We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01409915
Recruitment Status : Completed
First Posted : August 4, 2011
Results First Posted : March 23, 2021
Last Update Posted : June 2, 2021
Sponsor:
Collaborator:
The Dana Foundation
Information provided by (Responsible Party):
University of Colorado, Denver

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Alzheimer's Disease
Interventions Drug: Sagramostim
Drug: Saline -- placebo comparator
Enrollment 44
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Saline placebo comparator: subcutaneous injection
Period Title: Overall Study
Started 21 23
Completed 20 20
Not Completed 1 3
Reason Not Completed
Adverse Event             0             2
Withdrawal by Subject             0             1
Protocol Violation             1             0
Arm/Group Title Sagramostim (Leukine) Control Group Total
Hide Arm/Group Description 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Saline placebo comparator: subcutaneous injection Total of all reporting groups
Overall Number of Baseline Participants 20 20 40
Hide Baseline Analysis Population Description
The analysis population is Per Protocol, with all participants enrolled and randomized in the clinical trial, and who complete the treatment without major breaches in the study protocol.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants 20 participants 40 participants
67.10  (6.57) 70.15  (6.42) 68.63  (6.59)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 20 participants 40 participants
Female
12
  60.0%
11
  55.0%
23
  57.5%
Male
8
  40.0%
9
  45.0%
17
  42.5%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race / Ethnicity Number Analyzed 20 participants 20 participants 40 participants
White / Caucasian
20
 100.0%
19
  95.0%
39
  97.5%
Black / African American
0
   0.0%
1
   5.0%
1
   2.5%
Asian / Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Hispanic / Latino
0
   0.0%
0
   0.0%
0
   0.0%
Other
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 20 participants 20 participants 40 participants
20
 100.0%
20
 100.0%
40
 100.0%
Education Level (Mean years)  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants 20 participants 40 participants
15.70  (2.92) 15.80  (2.71) 15.75  (2.78)
1.Primary Outcome
Title Adverse Events (AEs) by Body System
Hide Description 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
Time Frame 20 weeks (From Consent to Follow-up 2)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis set (SAS), all participants enrolled and randomized and who received at least one injection of sargramostim or placebo
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description:
250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks.
Saline placebo comparator: subcutaneous injection
Overall Number of Participants Analyzed 21 22
Measure Type: Number
Unit of Measure: Adverse Events
Cardiovascular AE 5 2
Constitutional 6 5
Dental 1 0
Dermatological 16 5
ENT 2 0
Gastrointestinal 8 5
Musculoskeletal 8 11
Neurological 9 2
Psychological 0 2
Respiratory 4 4
2.Secondary Outcome
Title 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)
Hide Description 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.
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)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population is Per Protocol, with all participants enrolled and randomized in the clinical trial, and who complete the treatment without major breaches in the study protocol.
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description:
250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks.
Saline placebo comparator: subcutaneous injection
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline 17.10  (4.57) 20.75  (4.97)
End of Treatment (3 weeks) 18.55  (4.99) 20.40  (5.28)
Follow-up 1 (45 days post treatment) 18.00  (5.52) 19.90  (5.19)
Follow-up 2 (90 days pot treatment) 17.10  (5.78) 19.40  (5.47)
3.Secondary Outcome
Title 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)
Hide Description 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
Time Frame Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population is Per Protocol, with all participants enrolled and randomized in the clinical trial, and who complete the treatment without major breaches in the study protocol.
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description:
250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks.
Saline placebo comparator: subcutaneous injection
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 43.20  (12.45) 36.20  (12.01)
End of Treatment (3 weeks) 43.54  (12.02) 36.68  (11.63)
Follow-up 1 (45 days post treatment) 47.67  (11.89) 36.33  (9.85)
Follow-up 2 (90 days post treatment) 45.87  (13.21) 36.85  (10.24)
4.Other Pre-specified Outcome
Title 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)
Hide Description 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
Time Frame Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population is Per Protocol, with all participants enrolled and randomized in the clinical trial, and who complete the treatment without major breaches in the study protocol.
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description:
250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks.
Saline placebo comparator: subcutaneous injection
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline 56.50  (12.30) 62.75  (8.98)
End of Treatment (3 weeks) 57.00  (11.93) 61.85  (9.32)
Follow-up 1 (45 days post treatment) 53.35  (14.02) 59.85  (9.05)
Follow-up 2 (90 days post treatment) 53.30  (15.00) 60.30  (9.00)
5.Other Pre-specified Outcome
Title 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)
Hide Description 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.
Time Frame Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population is Per Protocol, with all participants enrolled and randomized in the clinical trial, and who complete the treatment without major breaches in the study protocol.
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description:
250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks.
Saline placebo comparator: subcutaneous injection
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 7.10  (3.32) 6.10  (2.67)
End of Treatment (3 weeks) 7.53  (3.37) 5.95  (2.37)
Follow-up 1 (45 days post treatment) 8.42  (4.22) 6.81  (3.12)
Follow-up 2 (90 days post treatment) 8.57  (4.14) 7.03  (3.27)
6.Other Pre-specified Outcome
Title 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)
Hide Description 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.
Time Frame Baseline to End of Treatment (3 weeks), Follow-Up 1 (45 days post treatment) and Follow-Up 2 (90 days post treatment)
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population is Per Protocol, with all participants enrolled and randomized in the clinical trial, and who complete the treatment without major breaches in the study protocol.
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description:
250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks.
Saline placebo comparator: subcutaneous injection
Overall Number of Participants Analyzed 20 20
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 101.50  (46.17) 84.85  (48.83)
End of Treatment (3 weeks) 92.60  (45.49) 79.40  (44.56)
Follow-up 1 (45 days post treatment) 107.85  (45.81) 84.00  (45.64)
Follow-up 2 (90 days post treatment) 110.35  (45.55) 85.45  (48.10)
Time Frame Adverse events were completed over a 20 week period, from Consent to Follow-up 2
Adverse Event Reporting Description Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
 
Arm/Group Title Sagramostim (Leukine) Control Group
Hide Arm/Group Description 250 mcg /m2/day Leukine subcutaneously for 5 days/week for three weeks. Saline placebo comparator: subcutaneous injection
All-Cause Mortality
Sagramostim (Leukine) Control Group
Affected / at Risk (%) Affected / at Risk (%)
Total   0/21 (0.00%)      0/23 (0.00%)    
Hide Serious Adverse Events
Sagramostim (Leukine) Control Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/21 (4.76%)      1/23 (4.35%)    
Cardiac disorders     
Myocardial Infarction †  1/21 (4.76%)  1 0/23 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Pneumonia †  0/21 (0.00%)  0 1/23 (4.35%)  1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Sagramostim (Leukine) Control Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   19/21 (90.48%)      20/23 (86.96%)    
Gastrointestinal disorders     
Nausea †  3/21 (14.29%)  1/23 (4.35%) 
Diarrhea †  2/21 (9.52%)  3/23 (13.04%) 
General disorders     
Fatigue †  2/21 (9.52%)  4/23 (17.39%) 
Musculoskeletal and connective tissue disorders     
Pain/Myalgia †  4/21 (19.05%)  5/23 (21.74%) 
Fall †  1/21 (4.76%)  4/23 (17.39%) 
Nervous system disorders     
Headache, Benign NOS †  8/21 (38.10%)  2/23 (8.70%) 
Respiratory, thoracic and mediastinal disorders     
Upper Respiratory infection †  3/21 (14.29%)  3/23 (13.04%) 
Skin and subcutaneous tissue disorders     
Injection site reaction †  9/21 (42.86%)  5/23 (21.74%) 
Rash †  3/21 (14.29%)  0/23 (0.00%) 
†
Indicates events were collected by systematic assessment
The main weakness is the small sample size of the study.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: John O'Shaughnessy, MS, Clinical Research Services Principal Professional
Organization: University of Colorado Denver | Anschutz Medical Campus
Phone: 3037247924
EMail: john.oshaughnessy@cuanschutz.edu
Layout table for additonal information
Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT01409915    
Other Study ID Numbers: 12-1273
First Submitted: August 2, 2011
First Posted: August 4, 2011
Results First Submitted: February 25, 2021
Results First Posted: March 23, 2021
Last Update Posted: June 2, 2021