Trial Outcomes & Findings for PTI-125 for Mild-to-moderate Alzheimer's Disease Patients (NCT NCT03748706)
NCT ID: NCT03748706
Last Updated: 2021-07-07
Results Overview
Blood draws will be done to evaluate levels of PTI-125 in the plasma using non-compartmental methods in WinNonlin.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-dose
Results posted on
2021-07-07
Participant Flow
Participant milestones
| Measure |
PTI-125
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
PTI-125
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
PTI-125 for Mild-to-moderate Alzheimer's Disease Patients
Baseline characteristics by cohort
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Age, Continuous
|
67.8 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
13 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-doseBlood draws will be done to evaluate levels of PTI-125 in the plasma using non-compartmental methods in WinNonlin.
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Maximum Plasma Concentration (Cmax)
Day 1 Cmax
|
1020 ng/mL
Standard Deviation 442
|
|
Maximum Plasma Concentration (Cmax)
Day 28 Cmax
|
1100 ng/mL
Standard Deviation 417
|
PRIMARY outcome
Timeframe: Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-doseLevels of PTI-125 will be assessed to determine how long it takes to reach the Cmax
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Time to Maximum Plasma Concentration (Tmax)
Day 1 Tmax
|
2.00 hours
Interval 1.0 to 3.0
|
|
Time to Maximum Plasma Concentration (Tmax)
Day 28 Tmax
|
2.06 hours
Interval 1.0 to 5.93
|
PRIMARY outcome
Timeframe: Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-doseLevels of PTI-125 will be assessed to determine the last time point where PTI-125 can be detected.
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Last Quantifiable Plasma Concentration (Clast)
Day 1 Clast
|
176 ng/mL
Standard Deviation 112
|
|
Last Quantifiable Plasma Concentration (Clast)
Day 28 Clast
|
238 ng/mL
Standard Deviation 168
|
PRIMARY outcome
Timeframe: Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-doseLevels of PTI-125 will be assessed to determine the elapsed time to where PTI-125 can last be detected in the plasma.
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Time to Last Quantifiable Plasma Concentration (Tlast)
Day 1 Tlast
|
12.0 hours
Standard Deviation 0.0150
|
|
Time to Last Quantifiable Plasma Concentration (Tlast)
Day 28 Tlast
|
12.0 hours
Standard Deviation 0.0285
|
PRIMARY outcome
Timeframe: Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-doseAUC for PTI-125 plasma concentration from time zero to the last quantifiable plasma concentration.
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Area Under the Curve (AUClast)
Day 1 AUClast
|
5320 h*ng/mL
Standard Deviation 2230
|
|
Area Under the Curve (AUClast)
Day 28 AUClast
|
6700 h*ng/mL
Standard Deviation 3240
|
PRIMARY outcome
Timeframe: Study Day 1 and Day 28 at 20, 40, and 60 min and at 1.5, 2, 2.5, 3, 4, 6, 8, 10 and 12 h post-doseAssessment of the half-life in plasma of PTI-125
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
Plasma Half-life (T1/2)
Day 28 T1/2
|
4.35 hours
Standard Deviation 1.39
|
|
Plasma Half-life (T1/2)
Day 1 T1/2
|
4.51 hours
Standard Deviation 2.43
|
SECONDARY outcome
Timeframe: Study Day 1 and Day 28Blood samples will be tested for the complementary diagnostic/biomarker for Alzheimer's disease.
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
SavaDx (Biomarker)
|
-39.8 % change from baseline
Standard Deviation 0.19
|
SECONDARY outcome
Timeframe: Change from Baseline to Day 28A cerebrospinal fluid sample collection will be performed for Aβ42, tau, YKL40 and other potential CSF biomarkers
Outcome measures
| Measure |
PTI-125
n=13 Participants
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
CSF Biomarkers
Total tau
|
-19.8 % change from baseline
Standard Deviation 0.04
|
|
CSF Biomarkers
Abeta42
|
4.3 % change from baseline
Standard Deviation 0.05
|
|
CSF Biomarkers
p-tau181
|
-34.4 % change from baseline
Standard Deviation 0.05
|
|
CSF Biomarkers
Neurogranin
|
-32 % change from baseline
Standard Deviation 0.02
|
|
CSF Biomarkers
Neurofilament light chain
|
-22 % change from baseline
Standard Deviation 0.02
|
|
CSF Biomarkers
YKL-40
|
-9 % change from baseline
Standard Deviation 0.01
|
|
CSF Biomarkers
IL-6
|
-14 % change from baseline
Standard Deviation 0.01
|
|
CSF Biomarkers
IL-1 beta
|
-11 % change from baseline
Standard Deviation 0.01
|
|
CSF Biomarkers
TNF alpha
|
-5 % change from baseline
Standard Deviation 0.01
|
Adverse Events
PTI-125
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
PTI-125
n=13 participants at risk
PTI-125 100 mg oral tablets administered twice daily (BID)
PTI-125, 100 mg tablets: PTI-125, 100 mg tablets taken twice a day for 28 days
|
|---|---|
|
General disorders
Fall
|
7.7%
1/13 • Number of events 1 • Within the 28-day treatment period.
Moderate; mild; severe Serious; not serious Unlikely; likely; possibly related to drug treatment
|
|
Metabolism and nutrition disorders
B12 deficiency
|
7.7%
1/13 • Number of events 1 • Within the 28-day treatment period.
Moderate; mild; severe Serious; not serious Unlikely; likely; possibly related to drug treatment
|
|
Musculoskeletal and connective tissue disorders
Hypercalcemia
|
7.7%
1/13 • Number of events 1 • Within the 28-day treatment period.
Moderate; mild; severe Serious; not serious Unlikely; likely; possibly related to drug treatment
|
|
Renal and urinary disorders
Renal colic
|
7.7%
1/13 • Number of events 1 • Within the 28-day treatment period.
Moderate; mild; severe Serious; not serious Unlikely; likely; possibly related to drug treatment
|
|
General disorders
Dehydration
|
7.7%
1/13 • Number of events 1 • Within the 28-day treatment period.
Moderate; mild; severe Serious; not serious Unlikely; likely; possibly related to drug treatment
|
|
Respiratory, thoracic and mediastinal disorders
Flu
|
7.7%
1/13 • Number of events 1 • Within the 28-day treatment period.
Moderate; mild; severe Serious; not serious Unlikely; likely; possibly related to drug treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Top enrolling PIs are included as authors on Cassava publications, but no individual PI has the right to publish alone without Cassava authorization.
- Publication restrictions are in place
Restriction type: OTHER