Study of the Safety & Efficacy of Leukine® in the Treatment of Alzheimer's Disease
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ClinicalTrials.gov Identifier: NCT01409915 |
Recruitment Status :
Completed
First Posted : August 4, 2011
Results First Posted : March 23, 2021
Last Update Posted : June 2, 2021
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Condition or disease | Intervention/treatment | Phase |
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Alzheimer's Disease | Drug: Sagramostim Drug: Saline -- placebo comparator | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 44 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | Pilot Phase 2 Trial of the Safety & Efficacy of Granulocyte-Macrophage Colony-Stimulating Factor (Leukine®) in the Treatment of Alzheimer's Disease |
Study Start Date : | March 2011 |
Actual Primary Completion Date : | December 9, 2019 |
Actual Study Completion Date : | December 9, 2019 |

Arm | Intervention/treatment |
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Experimental: Sagramostim (Leukine)
5 subjects 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Data and Safety Monitoring Board will then review data and recommend whether to continue at the same current recommended dose for additional subjects or to reduce the dose by half if excessive leukocytosis occurs
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Drug: Sagramostim
5 subjects 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Data and Safety Monitoring Board will then review data and recommend whether to continue at the same current recommended dose for additional subjects or to reduce the dose by half if excessive leukocytosis occurs
Other Names:
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Placebo Comparator: Control Group
Saline -- placebo comparator. Given as a subcutaneous injection.
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Drug: Saline -- placebo comparator
subcutaneous injection
Other Name: Sterile solution of sodium chloride in water |
- Adverse Events (AEs) by Body System [ Time Frame: 20 weeks (From Consent to Follow-up 2) ]Count of AE's from Consent to Follow-up 2 within a safety analysis set consisting of all participants who were enrolled and randomized and who received at least one injection of sargramostim or placebo
- MMSE (Mini Mental State Examination) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment) [ Time Frame: From Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment) ]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) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment) [ Time Frame: Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment) ]Alzheimer's Disease Assessment Scale - cognitive subscale (ADAS-Cog13). The ADAS-Cog13 is the most popular cognitive testing instrument used in clinical trials of nootropics (drugs or agents that improve cognitive function). It 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. Score ranges from 0-85, with a higher score representing more severe impairment
- Alzheimer's Disease Cooperative Study -Activities of Daily Living Inventory (ADCS-ADL) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment) [ Time Frame: Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment) ]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. The ADCS-ADL assesses functional capacity across a wide spectrum of severity
- Clinical Dementia Rating Scale - Sum of Boxes (CDR-SB) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment) [ Time Frame: Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment) ]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). The Sum of Boxes score (CDR-SB) score was tallied for each administration using the rules from the Washington University Knight ADRD scoring algorithm. Scores range from 0-18. The higher the score, the worse the impairment.
- Trail Making Test - Part A (TMT-A) From Baseline to End of Treatment (3 Weeks), Follow-Up 1 (45 Days Post Treatment) and Follow-Up 2 (90 Days Post Treatment) [ Time Frame: Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment) ]The Trail Making Test- part A (TMT-A) is a assessment of psychomotor speed and is a timed test in which participants must connect a series of numbers randomly placed on a page. Time range is between 0 and 150 seconds, with higher score representing worse performance.

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Ages Eligible for Study: | 55 Years to 85 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- age 55 to 85 years;
- should have a mild-to-moderate AD diagnosis (MMSE 10-26 inclusive);
- should have evidence of elevated cortical amyloid by PET using florbetapir F18 (Amyvid) [i.e. a positive scan], assessed qualitatively according to the Amyvid product label.
- if on anti-dementia treatment should be on stable treatment for at least 2 months (i.e. cholinesterase inhibitor and/or Memantine or Axona);
- stable on all other medications for at least 30 days prior to screen;
- should be fluent in English;
- should be physically able to participate by medical history, clinical exam and tests;
- should have a study partner to accompany them to scheduled visits.
Exclusion Criteria:
- clinically relevant arrhythmias;
- a resting pulse less than 50;
- active cancer other than non-melanoma skin cancers;
- use of another investigatory drug within 2 months of screening;
- significant stroke or head trauma by history or MRI;
- contraindication for having a MRI;
- diagnostic and Statistical Manual of Mental Disorders-IV criteria for a current major psychiatric disorder;
- sensitivity to yeast or yeast products;
- impaired kidney function as measured by a Glomerular Filtration Rate less than 60 milliliters/min;
- preexisting fluid retention, pulmonary infiltrates, or congestive heart failure;
- history of moderate-to-severe lung disease;
- history of moderate-to-severe liver disease;
- pregnant women, or any women who feel they are likely to become pregnant during the study;
- prisoners.

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): NCT01409915
United States, Colorado | |
University of Colorado Denver, Anschutz Medical Campus | |
Aurora, Colorado, United States, 80045 |
Principal Investigator: | Huntington Potter, PhD | University of Colorado, Denver |
Documents provided by University of Colorado, Denver:
Responsible Party: | University of Colorado, Denver |
ClinicalTrials.gov Identifier: | NCT01409915 |
Other Study ID Numbers: |
12-1273 |
First Posted: | August 4, 2011 Key Record Dates |
Results First Posted: | March 23, 2021 |
Last Update Posted: | June 2, 2021 |
Last Verified: | May 2021 |
Alzheimer's disease neuropsychological assessment Granulocyte-Macrophage Colony-Stimulating Factor Leukine |
Alzheimer Disease Brain Diseases Central Nervous System Diseases Nervous System Diseases Neurodegenerative Diseases Neurocognitive Disorders Mental Disorders |
Molgramostim Sargramostim Dementia Tauopathies Immunologic Factors Physiological Effects of Drugs Antineoplastic Agents |