A 6-Month Study to Evaluate the Safety & Potential Efficacy of Trappsol Cyclo in Patients With Early Alzheimer's Disease

Study Overview
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Brief Summary
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Approximately 90 patients, aged 50 to 80 years, with a diagnosis of early Alzheimer's disease will take part in this research study. This study will be conducted in the US. There will be 3 treatment groups: 2 Active doses and 1 group will receive placebo completely by chance. Patients, caregiver, Sponsor, nor study staff will know which treatment is assigned. There are 3 periods in this study: Screening to confirm suitability, Treatment to receive study medication, and Follow-up to check overall health post-participation

Detailed Description
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This is a randomized, placebo-controlled, double-blind, parallel-group study that will assess the safety, tolerability, and potential efficacy of Trappsol Cyclo in patients with EAD as defined according to the FDA Guidance for Industry on Early Alzheimer's Disease: Developing Drugs for Treatment. The study will enroll approximately 90 (30 patients/treatment arm) male and female patients aged 50 to 80 years at Screening with characteristic pathophysiologic changes of AD who meet National Institute on Aging-Alzheimer's Association (NIA-AA) criteria for either AD with MCI or mild AD collectively known as EAD (Stages 3 and 4). Enrolled patients must have evidence of progressive cognitive decline in the last year as determined by serial cognitive test scores, if available, or patient or informant/caregiver/study partner (hereafter called caregiver) report as documented by the Investigator

Official Title
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A Randomized, Placebo-controlled, Double-blind, Parallel-group, 6-Month Study to Evaluate the Safety, Tolerability, and Potential Efficacy of Monthly Trappsol® Cyclo™ Infusions in Patients With Early Alzheimer's Disease

Conditions
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Alzheimer's Disease

Intervention / Treatment
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* Drug: Hydroxypropyl Beta Cyclodextrin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: MCI due to AD (Stage 3) MMSE-2:SV score 20 and 28 at both Screening (V1) and Baseline (V2) with no more than a 3 point change between visits Positive PrecivityAD blood test biomarker for AD with high APS (58-100) Locally or centrally read MRI of ARIA Exclusion Criteria: Clinically significant renal disease Evidence of a neurodegenerative disease other than AD Severe hypothyroidism Abnormally low levels of serum Vitamin B12 Lacks visual, auditory acuity and/or language abilities adequate to perform cognitive assessments

Ages Eligible for Study
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Minimum Age: 50 Years
Maximum Age: 80 Years

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
Interventional Model Description: This is a randomized, placebo-controlled, double-blind, parallel-group study
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Experimental<br>Intravenous administration over at least 4 hours by IV infusion Trappsol Cyclo either 500 mg/kg or 1000 mg/kg every 4 weeks | Drug: Hydroxypropyl Beta Cyclodextrin<br>* Minimum active dose of 500 mg/kg (equivalent to 18,500 mg/m2) as an intravenous (IV) infusion once every 28 days<br>* Other names: Trappsol Cyclo;|
| Placebo Comparator: Placebo<br>Intravenous administration of 0.5N saline over at least 4 hours every 4 weeks | Drug: Placebo<br>* 0.5N saline as an intravenous (IV) infusion once every 28 days<br>* Other names: 0.5N saline;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety assessments to include incidence of Adverse Events and Serious Adverse Events | Incidence of AEs, SAEs, incidence of abnormal laboratory test results, abnormal ECGs, abnormal physical exams, abnormal vital signs and abnormal hearing assessments assessments | up to 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in total ADAS-Cog-14 score from Baseline | Memory, Language, and Executive Function | Week 12 and 24 | 
| Change in CDR-SB from Baseline | Memory, Orientation, Judgment and Problem Solving, Community Affairs, Home and Hobbies, and Personal Care | Weeks 12 and 24 | 
| Change in MMSE-2:SV total score from Baseline | Orientation, Attention, Memory, Language, and Visual-Spatial Skills | Weeks 12 and 24 | 
| Change in ADCS-CGIC from Baseline | Cognitive, Behavior, and Social and Daily Functioning | Weeks 12 and 24 | 
| Change in ADCS-ADL from Baseline | Basic Activities of Daily Living Items and Instrumental Activities of Daily Living Items | Weeks 12 and 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Alzheimer, Trappsol Cyclo