Alzheimer's Disease
Conditions
Brief summary
This is a 96-week extension study of open-label simufilam 100 mg b.i.d. for mild-to-moderate Alzheimer's disease subjects who completed the Phase 2 study, PTI-125-04. The study will evaluate safety and long-term treatment. Safety will be assessed by AE monitoring, clinical labs, urinalysis, vital signs, ECGs, and C-SSRS.
Detailed description
This is an open-label 96-week extension study of open-label simufilam 100 mg b.i.d. for subjects who completed the Phase 2 study, PTI-125-04. All subjects will provide consent to enroll into this study. Simufilam will be administered as coated oral tablets. The last study visit, Month 24, from the PTI-125-04 study will be used for the Study Day 1 visit assessments in this extension study. Clinic visits will occur every 12 weeks ±10 days. A complete physical examination will be performed at Study Day 1, Week 48 and Week 96. Subjects will return to the clinic every 12 weeks for safety assessments of vital signs, AE monitoring, C-SSRS, and drug dispensation and accountability. Blood draws for clinical laboratory testing, urine collection for urinalysis, and ECGs will be performed at Study Day 1 and Weeks 24, 48, 72, and 96. Safety will be evaluated by adverse event monitoring, vital signs, clinical labs, and the Columbia Suicide Severity Rating Scale (C-SSRS).
Interventions
DRUGsimufilam
simufilam 100 mg oral tablet, twice daily
Sponsors
Cassava Sciences, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label extension study
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Must have completed the PTI-125-04 study or Week 96 in the PTI-125-09 study. 2. Male subjects must be willing to continue use of contraception during the study. With female partners of childbearing potential, male subjects, regardless of their fertility status, must agree to either remain abstinent or use condoms in combination with one additional highly effective method of contraception (e.g., oral or implanted contraceptives, or intrauterine devices) or an effective method of contraception (e.g., diaphragms with spermicide or cervical sponges) during the study and for 14 days after study drug dosing has been completed.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Event Monitoring | Baseline to 96 Weeks | Number of Participants with Adverse Events |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Simufilam 100 mg simufilam 100 mg oral tablet, twice daily
simufilam: simufilam 100 mg oral tablets | 90 |
| Total | 90 |
Baseline characteristics
| Characteristic | Simufilam 100 mg |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 71 Participants |
| Age, Categorical Between 18 and 65 years | 19 Participants |
| Age, Continuous | 73.2 years STANDARD_DEVIATION 8.78 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 7 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 83 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 3 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 87 Participants |
| Sex: Female, Male Female | 55 Participants |
| Sex: Female, Male Male | 35 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 90 |
| other Total, other adverse events | 43 / 90 |
| serious Total, serious adverse events | 15 / 90 |
Outcome results
Primary
Adverse Event Monitoring
Number of Participants with Adverse Events
Time frame: Baseline to 96 Weeks
Population: Safety Analysis Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Simufilam 100 mg | Adverse Event Monitoring | 90 Participants |