Phase II Trial to Evaluate Safety and Efficacy of GM-CSF/Sargramostim in Alzheimer's Disease (SESAD)
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|ClinicalTrials.gov Identifier: NCT04902703|
Recruitment Status : Recruiting
First Posted : May 26, 2021
Last Update Posted : July 21, 2022
|Condition or disease||Intervention/treatment||Phase|
|Alzheimer Disease||Drug: Sargramostim Drug: Saline - placebo comparator||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||42 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||The study is double-blind, placebo-controlled in the Alzheimer's Disease population, and will include individuals with mild AD and moderate AD. Individuals who meet inclusion/exclusion criteria will be randomized in a double-blind manner, to receive either sargramostim 250 µg/m2/day subcutaneously (5 days per week) or placebo (5 days per week) in an approximate 2:1 randomization ratio.|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||Phase II Trial to Evaluate Safety and Efficacy of GM-CSF/Sargramostim in Alzheimer's Disease (SESAD)|
|Actual Study Start Date :||June 1, 2022|
|Estimated Primary Completion Date :||July 2024|
|Estimated Study Completion Date :||July 2024|
250 mcg/m2/day subcutaneously 5 days/week for 24 weeks
Sargramostim is a granulocyte macrophage colony stimulating factor that will be administered at a dose of 250 mcg/m2 per day subcutaneously, 5 days/week, for 24 weeks
Placebo Comparator: Placebo Control - Saline
Placebo comparator (saline) subcutaneously 5days/week for 24 weeks
Drug: Saline - placebo comparator
Saline will be administered subcutaneously, 5 days/week, for 24 weeks
Other Name: Sterile solution of sodium chloride in water
- Safety as measured by number of Adverse Events (AEs) by body system [ Time Frame: Informed consent to Follow-up Visit (38 weeks) ]The safety of sargramostim will be assessed through number of adverse events (AEs) by body system from consent to follow-up within a safety analysis set consisting of all individuals who were enrolled and and randomized and who received at least on injection of sargramostim or placebo.
- Mini-Mental State Examination [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]Mini-Mental State Examination (MMSE) is a brief psychometric instrument developed to assess cognitive function in elderly populations. It is a standard assessment used by all NIH Alzheimer's Disease Centers (ADCCs and ADRCs) to identify and monitor individuals with AD. The range for scores in the MMSE is from 0 to 30, with lower scores indicating greater impairment.
- Alzheimer's Disease Assessment Scale - cognitive subscale (ADAS-Cog13) [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]The ADAS-Cog13 measures the severity of the most important symptoms of AD. and consists of 13 tasks measuring the disturbances of memory, language, praxis, attention and other cognitive abilities. , which are often referred to as the core symptoms of AD. The score ranges form 0-85, with higher score denoting worse performance
- Clinical Dementia Rating Scale - Sum of Boxes (CDR-SB) [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]The CDR is a study partner/caregiver and participant based interview to assess changes in domains such as memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care. Each domain is rated as 0 (no dementia), 0.5 (uncertain dementia), 1 (mild dementia), 2 (moderate dementia), or 3 (severe dementia). Range is 0-18. Higher scores denote worse functioning
- Trail Making Test - Part A (TMT-A) [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]Psychomotor speed will be assessed by the Trail Making Test-A, a timed test in which participants must connect a series of numbers randomly placed on a page. Time range is 0-150 seconds, with a higher time denoting worse performance
- Alzheimer's Disease Cooperative Study -Activities of Daily Living Inventory (ADCS-ADL) [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]The ADCS-ADL is a caregiver/study partner rated questionnaire of 23 items, with possible scores over a range of 0-78, where 78 implies full functioning with no impairment.
- Montreal Cognitive Assessment (MoCA) [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]The Montreal Cognitive Assessment (MoCA) is a brief, assessment developed for detection and tracking of cognitive impairment and is sensitive for detecting Alzheimer's disease. Measuring multiple domains, it is commonly used in both clinical and research settings, and is well validated, with a range of scores from 0-30.
- Neuropsychiatric Inventory (NPI) [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]Neuropsychiatric Inventory (NPI) is a study partner/caregiver interview to assess any changes in neuropsychiatric status in such domains as hallucinations, delusions, agitation, depression, anxiety, disinhibition, apathy and aberrant motor behaviors. It assesses presence of symptoms, and scores by severity X frequency (Range 0-12 in each symptom category), and caregiver distress (Range 0-5). The higher the scores, the the greater the presence of the impact of the symptom.
- Verbal Fluency [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]Verbal fluency (producing words that start with a specific letter of the alphabet, also known as letter fluency) is a timed test where a participant produces as many works as they can in 60 seconds. The more correct words that a participant produces, the higher the score.
- Verbal Fluency [ Time Frame: Baseline to End of Treatment, Follow-up (30 weeks) ]Semantic fluency (producing words that belong in a category, such as animals, also known as category fluency) is a timed test where a participant produces as many works as they can in 60 seconds within a category. The more correct words that a participant produces, the higher the score.
- Exploratory: Fluorodeoxyglucose Positron Emission Tomography (FDG PET) assessment of brain metabolism [ Time Frame: Baseline to End of Treatment (24 weeks) ]FDG-PET assessment of metabolic activity, overall, as well in different brain regions, particularly in the medial temporal lobe in potential correlation with changes in MTA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04902703
|Contact: Neurology Research Partners, CU Department of Neurologyemail@example.com|
|United States, Colorado|
|University of Colorado Anschutz Medical Campus||Recruiting|
|Aurora, Colorado, United States, 80045|
|Contact: Neurology Research Partners 303-724-4644 firstname.lastname@example.org|
|Principal Investigator:||Peter Pressman, MD||University of Colorado Alzheimer's and Cognition Center|