfoteam.org. Given that Hispanic and Black communities experience a disproportionate burden of the disease, the trial is emphasizing enrollment of participants from underrepresented minority groups.

The clinical research trial is coordinated by the Alzheimer’s Disease Cooperative Study (ADCS) at University of California San Diego School of Medicine and is led by principal investigators from the Burke Neurological Institute (Weill Cornell Medicine), Columbia University Irving Medical Center and UC San Diego. This research will be supported by the NIH’s National Institute on Aging (R01AG076634). This content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH’s National Institute on Aging.

The ClinicalTrials.gov ID number for this study is: NCT06223360.

For more information about the trial and to find a study site, visit benfoteam.org.