GAIN Study
Study Sponsor: Cortexyme

The GAIN (GingipAIN Inhibitor for Treatment of Alzheimer’s Disease) Trial is based on a growing body of scientific evidence that the bacteria P. gingivalis, most commonly associated with degenerative gum disease, can infect the brain and cause Alzheimer’s disease.

This clinical trial is evaluating whether the investigational oral drug atuzaginstat is safe and can slow or halt the progression of Alzheimer’s disease by inactivating the toxic proteins, called gingipains, released by the bacteria and stop or slow further damage to healthy brain cells.  

Clinical Trials Are the Path to New Treatments

With a lack of effective long-term treatments, researchers are working hard to find new and better approaches for slowing the progression of Alzheimer’s disease. Advances in treatment are only possible through clinical research trials like GAIN. A clinical trial is a medical research study to explore whether an investigational drug is safe and effective for humans. These trials follow strict scientific standards and regulatory requirements to protect patients and help produce reliable results.

A clinical trial may find that a new intervention improves patient outcomes, offers no benefit or causes unexpected harm. All of these results are important because they advance medical knowledge and help improve patient care. Study physicians reviewed risks and potential benefits with patients and their caregivers before patients were enrolled in the GAIN Trial. All new drug candidates like atuzaginstat are studied in clinical trials to generate data that can be evaluated by the US Food and Drug Administration (FDA) and other regulators for safety and effectiveness before being approved for common use.

Subjects enrolled in the GAIN Trial were randomly assigned to one of three groups:

  • One-third are taking an 80 milligram pill of the study drug, atuzaginstat, by mouth twice a day
  • One-third are taking a 40 milligram pill of atuzaginstat by mouth twice a day
  • One-third are taking a placebo (sugar pill) by mouth twice a day 

In order to scientifically assess the effects of the drug, neither the participants nor the clinical team  know during the study what group each patient is in. Participation in the study lasts about one year.

All eligible U.S. participants who complete the one-year study may have the opportunity to enroll in a follow-on study in which all subjects receive atuzaginstat. 

Gain is Not Actively Enrolling Subjects at This Time

Scientific experts have discovered that a bacteria called P. gingivalis, most commonly associated with degenerative gum disease, can infect the brain in older people who are more susceptible. Once in the brain the bacteria releases toxic proteins, called gingipains, that have been shown to destroy neurons and cause other signature signs of Alzheimer’s disease in the brains of  animals.  Atuzaginstat is designed to inactivate the toxic gingipains created by the P. gingivalis bacteria, reduce inflammation, reduce the bacterial infection and slow or halt progression of Alzheimer’s disease.

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COR388 is being developed by Cortexyme, Inc.

Gain is Not Actively Enrolling Subjects at This Time