CompletedPhase 2Results posted

Study of the Safety & Efficacy of Leukine® in the Treatment of Alzheimer's Disease

Pilot Phase 2 Trial of the Safety & Efficacy of Granulocyte-Macrophage Colony-Stimulating Factor (Leukine®) in the Treatment of Alzheimer's Disease

Lead sponsor

University of Colorado, Denver

Asset

Sargramostim

Listed sites

Recruiting sites

Enrollment

actual

Study population

Alzheimer’s disease

Key I/E criteria

•mild-to-moderate AD•Amyloid biomarker required (PET)•MMSE 10-26•Study partner/caregiver required•MRI contraindications excluded

Primary endpoint

•Adverse Events (AEs) by Body System

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

Org study ID12-1273

NCT IDNCT01409915

Timeline

Milestones

Study start2011-03 (month precision)

Study first posted2011-08-04estimated

Primary completion2019-12-09actual

Study completion2019-12-09actual

Results first posted2021-03-23actual

Last update posted2021-06-02actual

Assets

Drug assets

Sargramostim

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age55 Years

Maximum age85 Years

SexAll

Healthy volunteersNot accepted

Inclusion criteria

1. age 55 to 85 years;

2. should have a mild-to-moderate AD diagnosis (MMSE 10-26 inclusive);

3. 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.

4. if on anti-dementia treatment should be on stable treatment for at least 2 months (i.e. cholinesterase inhibitor and/or Memantine or Axona);

5. stable on all other medications for at least 30 days prior to screen;

6. should be fluent in English;

7. should be physically able to participate by medical history, clinical exam and tests;

8. should have a study partner to accompany them to scheduled visits

Exclusion criteria

1. clinically relevant arrhythmias;

2. a resting pulse less than 50;

3. active cancer other than non-melanoma skin cancers;

4. use of another investigatory drug within 2 months of screening;

5. significant stroke or head trauma by history or MRI;

6. contraindication for having a MRI;

7. diagnostic and Statistical Manual of Mental Disorders-IV criteria for a current major psychiatric disorder;

8. sensitivity to yeast or yeast products;

9. impaired kidney function as measured by a Glomerular Filtration Rate less than 60 milliliters/min;

10. preexisting fluid retention, pulmonary infiltrates, or congestive heart failure;

11. history of moderate-to-severe lung disease;

12. history of moderate-to-severe liver disease;

13. pregnant women, or any women who feel they are likely to become pregnant during the study;

14. prisoners.

Endpoints (12)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Global cognition

Executive function / language

Function / daily living

Safety / tolerability / PK

Global cognition

6 endpoints

Secondary/protocol endpoint

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)

descriptive

Secondary/protocol endpoint

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)

ADAS-Cog

descriptive

Secondary/registry result

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)

descriptive

Posted result

Group	Value (mean), score on a scale	Standard deviation
Sagramostim (Leukine)Baselinen=20 Participants	17.10	4.57
End of Treatment (3 weeks)n=20 Participants	18.55	4.99
Follow-up 1 (45 days post treatment)n=20 Participants	18.00	5.52
Follow-up 2 (90 days pot treatment)n=20 Participants	17.10	5.78
Control GroupBaselinen=20 Participants	20.75	4.97
End of Treatment (3 weeks)n=20 Participants	20.40	5.28
Follow-up 1 (45 days post treatment)n=20 Participants	19.90	5.19
Follow-up 2 (90 days pot treatment)n=20 Participants	19.40	5.47

Secondary/registry result

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)

ADAS-Cog

descriptive

Posted result

Group	Value (mean), units on a scale	Standard deviation
Sagramostim (Leukine)Baselinen=20 Participants	43.20	12.45
End of Treatment (3 weeks)n=20 Participants	43.54	12.02
Follow-up 1 (45 days post treatment)n=20 Participants	47.67	11.89
Follow-up 2 (90 days post treatment)n=20 Participants	45.87	13.21
Control GroupBaselinen=20 Participants	36.20	12.01
End of Treatment (3 weeks)n=20 Participants	36.68	11.63
Follow-up 1 (45 days post treatment)n=20 Participants	36.33	9.85
Follow-up 2 (90 days post treatment)n=20 Participants	36.85	10.24

Other/protocol endpoint

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)

Clinical Dementia Rating-Sum of Boxes (CDR-SB)

ratio, descriptive

Other_pre_specified/registry result

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)

Clinical Dementia Rating-Sum of Boxes (CDR-SB)

ratio, descriptive

Posted result

Group	Value (mean), units on a scale	Standard deviation
Sagramostim (Leukine)Baselinen=20 Participants	7.10	3.32
End of Treatment (3 weeks)n=20 Participants	7.53	3.37
Follow-up 1 (45 days post treatment)n=20 Participants	8.42	4.22
Follow-up 2 (90 days post treatment)n=20 Participants	8.57	4.14
Control GroupBaselinen=20 Participants	6.10	2.67
End of Treatment (3 weeks)n=20 Participants	5.95	2.37
Follow-up 1 (45 days post treatment)n=20 Participants	6.81	3.12
Follow-up 2 (90 days post treatment)n=20 Participants	7.03	3.27

Executive function / language

2 endpoints

Other/protocol endpoint

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)

descriptive

Other_pre_specified/registry result

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)

descriptive

Posted result

Group	Value (mean), units on a scale	Standard deviation
Sagramostim (Leukine)Baselinen=20 Participants	101.50	46.17
End of Treatment (3 weeks)n=20 Participants	92.60	45.49
Follow-up 1 (45 days post treatment)n=20 Participants	107.85	45.81
Follow-up 2 (90 days post treatment)n=20 Participants	110.35	45.55
Control GroupBaselinen=20 Participants	84.85	48.83
End of Treatment (3 weeks)n=20 Participants	79.40	44.56
Follow-up 1 (45 days post treatment)n=20 Participants	84.00	45.64
Follow-up 2 (90 days post treatment)n=20 Participants	85.45	48.10

Function / daily living

2 endpoints

Other/protocol endpoint

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)

ADCS-Activities of Daily Living (ADCS-ADL)

descriptive

Other_pre_specified/registry result

Time frame:Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)

ADCS-Activities of Daily Living (ADCS-ADL)

descriptive

Posted result

Group	Value (mean), score on a scale	Standard deviation
Sagramostim (Leukine)Baselinen=20 Participants	56.50	12.30
End of Treatment (3 weeks)n=20 Participants	57.00	11.93
Follow-up 1 (45 days post treatment)n=20 Participants	53.35	14.02
Follow-up 2 (90 days post treatment)n=20 Participants	53.30	15.00
Control GroupBaselinen=20 Participants	62.75	8.98
End of Treatment (3 weeks)n=20 Participants	61.85	9.32
Follow-up 1 (45 days post treatment)n=20 Participants	59.85	9.05
Follow-up 2 (90 days post treatment)n=20 Participants	60.30	9.00

Safety / tolerability / PK

2 endpoints

Primary/protocol endpoint

Adverse Events (AEs) by Body System

Time frame:20 weeks (From Consent to Follow-up 2)

event count, event

Primary/registry result

Adverse Events (AEs) by Body System

Time frame:20 weeks (From Consent to Follow-up 2)

event count, event

Posted result

Group	Value (number), Adverse Events	Reported bounds
Sagramostim (Leukine)Cardiovascular AEn=21 Participants	5	-
Constitutionaln=21 Participants	6	-
Dentaln=21 Participants	1	-
Dermatologicaln=21 Participants	16	-
ENTn=21 Participants	2	-
Gastrointestinaln=21 Participants	8	-
Musculoskeletaln=21 Participants	8	-
Neurologicaln=21 Participants	9	-
Psychologicaln=21 Participants	0	-
Respiratoryn=21 Participants	4	-
Control GroupCardiovascular AEn=22 Participants	2	-
Constitutionaln=22 Participants	5	-
Dentaln=22 Participants	0	-
Dermatologicaln=22 Participants	5	-
ENTn=22 Participants	0	-
Gastrointestinaln=22 Participants	5	-
Musculoskeletaln=22 Participants	11	-
Neurologicaln=22 Participants	2	-
Psychologicaln=22 Participants	2	-
Respiratoryn=22 Participants	4	-

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableClinicalTrials.gov results section

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.