Investigating the Efficacy of Ergothioneine to Delay Cognitive Decline (Pilot)

NCT ID: NCT03641404

Last Updated: 2025-06-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-08-01

Study Completion Date

2023-12-31

Brief Summary

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With the growing burden of dementia (including Alzheimer's disease), and the lack of efficacious therapies, there is an urgent need to identify new therapeutics.

Ergothioneine (ET) is a naturally occurring thiol derivative of histidine, obtained solely through diet and is able to accumulate in the body and brain, through the action of a specific transporter, OCTN1. In addition to a wide variety of in vitro and in vivo (animal) studies demonstrating the antioxidant, anti-inflammatory properties of ET, several studies have demonstrated the neuroprotective potential of ET in various cell and animal models.

Based on the ability of ET to counteract the underlying pathology of AD dementia, it is hypothesize that ET supplementation may prevent cognitive decline, especially in individuals at risk of cognitive impairment. This will be assessed using a randomized, double blinded, placebo-controlled, intervention study to test the ability of ET to delay or reverse cognitive impairment in elderly individuals with mild cognitive impairment.

Detailed Description

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Ergothioneine (ET) is a naturally occurring thiol/thione obtained in humans solely through diet. It is able to accumulate in specific cells and tissues (including the brain), via a specific transporter, OCTN1, at high levels. Although the exact physiological function(s) of ET have yet to be elucidated, numerous reports have demonstrated that this compound can scavenge reactive oxygen species (such as hydroxyl radicals, hypochlorous acid, and peroxynitrite), modulate inflammation, and chelate divalent metal ions. These processes are all implicated in the pathology of dementia. Various studies in cell and animal models have also highlighted the potential neuroprotective capabilities of ET following insult by various neurotoxic agents such as cisplatin and amyloid beta peptide.

Studies demonstrated that ET dose-dependently protected PC12 cells against beta amyloid-induced apoptotic death, and later was shown to protect against neuronal injury caused by direct administration of amyloid beta into the mouse hippocampus, thereby increasing scores in active avoidance and water maze tests. ET also dose-dependently extend lifespan of a transgenic Caenorhabditis elegans model of AD by reducing amyloid oligomer formation. Other studies also demonstrated that ET is also able to attenuate oxidative stress and prevents cognitive deficits in a D-galactose-induced dementia mouse model; protect against N-methyl-D-aspartate-induced cytotoxicity in rat retinal neurons; and prevent cisplatin-induced neuronal damage in cell cultures and mice.

To date no studies have evaluated the therapeutic ability of ET, clinically, to delay or halt cognitive decline. Prior studies administering pure ET to humans provide insights into the pharmacokinetics and demonstrate the safety of this compound, laying the foundations for this clinical study. The present proposal will shed light onto a relatively lesser known natural compound and the therapeutic capabilities it possesses, which has the potential to significantly impact the economic and societal burdens of dementia.

Conditions

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Mild Cognitive Impairment

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomized, double-blinded, placebo-controlled study
Primary Study Purpose

PREVENTION

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
Investigational compound and placebo coded by manufacturer. Codes provided in envelope will only be unsealed at the end of the study (data assessment) or in case of emergency.

Study Groups

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Ergothioneine

Subjects will consume 25mg ergothioneine (capsule), 3 times weekly (Monday, Wednesday, and Friday) for a total of 52 weeks.

Group Type ACTIVE_COMPARATOR

L-ergothioneine

Intervention Type DRUG

Ergothioneine is naturally occurring thiol compound obtained solely from diet in humans. Ergothioneine is widely reported to be a natural antioxidant and anti-inflammatory compound. In addition we hypothesize that this compound will be beneficial in improving cognition.

Placebo

Subjects will be given placebo (99% microcrystalline cellulose, 1% magnesium stearate; capsule), 3 times weekly (Monday, Wednesday, and Friday) for a total of 52 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo. Study control (99% microcrystalline cellulose, 1% magnesium stearate)

Interventions

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L-ergothioneine

Ergothioneine is naturally occurring thiol compound obtained solely from diet in humans. Ergothioneine is widely reported to be a natural antioxidant and anti-inflammatory compound. In addition we hypothesize that this compound will be beneficial in improving cognition.

Intervention Type DRUG

Placebo

Placebo. Study control (99% microcrystalline cellulose, 1% magnesium stearate)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Elderly individuals 60 - 90 years of age
* Chinese ethnicity (from other local cohort studies)
* Demonstrate amnestic mild cognitive impairment (assessed by panel of psychiatrists)
* Independent and able to travel to study site without assistance
* No other severe underlying conditions or terminal illnesses
* Capable of understanding the study and requirements and able to provide informed consent to participate
* Willing to commit to the year-long study, comply with study administration and periodic blood and urine sampling

Exclusion Criteria

* Inability to understand the risks and requirements of the study for any reason
* Any intolerance to lactose, and/or allergies to mushrooms
* History of cardiovascular complications, diabetes, hypertension or hypercholesterolemia, or other pre-existing condition that may prevent them from completing the study
* Evidence of anaemia or other significant haematological conditions
* History or mental illness, depression or other underlying psychiatric illnesses
* History of drug or alcohol abuse
* Involvement in another study requiring administration of an investigational compound in the past 30 days
* Subjects whose blood analysis reveal and extremes of liver or kidney function markers (from baseline screening)
* Deemed unfit for any reason as determined by the principal/co-investigator
Minimum Eligible Age

60 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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National University of Singapore

OTHER

Sponsor Role collaborator

National University Health System, Singapore

OTHER

Sponsor Role collaborator

National University Hospital, Singapore

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rathi Mahendran

Role: PRINCIPAL_INVESTIGATOR

National University of Singapore

Barry Halliwell

Role: PRINCIPAL_INVESTIGATOR

National University of Singapore

Christopher Chen

Role: PRINCIPAL_INVESTIGATOR

National University Hospital, Singapore

Locations

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National University Cancer Institute, Singapore

Singapore, Singapore, Singapore

Site Status

Countries

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Singapore

References

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Halliwell B, Cheah IK, Tang RMY. Ergothioneine - a diet-derived antioxidant with therapeutic potential. FEBS Lett. 2018 Oct;592(20):3357-3366. doi: 10.1002/1873-3468.13123. Epub 2018 Jun 15.

Reference Type BACKGROUND
PMID: 29851075 (View on PubMed)

Cheah IK, Halliwell B. Ergothioneine; antioxidant potential, physiological function and role in disease. Biochim Biophys Acta. 2012 May;1822(5):784-93. doi: 10.1016/j.bbadis.2011.09.017. Epub 2011 Oct 4.

Reference Type BACKGROUND
PMID: 22001064 (View on PubMed)

Halliwell B, Cheah IK, Drum CL. Ergothioneine, an adaptive antioxidant for the protection of injured tissues? A hypothesis. Biochem Biophys Res Commun. 2016 Feb 5;470(2):245-250. doi: 10.1016/j.bbrc.2015.12.124. Epub 2016 Jan 6.

Reference Type BACKGROUND
PMID: 26772879 (View on PubMed)

Cheah IK, Feng L, Tang RMY, Lim KHC, Halliwell B. Ergothioneine levels in an elderly population decrease with age and incidence of cognitive decline; a risk factor for neurodegeneration? Biochem Biophys Res Commun. 2016 Sep 9;478(1):162-167. doi: 10.1016/j.bbrc.2016.07.074. Epub 2016 Jul 19.

Reference Type BACKGROUND
PMID: 27444382 (View on PubMed)

Cheah IK, Tang RM, Yew TS, Lim KH, Halliwell B. Administration of Pure Ergothioneine to Healthy Human Subjects: Uptake, Metabolism, and Effects on Biomarkers of Oxidative Damage and Inflammation. Antioxid Redox Signal. 2017 Feb 10;26(5):193-206. doi: 10.1089/ars.2016.6778. Epub 2016 Sep 7.

Reference Type BACKGROUND
PMID: 27488221 (View on PubMed)

Tang RMY, Cheah IK, Yew TSK, Halliwell B. Distribution and accumulation of dietary ergothioneine and its metabolites in mouse tissues. Sci Rep. 2018 Jan 25;8(1):1601. doi: 10.1038/s41598-018-20021-z.

Reference Type BACKGROUND
PMID: 29371632 (View on PubMed)

Gruber J, Fong S, Chen CB, Yoong S, Pastorin G, Schaffer S, Cheah I, Halliwell B. Mitochondria-targeted antioxidants and metabolic modulators as pharmacological interventions to slow ageing. Biotechnol Adv. 2013 Sep-Oct;31(5):563-92. doi: 10.1016/j.biotechadv.2012.09.005. Epub 2012 Sep 27.

Reference Type BACKGROUND
PMID: 23022622 (View on PubMed)

Halliwell B, Cheah I. Are age-related neurodegenerative diseases caused by a lack of the diet-derived compound ergothioneine? Free Radic Biol Med. 2024 May 1;217:60-67. doi: 10.1016/j.freeradbiomed.2024.03.009. Epub 2024 Mar 14.

Reference Type BACKGROUND
PMID: 38492784 (View on PubMed)

Halliwell B, Tang RMY, Cheah IK. Diet-Derived Antioxidants: The Special Case of Ergothioneine. Annu Rev Food Sci Technol. 2023 Mar 27;14:323-345. doi: 10.1146/annurev-food-060822-122236. Epub 2023 Jan 9.

Reference Type BACKGROUND
PMID: 36623925 (View on PubMed)

Wu LY, Cheah IK, Chong JR, Chai YL, Tan JY, Hilal S, Vrooman H, Chen CP, Halliwell B, Lai MKP. Low plasma ergothioneine levels are associated with neurodegeneration and cerebrovascular disease in dementia. Free Radic Biol Med. 2021 Dec;177:201-211. doi: 10.1016/j.freeradbiomed.2021.10.019. Epub 2021 Oct 19.

Reference Type BACKGROUND
PMID: 34673145 (View on PubMed)

Cheah IK, Ng LT, Ng LF, Lam VY, Gruber J, Huang CYW, Goh FQ, Lim KHC, Halliwell B. Inhibition of amyloid-induced toxicity by ergothioneine in a transgenic Caenorhabditis elegans model. FEBS Lett. 2019 Aug;593(16):2139-2150. doi: 10.1002/1873-3468.13497. Epub 2019 Jun 30.

Reference Type BACKGROUND
PMID: 31211853 (View on PubMed)

Wu LY, Kan CN, Cheah IK, Chong JR, Xu X, Vrooman H, Hilal S, Venketasubramanian N, Chen CP, Halliwell B, Lai MKP. Low Plasma Ergothioneine Predicts Cognitive and Functional Decline in an Elderly Cohort Attending Memory Clinics. Antioxidants (Basel). 2022 Aug 30;11(9):1717. doi: 10.3390/antiox11091717.

Reference Type BACKGROUND
PMID: 36139790 (View on PubMed)

Cheah IK, Halliwell B. Ergothioneine, recent developments. Redox Biol. 2021 Jun;42:101868. doi: 10.1016/j.redox.2021.101868. Epub 2021 Jan 26.

Reference Type BACKGROUND
PMID: 33558182 (View on PubMed)

Yau YF, Cheah IK, Mahendran R, Tang RM, Chua RY, Goh RE, Feng L, Li J, Kua EH, Chen C, Halliwell B. Investigating the efficacy of ergothioneine to delay cognitive decline in mild cognitively impaired subjects: A pilot study. J Alzheimers Dis. 2024 Dec;102(3):841-854. doi: 10.1177/13872877241291253. Epub 2024 Nov 15.

Reference Type DERIVED
PMID: 39544014 (View on PubMed)

Other Identifiers

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1823117J (Clinical Trial No.)

Identifier Type: OTHER

Identifier Source: secondary_id

2017/00982

Identifier Type: -

Identifier Source: org_study_id

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