Mycose AdminiStration for HealIng Alzheimer NEuropathy (MASHIANE)

Study Overview
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Brief Summary
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Alzheimer's disease (AD) is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills, and, eventually, the ability to function independently. Despite the significant effort to understand the basic biology of the disease and pharmaceutical advances to develop drugs, there is no effective therapy available to treat AD or slow the disease progression. β-amyloid accumulation outside brain cells and abnormal accumulations of tau protein inside neurons are taught to be two main changes in the brain that lead to AD. Progressive accumulation of β-amyloid interferes with the neuron-to-neuron communication at synapses, contributing to neural cell death. Also, tau tangles block the transport of nutrients and other essential molecules into the neurons. Many molecules have been shown to inhibit amyloid aggregation. The anti-amyloidogenic activity of trehalose was confirmed in both in vitro and in vivo studies and its inhibitory effects on β-amyloid formation in AD have also been demonstrated. Trehalose is a non-toxic disaccharide and no dose-dependent adverse effects were seen in any of the safety studies. It can act as a chemical chaperone and stabilizes the natively folded structure of protein and also trehalose has been identified as an autophagy inducer and promotes the clearance of aggregated proteins. Therefore, trehalose could be a valuable candidate for the treatment and prevention of amyloid-related disease. Based on the proposed hypothesis, this study aim to investigate the potential efficacy of trehalose administration in patients with AD.

Detailed Description
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Purpose of the study: A randomized, triple-blind, pilot clinical trial has been designed to evaluate the effectiveness of trehalose on reducing the symptoms in AD patients. Study intervention: twenty patients with Alzheimer's disease were randomly divided into an intervention and a control group. Trehalose will be administrated intravenously (15 g/week) for 12 weeks in the intervention group and the control group will be received normal saline as a placebo.

Official Title
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Evaluation of the Therapeutic Effects of Trehalose in Patients With Alzheimer Disease

Conditions
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Alzheimer Disease

Intervention / Treatment
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* Drug: Trehalose


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Mini-Mental State Examination (MMSE) score range from 10 to 23 Not having other cognitive disorders Exclusion Criteria: MMSE score higher than 23 or lower than 10 The presence of other cognitive disorders which will be evaluated by clinical assessment and brain imaging The presence of factors affecting cognitive impairment such as depression Vascular dementia and Lewy body dementia Previous history of head trauma Use of alcohol and other drugs that affect cognitive functioning
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Trehalose<br>Trehalose Participants will be received intravenous trehalose infusion weekly (15 g/week) for a period of 12 weeks. | Drug: Trehalose<br>* Trehalose is a natural disaccharide sugar found extensively among miscellaneous organisms including bacteria, plants, insects, yeast, fungi, and invertebrates. By preventing protein denaturation, it plays various protective roles against stress conditions such as heat, freeze, oxidation, desiccation and dehydration. Owing to this capacity, trehalose is an FDA-approved pharmaceutical excipient that is used as a stabilizer in numerous medicines including parenteral products. In this study, all injections will be conducted by a trained nurse in the presence of a specialist physician at a duration of 45-90 minutes.<br>* Other names: Mycose;|
| Placebo Comparator: Placebo<br>Participants will be received equal volume of normal saline weekly for a period of 12 weeks. | Drug: Trehalose<br>* Trehalose is a natural disaccharide sugar found extensively among miscellaneous organisms including bacteria, plants, insects, yeast, fungi, and invertebrates. By preventing protein denaturation, it plays various protective roles against stress conditions such as heat, freeze, oxidation, desiccation and dehydration. Owing to this capacity, trehalose is an FDA-approved pharmaceutical excipient that is used as a stabilizer in numerous medicines including parenteral products. In this study, all injections will be conducted by a trained nurse in the presence of a specialist physician at a duration of 45-90 minutes.<br>* Other names: Mycose;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes of Mini-Mental State Exam (MMSE) | Global cognition will be assessed by MMSE test, which will be conducted at baseline and week 12. | From baseline to 12 weeks | 
| Changes in Clinical Dementia Rating Scale (CDR) | Clinical Dementia Rating Scale (CDR) has six different cognitive and behavioral domains such as memory, orientation, judgment and problem solving, community affairs, home and hobbies performance, and personal care, which will be conducted at baseline and week 12. | From baseline to 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Alzheimer Disease, Trehalose, Clinical Trial, Autophagy