A phase 2 randomized, placebo-controlled study on the efficacy and safety of AR1001, a phosphodiesterase-5 inhibitor, in patients with mild-to-moderate Alzheimer's disease.

ABSTRACT:

Background

AR1001 is a phosphodiesterase-5 inhibitor that produces improved cognitive performance and reduces amyloid-β and phosphorylated tau burdens in preclinical models of Alzheimer's disease (AD).

Objectives

To evaluate the safety and efficacy of AR1001 in participants with mild-to-moderate Alzheimer's disease (AD).

Design

Randomized, double-blind, placebo-controlled phase 2 trial conducted at 21 sites in the United States.

Participants

Adults aged 55-80 years with mild-to-moderate dementia as determined by National Institutes of Aging-Alzheimer's Association (NIA-AA) stage 4 or 5 and Mini-mental State Exam (MMSE) score 16-26.

Intervention

Once daily oral administration of placebo, 10 mg AR1001, or 30 mg AR1001 for 26 weeks followed by 26 weeks opti

SUBMITTER: Greeley D 

PROVIDER: S-EPMC12501329 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature