Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of AstroStem in Treatment of Alzheimer's Disease (NCT NCT03117738)
NCT ID: NCT03117738
Last Updated: 2021-08-10
Results Overview
Number of subjects with treatment related adverse events as assessed by analysis of adverse events including symptoms, and abnormal findings on physical examination, vital signs, ECG, and standard laboratory examination results
COMPLETED
PHASE1/PHASE2
21 participants
30 Weeks
2021-08-10
Participant Flow
Participant milestones
| Measure |
AstroStem
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC) 2x10e8 cells
|
Placebo-Control
Saline with 30% auto-serum
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
10
|
|
Overall Study
COMPLETED
|
7
|
5
|
|
Overall Study
NOT COMPLETED
|
4
|
5
|
Reasons for withdrawal
| Measure |
AstroStem
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC) 2x10e8 cells
|
Placebo-Control
Saline with 30% auto-serum
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
3
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
4
|
Baseline Characteristics
A Study to Evaluate the Safety and Efficacy of AstroStem in Treatment of Alzheimer's Disease
Baseline characteristics by cohort
| Measure |
AstroStem
n=11 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=10 Participants
Placebo-Control: Saline with 30% auto-serum
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
72.4 years
STANDARD_DEVIATION 7.6 • n=99 Participants
|
75.6 years
STANDARD_DEVIATION 8.2 • n=107 Participants
|
73.9 years
STANDARD_DEVIATION 7.6 • n=206 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Not-Hispanic or Latino
|
9 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
White
|
10 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Height
|
167.9 cm
STANDARD_DEVIATION 14.4 • n=99 Participants
|
168.3 cm
STANDARD_DEVIATION 9.7 • n=107 Participants
|
168.1 cm
STANDARD_DEVIATION 12.1 • n=206 Participants
|
|
Weight
|
74.8 kg
STANDARD_DEVIATION 17.2 • n=99 Participants
|
64.6 kg
STANDARD_DEVIATION 12.0 • n=107 Participants
|
69.9 kg
STANDARD_DEVIATION 15.5 • n=206 Participants
|
|
BMI
|
26.3 kg/m^2
STANDARD_DEVIATION 4.3 • n=99 Participants
|
22.8 kg/m^2
STANDARD_DEVIATION 3.5 • n=107 Participants
|
24.6 kg/m^2
STANDARD_DEVIATION 4.3 • n=206 Participants
|
PRIMARY outcome
Timeframe: 30 WeeksNumber of subjects with treatment related adverse events as assessed by analysis of adverse events including symptoms, and abnormal findings on physical examination, vital signs, ECG, and standard laboratory examination results
Outcome measures
| Measure |
AstroStem
n=11 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=10 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
Treatment Related Adverse Events
|
6 Participants
|
3 Participants
|
PRIMARY outcome
Timeframe: Baseline and 30 WeeksPopulation: 3 of the 11 astrostem groups were dropped out and 1 was violated protocol. 1 of the 10 Placebo-control groups was dropped out and 2 were violated protocol.
Change of ADAS-Cog (Alzheimer's Disease Assessment Scale-cognitive subscale) from Baseline at Week 30 Score range: 0-70 A score of 70 represents the most severe impairment and 0 represents the least impairment
Outcome measures
| Measure |
AstroStem
n=7 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=7 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
ADAS-Cog (Alzheimer's Disease Assessment Scale-cognitive Subscale)
|
5.9 score on a scale
Standard Deviation 6.8
|
3.0 score on a scale
Standard Deviation 5.4
|
SECONDARY outcome
Timeframe: Baseline and 30 WeeksPopulation: 3 of the 11 astrostem groups were dropped out and 1 was violated protocol. 1 of the 10 Placebo-control groups was dropped out and 2 were violated protocol.
Change of MMSE from baseline at Week 30 Score range: 0-30 A score of 20 to 24 suggests mild dementia, 13 to 20 suggests moderate dementia, and less than 12 indicates severe dementia.
Outcome measures
| Measure |
AstroStem
n=7 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=7 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
MMSE (Mini-mental Status Examination)
|
-3.4 score on a scale
Standard Deviation 2.8
|
-1.4 score on a scale
Standard Deviation 2.8
|
SECONDARY outcome
Timeframe: Baseline and 30 WeeksPopulation: 3 of the 11 astrostem groups were dropped out and 1 was violated protocol. 1 of the 10 Placebo-control groups was dropped out and 2 were violated protocol.
Changes of CDR-SOB from baseline at Week 30 Score range: 0-18.0 0 = normal, 0.5-4.0 = questionable cognitive dementia, 4.5-9.0 = mild dementia, 9.5-15.5 = moderate dementia, and 16.0-18.0 = severe dementia
Outcome measures
| Measure |
AstroStem
n=7 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=7 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
CDR-SOB (Clinical Dementia Rating-Sum of Boxes)
|
1.3 score on a scale
Standard Deviation 1.7
|
0.9 score on a scale
Standard Deviation 1.5
|
SECONDARY outcome
Timeframe: Baseline and 30 WeeksPopulation: 3 of the 11 astrostem groups were dropped out and 1 was violated protocol. 1 of the 10 Placebo-control groups was dropped out and 2 were violated protocol.
Changes of NPI from baseline at Week 30 Score range: 0-144 higher score indicates higher disturbance.
Outcome measures
| Measure |
AstroStem
n=7 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=7 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
NPI (Neuropsychiatric Inventory)
|
-1.4 score on a scale
Standard Deviation 8.5
|
-5.7 score on a scale
Standard Deviation 11.5
|
SECONDARY outcome
Timeframe: Baseline and 30 WeeksPopulation: 3 of the 11 astrostem groups were dropped out and 1 was violated protocol. 1 of the 10 Placebo-control groups was dropped out and 2 were violated protocol.
Changes of GDS from baseline at Week 30 Score range: 0-15 Scores of 0-4 are considered normal, depending on age, education, and complaints; 5-8 indicate mild depression; 9-11 indicate moderate depression; and 12-15 indicate severe depression.
Outcome measures
| Measure |
AstroStem
n=7 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=7 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
GDS (Geriatric Depression Scale)
|
-0.3 score on a scale
Standard Deviation 1.3
|
0.3 score on a scale
Standard Deviation 2.1
|
SECONDARY outcome
Timeframe: Baseline and 30 WeeksPopulation: 3 of the 11 astrostem groups were dropped out and 1 was violated protocol. 1 of the 10 Placebo-control groups was dropped out and 2 were violated protocol.
Change of ADCS-ADL from baseline at Week 30 Score range: 0-78 Lower score indicates greater severity.
Outcome measures
| Measure |
AstroStem
n=7 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=7 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
ADCS-ADL (Alzheimer's Disease Cooperative Study Activities of Daily Living)
|
-4.4 score on a scale
Standard Deviation 9.0
|
-4.7 score on a scale
Standard Deviation 9.8
|
SECONDARY outcome
Timeframe: Baseline and 30 WeeksChanges of C-SSRS from baseline at Week 30 Score range: 0-25 Higher score indicates higher severity.
Outcome measures
| Measure |
AstroStem
n=11 Participants
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=10 Participants
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
C-SSRS (Columbia Suicide Severity Rating Scale)
|
-2.1 score on a scale
Standard Deviation 6.6
|
0.0 score on a scale
Standard Deviation 0.0
|
Adverse Events
AstroStem
Placebo-Control
Serious adverse events
| Measure |
AstroStem
n=11 participants at risk
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=10 participants at risk
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
oesophageal squamous cell carcinoma stage IV
|
9.1%
1/11 • Number of events 1 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Gastrointestinal disorders
diarrhea
|
9.1%
1/11 • Number of events 1 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary embolism
|
9.1%
1/11 • Number of events 1 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Infections and infestations
Acute Pulmonary Oedema
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • Number of events 1 • 52 weeks
|
Other adverse events
| Measure |
AstroStem
n=11 participants at risk
AstroStem: Autologous adipose tissue derived mesenchymal stem cells (AdMSC)
|
Placebo-Control
n=10 participants at risk
Placebo-Control: Saline with 30% auto-serum
|
|---|---|---|
|
Eye disorders
Open Angle Glaucoma
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Gastrointestinal disorders
Dysphagia
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Gastrointestinal disorders
Gastrointestinal Reflux Disease
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
General disorders
Fatigue
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
General disorders
Infusion Site Reaction
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
General disorders
Oedema Peripheral
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
General disorders
Pain
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
General disorders
Pyrexia
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
Injury, poisoning and procedural complications
Fall
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Metabolism and nutrition disorders
Dehydration
|
9.1%
1/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
Metabolism and nutrition disorders
Abnormal Loss of Weight
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
Nervous system disorders
Syncope
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Psychiatric disorders
Agitation
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
9.1%
1/11 • 52 weeks
|
0.00%
0/10 • 52 weeks
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
|
Infections and infestations
Pneumonia
|
0.00%
0/11 • 52 weeks
|
10.0%
1/10 • 52 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place