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A Pilot Clinical Trial of Exendin-4 in Alzheimer's Disease

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ClinicalTrials.gov Identifier: NCT01255163
Recruitment Status : Terminated (AstraZeneca withdrew support for the study.)
First Posted : December 7, 2010
Results First Posted : February 21, 2018
Last Update Posted : February 21, 2018
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute on Aging (NIA) )

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Alzheimer's Disease
Mild Cognitive Impairment
Interventions: Drug: Exendin-4 SC
Drug: Placebo SC

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Recruitment took place between 10/5/2010 and 7/25/2016. A total of 57 participants signed informed consent to undergo screening procedures. Of those, 28 qualified, 1 withdrew prior to randomization, 27 were randomized to exenatide or placebo, and 18 completed the study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The study includes a baseline visit to determine eligibility. Therefore, 57 participants were enrolled (i.e. signed informed consent prior to screening). Of those, 28 qualified, 1 withdrew prior to randomization, 27 were randomized to exenatide or placebo, and 18 completed the study.

Reporting Groups
  Description
Exendin-4 Exenatide 5 mcg or 10 mcg SC twice daily
Placebo Placebo SC twice daily

Participant Flow:   Overall Study
    Exendin-4   Placebo
STARTED   13   14 
COMPLETED   9   9 
NOT COMPLETED   4   5 
Withdrawal by Subject                1                0 
Lost to Follow-up                2                0 
Adverse Event                0                2 
AstraZeneca stopped support of the study                1                3 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The baseline analysis population includes 27 participants who underwent randomization and received at least one dose of experimental medication. Of those 27, we have at least one outcome measure (i.e. at least the 6 months follow-up data point) for 21 participants. Of those 21, 18 completed the study (i.e. reached 18 months).

Reporting Groups
  Description
Exendin-4 Exenatide 5 mcg or 10 mcg SC twice daily
Placebo Placebo SC twice daily
Total Total of all reporting groups

Baseline Measures
   Exendin-4   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 13   14   27 
Age 
[Units: Years]
Mean (Standard Deviation)
 70.46  (7.137)   74.64  (7.034)   72.63  (7.265) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female   6   9   15 
Male   7   5   12 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      0   0.0%      0   0.0%      0   0.0% 
Not Hispanic or Latino      13 100.0%      14 100.0%      27 100.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      1   7.1%      1   3.7% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      1   7.7%      1   7.1%      2   7.4% 
White      12  92.3%      12  85.7%      24  88.9% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
     
United States   13   14   27 
Cerebrospinal fluid amyloid-beta 42 (CSF Abeta42) [1] 
[Units: Pg/dl]
Mean (Standard Deviation)
 158.85  (43.619)   158.86  (33.558)   158.85  (37.963) 
[1] Measured in CSF using Luminex xMAP technology and INNO-BIA Alz Bio3 kits (Research Use Only) provided by Fujirebio
Mini Mental State Examination (MMSE) [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 25.85  (3.997)   24.64  (5.198)   25.22  (4.61) 
[1] Scale range: 0 - 30 points (higher is better)
Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-cog70) [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 14.46  (6.654)   13.64  (6.879)   14.04  (6.653) 
[1] Alzheimer's dementia scale cognitive sub-scale range: 0 - 70 points (higher is worse)
Body Mass Index (BMI) [1] 
[Units: Kg/m^2]
Mean (Standard Deviation)
 26.631  (3.3673)   26.85  (5.2611)   26.744  (4.3687) 
[1] Body Mass Index defined as a person's weight in kilograms (kg) divided by his or her height in meters squared.
Fasting glucose 
[Units: Mg/dl]
Mean (Standard Deviation)
 94.92  (8.311)   90.14  (9.206)   92.44  (8.954) 
Fasting insulin 
[Units: uIU/ml]
Mean (Standard Deviation)
 8.53  (4.4337)   8.1  (4.5361)   8.254  (4.4628) 
Cerebrospinal fluid phospho181-tau (CSF p181-tau) [1] 
[Units: Pg/dl]
Mean (Standard Deviation)
 56.62  (34.606)   40.64  (16.137)   48.33  (27.369) 
[1] Measured in CSF using Luminex xMAP technology and INNO-BIA Alz Bio3 kits (Research Use Only) provided by Fujirebio
Cerebrospinal fluid (CSF) Total Tau [1] 
[Units: Pg/dl]
Mean (Standard Deviation)
 90.38  (53.053)   70.64  (22.802)   80.15  (40.744) 
[1] Measured in CSF using Luminex xMAP technology and INNO-BIA Alz Bio3 kits (Research Use Only) provided by Fujirebio
Clinical Dementia Rating (CDR) global score [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 0.577  (0.2774)   0.607  (0.2129)   0.593  (0.2417) 
[1] Clinical Dementia Rating global score range: 0 (no dementia); 0.5 (Mild Cognitive Impairment); 1 (mild dementia); 2 (moderate dementia); 3 (severe dementia). Higher is worse.
Clinical Dementia Rating (CDR) sum of boxes [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 2.769  (1.775)   3  (1.7759)   2.889  (1.745) 
[1] Sum of the Clinical Dementia Rating "boxes" (memory, orientation, judgment and problem solving, community affairs, home and hobbies, personal care). Each box is rated as 0, 0.5, 1, 2 or 3. Range for the sum of boxes is 0 - 18. Higher scores reflect a greater severity of dementia.
Nausea [1] 
[Units: Participants]
Count of Participants
 0   0   0 
[1] Nausea reported by participant


  Outcome Measures

1.  Primary:   Number of Participants With Incidence of Nausea   [ Time Frame: 18 months ]

2.  Secondary:   Mini Mental State Examination (MMSE)   [ Time Frame: 18 months ]

3.  Secondary:   Alzheimer's Disease Assessment Scale-cognitive Subscale (ADAS-cog70)   [ Time Frame: 18 months ]

4.  Secondary:   Clinical Dementia Rating (CDR) Global Score   [ Time Frame: 18 months ]

5.  Secondary:   Clinical Dementia Rating (CDR) Sum of Boxes   [ Time Frame: 18 months ]

6.  Secondary:   Cerebrospinal Fluid (CSF) Total Tau   [ Time Frame: 18 months ]

7.  Secondary:   Cerebrospinal Fluid phospho181-tau (CSF p181-tau)   [ Time Frame: 18 months ]

8.  Secondary:   Cerebrospinal Fluid Amyloid-beta 42 (CSF Abeta42)   [ Time Frame: 18 months ]

9.  Secondary:   Body Mass Index (BMI)   [ Time Frame: 18 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The study was terminated after AstraZeneca withdrew support. Early termination lead to small number of subjects analyzed.


  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dimitrios Kapogiannis, Clinical Investigator
Organization: National Institute on Aging (NIA/NIH)
phone: 410-350-3953
e-mail: kapogiannisd@mail.nih.gov


Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute on Aging (NIA) )
ClinicalTrials.gov Identifier: NCT01255163     History of Changes
Other Study ID Numbers: 100423
10-AG-0423
First Submitted: December 4, 2010
First Posted: December 7, 2010
Results First Submitted: November 8, 2017
Results First Posted: February 21, 2018
Last Update Posted: February 21, 2018