Alzheimer's DiseasePhase 2NCT05352763
Open-Label Extension of the PTI-125-04 Study in Mild-to-Moderate Alzheimer's Disease
A Phase 2 study testing simufilam in people with Alzheimer's Disease, run by Cassava Sciences, Inc. at 11 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Alzheimer's Disease
- What is being tested?
- simufilam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 12 May 2022to 31 Dec 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
- Must have completed the PTI-125-04 study or Week 96 in the PTI-125-09 study.
- 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.
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States9 sites
Senior Adults Specialty Research
Austin, Texas, 78757
the Ohio State University
Columbus, Ohio, 43221
Brain Matters Research
Delray Beach, Florida, 33445
Neuropsychiatric Research Center of Southwest Florida
Fort Myers, Florida, 33912
Cognitive Clinical Trials
Gilbert, Arizona, 85296
Valley Research Center, Inc.
Imperial, California, 92251
IMIC Research
Palmetto Bay, Florida, 33157
Cognitive Clinical Trials
Papillion, Nebraska, 68046
Advanced Memory Research Institute of NJ
Toms River, New Jersey, 08755
Canada2 sites
Recherches Neuro-Hippocampe Inc., d/b/a Ottawa Memory Clinic
Ottawa, Ontario, K1Z1G3
Toronto Memory Program ULC
Toronto, Ontario, M3B 2S7
About this study
Official title: An Open-Label Extension of the PTI-125-04 Study Evaluating the Safety and Long-Term Treatment of Simufilam in Mild-to-Moderate Alzheimer's Disease Patients
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.
Read the 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).
- Conditions
- Alzheimer's Disease
- Being tested
- simufilam
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
simufilamDrug
Also called PTI-125
simufilam 100 mg oral tablet, twice daily
| Study group | Role | What they receive |
|---|---|---|
| Simufilam 100 mg | Experimental | simufilam 100 mg oral tablets, b.i.d. Receives: Drug: simufilam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event Monitoring
Number of Participants with Adverse Events
Measured: Baseline to 96 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2022
- Study start12 May 2022
- Primary completion31 Dec 2024
- Study completion31 Dec 2024
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Alzheimer's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label extension study
- Masking
- None (open label)
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cassava Sciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chris Cook, JD, Study chair, Chief Operating and Legal Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05352763
- Sponsor's protocol ID
- PTI-125-09
- Status last verified
- Jul 2025
- First submitted
- 4 Apr 2022
- Last updated
- 15 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension of the PTI-125-04 Study Evaluating the Safety and Long-Term Treatment of Simufilam in Mild-to-Moderate Alzheimer's Disease Patients
- ClinicalTrials.gov ID
- NCT05352763
- Acronym / sponsor ID
- PTI-125-09
- Conditions
- Alzheimer's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 90 participants (actual)
- Lead sponsor
- Cassava Sciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 May 2022 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 29 Apr 2022 (actual)
- Results
- Posted 15 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label extension study
| Arm | Type | Receives |
|---|---|---|
| Simufilam 100 mg | Experimental | Drug: simufilamsimufilam 100 mg oral tablets, b.i.d. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| simufilamAlso: PTI-125simufilam 100 mg oral tablet, twice daily | Drug | Simufilam 100 mg |
Primary outcome
Adverse Event Monitoring
Time frame: Baseline to 96 Weeks
Number of Participants with Adverse Events
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Alzheimer's Disease
RecruitingPhase 2
Neuroplasticity Enhancement From Cognitive Training Reinforced by Psilocybin
Alzheimer's Disease
Not yet recruiting
Acupressure as Long-term Intervention to Slow the Progression of Early/Mild Alzheimer's Disease
Alzheimer's Disease
RecruitingObservational study
Effectiveness and Safety of Disease-Modifying Therapy for Alzheimer's Disease: A Multicenter Retrospective Study
Alzheimer's Disease (AD)
RecruitingPhase 3
A Phase 3 Study to Evaluate the Safety and Efficacy of KarXT + KarX-EC for the Treatment of Agitation Associated With Alzheimer's Disease (ADAGIO-1)
Alzheimer Disease
RecruitingPhase 3
A Study of Trontinemab in Cognitively Unimpaired Individuals at Risk for Progression to Symptomatic Alzheimer's Disease
Alzheimer's Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chris Cook, JD study chair
Chief Operating and Legal Officer
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing simufilam in people with Alzheimer's Disease, run by Cassava Sciences, Inc. at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Event Monitoring (time frame: Baseline to 96 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2025.