Safety and Efficacy of Allopregnanolone as a Regenerative Therapeutic for Alzheimer's Disease in a Multicenter, Double-Blind, Randomized, Placebo-Controlled Phase 2 Trial

What this Trial Is About

Researchers are evaluating Allopregnanolone as a potential regenerative treatment for people with mild Alzheimer's disease dementia. This phase 2 clinical trial aims to assess the long-term safety and effectiveness of Allopregnanolone in restoring brain structure and improving cognitive function in participants aged 55 to 80 who have probable Alzheimer's disease and a Mini-Mental State Exam score between 20 and 26. Participants will undergo a 2 to 4-week screening period before being randomly assigned to receive either 4 mg of Allopregnanolone or a matching placebo. Both treatments are given as intravenous infusions over 30 minutes once a week for 6 months in a clinical setting. After this period, those initially given placebo will switch to receive Allopregnanolone for an additional 3 months in an open-label phase. Brain imaging scans will be performed at the start, and at 3 and 6 months to evaluate treatment effects. During the study, participants will have regular assessments including cognitive tests like the Alzheimer's Disease Assessment Scale-Cognitive Subscale and brain scans to measure hippocampal volume, which is the main outcome. Safety and tolerability will also be monitored throughout the 6-month blinded period. The study lasts for about 9 months in total, including the open-label extension, with close monitoring to understand how the treatment affects brain health and daily function.

Allopregnanolone Regenerative Therapeutic for Mild Alzheimer's Disease