The PEGASUS study is a research study for people who have been diagnosed with Alzheimer’s disease or Mild Cognitive Impairment (MCI). The study is being conducted to confirm the safety and tolerability of the drug AMX0035, which is a combination of two drugs, Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA). In studies of mice, PB and TUDCA, when used together, were found to protect neurons in the brain from dying and reduce beta-amyloid deposits. Scientists believe an increased amount of amyloid in the brain plays an important role in the development and progression of Alzheimer’s disease. Both PB and TUDCA were found to be safe and tolerable in people with neurodegenerative diseases, including ALS and Huntington’s Disease, when used individually.
This is a placebo-controlled trial, and we are recruiting participants between the ages of 55-89 who have a diagnosis of probable Alzheimer’s disease or MCI. Participants will take medication on a daily basis and the length of the study, which involves 6 visits to Mount Sinai and 2 phone calls, is 8 months. There is no cost to participate. For more information, please contact the ADRC at 212-241-8329. PI: Judith Neugroschl, MD. IRB GCO 18-01284, 18-2125. GCO Expiration date: 10/15/2019.