Phase II clinical trial of KCHO-1 for Alzheimer's disease: study protocol for a multicenter, randomized, double-blind, multi-dose, placebo trial

BACKGROUND: Alzheimer's disease (AD) is the most common degenerative brain disorder leading to dementia, characterized by a slow onset and gradual progression. Early symptoms typically include difficulties in recalling recent events, which later extend to impairments in various cognitive functions such as language, judgment, and problem-solving, ultimately resulting in the complete loss of independent daily functioning. KCHO-1, a herbal extract with demonstrated anti-inflammatory and neuroprotective properties, has shown promise in mitigating the effects of neurodegenerative disorders. This study aims to develop a protocol for determining the optimal dosage of KCHO-1 in treating AD, providing a foundation for its potential therapeutic application. METHODS: This is a phase II, multi-center, 3-arm randomized controlled study. Sixty patients with AD will be randomly assigned to one of three groups. Each group will receive a standard treatment with 1.6 g of KCHO-1, 2.4 g of KCHO-1, or a placebo. The primary outcome is ADAS-K-cog. Secondary outcomes include results of K-MMSE, K-IADL, NPI-Q, CDR, GDS, KQOL-AD, R-MBPC, and S-GDpS. The frequencies of severe adverse events as well as overall adverse occurrences are listed and documented. The trial protocol has been approved by the Institutional Review Board of the Wonkwang University Hospital Gwangju Korean Medical Center and Wonkwang University Hospital (WKIRB 2021/14-19 and WKUH IRB 2023-10-005-012, respectively). The Ministry of Food and Drug Safety (MFDS) approved the drug as an investigational new drug (30731). DISCUSSION: The purpose of this study is to establish the ideal KCHO-1 dosage. Additionally, it seeks to validate KCHO-1's safety and effectiveness in reducing functional deterioration in AD patients. TRIAL REGISTRATION: Korean National Clinical Trial Registry CRIS; KCT0008433. Registered on 12 May 2023.