Phase 3 Double-blind, Randomized Trial Efficacy and Safety of AR1001 in Early Alzheimer’s Disease
Research type
Research Study
Full title
A Phase 3 Double-blind, Randomized, Placebo-controlled, Multi-center Trial to Evaluate the Efficacy and Safety of AR1001 over 52 Weeks in Participants with Early Alzheimer’s Disease.
IRAS ID
1009036
Contact name
Fred Kim
Contact email
Sponsor organisation
AriBio Co., Ltd
Clinicaltrials.gov Identifier
Research summary
Early Alzheimer's disease is a brain disorder that slowly destroys memory and thinking skills of performing tasks. Current Alzheimer treatments in the market have had modest effects on cognition for patients with moderate/severe Alzheimer’s disease. However novel approaches are needed to either address symptom improvement or modify disease progression, while offering an improved benefit-risk profile over that of existing therapies. AR1001 is a novel small molecule compound with potential to alleviate Alzheimer disease pathology via multiple mechanisms of actions. AR1001 also shows favourable attributes for a central nervous system (CNS) drug with good biological uptake, blood brain barrier (BBB) penetration and an excellent safety profile. Moreover, AR1001’s mechanism of action may be able to address the multifactorial causes of neurodegenerative diseases such as Alzheimer’s disease (AD), with a complex cause of a disease and abnormal changes in body function. AriBio Co., Ltd. are sponsoring to evaluate both efficacy and safety of AR1001 compared with Placebo in participants with early Alzheimer’s disease and will be evaluating the effect of AR1001 on AD-related biomarkers. This study is a global phase 3 double-blinded, trial to evaluate the efficacy and safety of AR1001 over 52 weeks of treatment in participants with early AD. The study population will be males or females, aged 55 to 90 years, who have a diagnosis of mild cognitive impairment or mild dementia due to Alzheimer’s dementia and have a positive brain amyloid pathology confirmed through imaging or the assessment of biofluids. Approximately 1,150 participants will be randomly assigned to one of two dose groups of participants who will receive daily tablets of AR1001 or placebo for daily self-administration. This trial will be approximately 2 years and consist of a screening phase, treatment phase, an optional drug extension phase (AR1001 will be offered to everyone) and follow up phase.
REC name
London - Hampstead Research Ethics Committee
REC reference
23/LO/1000
Date of REC Opinion
21 Feb 2024
REC opinion
Further Information Favourable Opinion