Trial Outcomes & Findings for Study of Azeliragon in Patients With Mild Alzheimer's Disease and Impaired Glucose Tolerance (NCT NCT03980730)
NCT ID: NCT03980730
Last Updated: 2022-01-21
Results Overview
The Alzheimer's Disease Assessment Scale - Cognitive Subscale 14 Item (ADAS-cog14) scoring range is from 0 to 90, with higher scores indicating greater cognitive impairment
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
43 participants
Primary outcome timeframe
Baseline to Month 6
Results posted on
2022-01-21
Participant Flow
Participant milestones
| Measure |
Azeliragon
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Overall Study
STARTED
|
21
|
22
|
|
Overall Study
COMPLETED
|
19
|
18
|
|
Overall Study
NOT COMPLETED
|
2
|
4
|
Reasons for withdrawal
| Measure |
Azeliragon
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Overall Study
Study Terminated by Sponsor
|
0
|
3
|
|
Overall Study
Adverse Event
|
2
|
0
|
|
Overall Study
Sponsor Request
|
0
|
1
|
Baseline Characteristics
Study of Azeliragon in Patients With Mild Alzheimer's Disease and Impaired Glucose Tolerance
Baseline characteristics by cohort
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
Total
n=43 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
76.3 years
STANDARD_DEVIATION 5.82 • n=99 Participants
|
77.0 years
STANDARD_DEVIATION 6.56 • n=107 Participants
|
76.7 years
STANDARD_DEVIATION 6.14 • n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=99 Participants
|
22 Participants
n=107 Participants
|
41 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=99 Participants
|
21 Participants
n=107 Participants
|
41 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Years Since Diagnosis of AD
|
1.9 years
STANDARD_DEVIATION 1.16 • n=99 Participants
|
1.8 years
STANDARD_DEVIATION 1.24 • n=107 Participants
|
1.9 years
STANDARD_DEVIATION 1.19 • n=206 Participants
|
|
Education Level
Some College
|
5 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Education Level
Other (Training, Certifications, etc.)
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Education Level
Master's Degree
|
1 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Education Level
High School
|
6 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
14 Participants
n=206 Participants
|
|
Education Level
Associate's Degree
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Education Level
Bachelor's Degree
|
7 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
ApoE4 Carrier Status
Non-Carrier
|
9 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
|
ApoE4 Carrier Status
Carrier
|
12 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
22 Participants
n=206 Participants
|
|
ADAS-Cog 14 Total Score
|
30.8 units on a scale
STANDARD_DEVIATION 7.53 • n=99 Participants
|
29.4 units on a scale
STANDARD_DEVIATION 8.87 • n=107 Participants
|
30.1 units on a scale
STANDARD_DEVIATION 8.17 • n=206 Participants
|
|
CDR Global Score
CDR Global = 1
|
11 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
CDR Global Score
CDR Global = 0.5
|
10 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
CDR Sum of Boxes
|
4.7 units on a scale
STANDARD_DEVIATION 1.37 • n=99 Participants
|
4.2 units on a scale
STANDARD_DEVIATION 2.22 • n=107 Participants
|
4.5 units on a scale
STANDARD_DEVIATION 1.85 • n=206 Participants
|
|
MMSE Total Score
|
23.7 units on a scale
STANDARD_DEVIATION 1.48 • n=99 Participants
|
23.5 units on a scale
STANDARD_DEVIATION 2.24 • n=107 Participants
|
23.6 units on a scale
STANDARD_DEVIATION 1.89 • n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline to Month 6Population: The full analysis set (FAS) includes all randomized subjects, whether or not they receive any study medication, and who have at least one post-Baseline assessment. The FAS was used for all hypothesis tests of efficacy.
The Alzheimer's Disease Assessment Scale - Cognitive Subscale 14 Item (ADAS-cog14) scoring range is from 0 to 90, with higher scores indicating greater cognitive impairment
Outcome measures
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Part 1 : Change From Baseline in the Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-cog14) at Month 6
|
1.8 score on a scale
Standard Error 0.82
|
0.4 score on a scale
Standard Error 0.69
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: The full analysis set (FAS) includes all randomized subjects, whether or not they receive any study medication, and who have at least one post-Baseline assessment.
The Amsterdam-Instrumental Activities of Daily Living score is calculated from a questionnaire consisting of 70 items in seven categories using an item response theory method of scoring. Scores range from 20 to 80 where lower scores indicate greater functional impairment.
Outcome measures
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Part 1: Change From Baseline in the Amsterdam-Instrumental Activities of Daily Living (Amsterdam-IADL) at Month 6
|
6.2 score on a scale
Standard Error 1.99
|
9.3 score on a scale
Standard Error 2.37
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: The full analysis set (FAS) includes all randomized subjects, whether or not they receive any study medication, and who have at least one post-Baseline assessment.
Clinical Dementia Rating - Sum of box (CDR-sb) scores range from 0 to 18 with higher scores indicating greater cognitive impairment.
Outcome measures
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Part 1: Change From Baseline in the Clinical Dementia Rating Scale-Sum of Boxes (CDR-sb) at Month 6
|
0.6 score on a scale
Standard Error 0.29
|
1.3 score on a scale
Standard Error 0.36
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: The full analysis set (FAS) includes all randomized subjects, whether or not they receive any study medication, and who have at least one post-Baseline assessment.
The total FAQ score ranges from 0 to 30, with higher scores indicating greater functional loss.
Outcome measures
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Part 1: Change From Baseline in the Functional Activities Questionnaire (FAQ) at Month 6
|
-0.5 score on a scale
Standard Error 0.84
|
2.8 score on a scale
Standard Error 0.84
|
SECONDARY outcome
Timeframe: Baseline to Month 6Population: Full Analysis Set
The MMSE scale range is 0 to 30 with lower scores indicating greater cognitive impairment.
Outcome measures
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Mean Change From Baseline in the Mini Mental State Exam (MMSE) at Month 6.
|
-1.8 score on a scale
Standard Error 0.41
|
-1.7 score on a scale
Standard Error 0.43
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline to Month 6Population: The safety set (SAF) includes all subjects who receive any study medication.
Outcome measures
| Measure |
Azeliragon
n=21 Participants
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 Participants
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Count and Percentage of Subjects With Treatment-emergent Adverse Events
Subjects with at least 1 adverse event
|
16 Participants
|
16 Participants
|
|
Count and Percentage of Subjects With Treatment-emergent Adverse Events
Subjects with at least 1 serious adverse event
|
1 Participants
|
0 Participants
|
|
Count and Percentage of Subjects With Treatment-emergent Adverse Events
Subjects who had drug withdrawn due to AE
|
2 Participants
|
0 Participants
|
Adverse Events
Azeliragon
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Azeliragon
n=21 participants at risk
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 participants at risk
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
General disorders
Gait disturbance
|
4.8%
1/21 • Number of events 1 • 6 months
|
0.00%
0/22 • 6 months
|
Other adverse events
| Measure |
Azeliragon
n=21 participants at risk
Azeliragon 5mg capsule administered orally, once daily for 6 months (Part 1)
Azeliragon: Azeliragon 5 mg capsule administered orally, once daily
|
Placebo
n=22 participants at risk
Matching placebo capsule administered orally, once daily for 6 months (Part 1)
Placebo: Matching placebo capsule administered orally, once daily
|
|---|---|---|
|
Infections and infestations
Urinary tract infection
|
9.5%
2/21 • 6 months
|
9.1%
2/22 • 6 months
|
|
Injury, poisoning and procedural complications
Fall
|
14.3%
3/21 • 6 months
|
13.6%
3/22 • 6 months
|
|
Metabolism and nutrition disorders
Diabetes mellitus
|
4.8%
1/21 • 6 months
|
18.2%
4/22 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/21 • 6 months
|
9.1%
2/22 • 6 months
|
|
Nervous system disorders
Cognitive disorder
|
0.00%
0/21 • 6 months
|
9.1%
2/22 • 6 months
|
|
Gastrointestinal disorders
Diarrhoea
|
9.5%
2/21 • 6 months
|
4.5%
1/22 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.5%
2/21 • 6 months
|
4.5%
1/22 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Where PI requires the use of the Study Results for publication, the PI shall seek the Sponsor's written approval which shall not be unreasonably withheld; provided, however, that (i) Sponsor may require removal of any Confidential Information of Sponsor or may delay publication for a reasonable period of time in order to secure protection any IP Rights; and, (ii) as the Study is designed as a multi-center Study, no publication shall be made until after the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER