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A Phase IIa Multi-Center Study of 18F-FDG PET, Safety, and Tolerability of AZD0530 in Mild Alzheimer's Disease

Phase 2
Completed
Conditions
Alzheimer's Disease
Interventions
Drug: Placebo
Drug: AZD0530 100mg daily
Drug: AZD0530 125mg daily
Registration Number
NCT02167256
Lead Sponsor
Yale University
Brief Summary

AZD0530 is an inhibitor of Src and Abl family kinases1. It has been developed as treatment for malignancies because these kinases play a role in tumor invasion and proliferation. However, the Src family kinases (SFKs) are highly expressed in brain and have major effects on synaptic plasticity2. Moreover, the investigators have recently shown that a specific SFK, namely Fyn, is aberrantly activated by specific conformations of the Amyloid Beta (Aß) peptide from Alzheimer's disease (AD). Genetic deletion of Fyn rescues AD deficits in preclinical models. This clinical trial will test the potential benefit of AZD0530 for Alzheimer's disease modification.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
159
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
AZD0530 100mg dailyAZD0530 125mg dailyPatients in the experimental group (50%) will be started on this dose. After 2 weeks, patients with a plasma drug level of \<100ng/ml will receive 125mg AZD0530 daily and remain in the same experimental group as patient receiving 100mg daily.
AZD0530 PlaceboPlacebo50% of patients will receive placebo treatment for the duration of the study,
AZD0530 100mg dailyAZD0530 100mg dailyPatients in the experimental group (50%) will be started on this dose. After 2 weeks, patients with a plasma drug level of \<100ng/ml will receive 125mg AZD0530 daily and remain in the same experimental group as patient receiving 100mg daily.
Primary Outcome Measures
NameTimeMethod
Change in Brain Glucose Uptake Measured Using 18F-FDG PET Imaging12 months

Composite measure of brain glucose uptake using F18-FDG PET in a pre-defined set of brain regions, between baseline and 12 months.

Number of Participants With One or More Serious/Other Adverse Events Subjects With Mild AD as Assessed by Analysis of Adverse Events, Including Symptoms, and Abnormal Findings on Physical and Neurological Examinations, and Standard Labs.12 months

Assessment of any adverse effects between drug and placebo-treated subjects

Secondary Outcome Measures
NameTimeMethod
Percent Change in Brain Volume Before and After Treatment12 months

Change in volume of pre-defined brain regions between baseline and 12 months of treatment.

The Effect of Treatment With AZD0530 on Cognitive and Behavioral Function12 months

The change in cognitive function between baseline and 12 months will be measured by the following tests:

1. Alzheimer Disease Assessment Scale - Cognitive 11 (ADAS-cog11). Measures: Cognitive function Maximum score: 70; Minimum score: 0. Higher score equals worse cognitive function

2. Mini-Mental State Examination (MMSE) Measures: Cognitive Function Maximum score: 30; Minimum score: 0. Higher score equal better cognitive function

3. Alzheimer Disease Cooperative Study - Activities of Daily Living Inventory (ADCS-ADL) Measures: Ability to perform routine daily activities Maximum score: 78; Minimum score: 0. Higher score equals better ability to perform activities of daily living

4. Clinical Dementia Rating Sum of Boxes (CDR-SO) Measures: Dementia severity Maximum score: 18; Minimum score: 0. Higher score equals more severe dementia.

Change in Brain Glucose Uptake Measured Using 18F-FDG PET Imaging12 months

The results from the primary outcome with brain FDG-PET imaging was analyzed by ApoE genotype. Composite measure of brain glucose uptake using F18-FDG PET in a pre-defined set of brain regions, between baseline and 12 months.

Cerebrospinal Fluid Levels of Total Tau, Phospho-tau (p-Tau), and Amyloid-beta 1-42 (Abeta 1-42)12 months

Measure concentration of Tau and Amyloid-beta 1-42 biomarkers in the cerebrospinal fluid between baseline and 12 months of treatment

Trial Locations

Locations (22)

Wien Center for Clinical Research/Mount Sinai Medical Center

🇺🇸

Miami Beach, Florida, United States

University of South Florida - Health Byrd Alzheimer Institute

🇺🇸

Tampa, Florida, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

University of Michigan, Ann Arbor

🇺🇸

Ann Arbor, Michigan, United States

University of Iowa

🇺🇸

Iowa City, Iowa, United States

University of Rochester Medical Center

🇺🇸

Rochester, New York, United States

Brigham and Women's Hospital

🇺🇸

Boston, Massachusetts, United States

Oregon Health & Science University

🇺🇸

Portland, Oregon, United States

University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

Mount Sinai School of Medicine

🇺🇸

New York, New York, United States

Indiana University

🇺🇸

Indianapolis, Indiana, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

Barrow Neurological Institute

🇺🇸

Phoenix, Arizona, United States

University of British Columbia, Clinic for AD & Related Disorders

🇨🇦

Vancouver, British Columbia, Canada

Yale Alzheimer's Disease Research Unit

🇺🇸

New Haven, Connecticut, United States

Georgetown University

🇺🇸

Washington, District of Columbia, United States

Roper St. Francis Hospital

🇺🇸

Charleston, South Carolina, United States

University of Washington

🇺🇸

Seattle, Washington, United States

University of Kentucky

🇺🇸

Lexington, Kentucky, United States

Wake Forest University Health Sciences

🇺🇸

Winston-Salem, North Carolina, United States

University of Pittsburgh, Alzheimer's Disease Research Center

🇺🇸

Pittsburgh, Pennsylvania, United States

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