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Simufilam for Alzheimer's Disease (PTI-125 Trial)

Phase 2
Waitlist Available
Research Sponsored by Cassava Sciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up month 12 to mont 18

Summary

This trial tests simufilam pills in patients with mild-to-moderate Alzheimer's disease. The treatment aims to improve memory and thinking by affecting certain brain proteins. The study will monitor safety and effectiveness over an extended period.

Who is the study for?
This trial is for adults aged 50-85 with mild-to-moderate Alzheimer's, living independently or in a setting without full-time nursing care. Participants must be non-smokers for at least 3 years, fluent in English or Spanish, and have certain cognitive scores. They should be generally healthy and not on specific high-dose medications. A caregiver must assist with medication administration.
What is being tested?
The study tests Simufilam (PTI-125) tablets taken twice daily by people who've had previous simufilam treatments or are newly diagnosed with Alzheimer's. After one year of treatment for all, there's a six-month period where half will switch to placebo randomly before another open-label phase.
What are the potential side effects?
While the trial primarily assesses safety over two years, potential side effects aren't detailed here but may include typical drug reactions such as gastrointestinal issues, allergic responses, changes in liver enzymes or blood counts based on its pharmacological class.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~month 12 to mont 18
This trial's timeline: 3 weeks for screening, Varies for treatment, and month 12 to mont 18 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog-11)
Change from baseline in ADAS-Cog-11 during open-label period 1
Change from baseline in CSF P-tau, Total Tau, Abeta42, neurofilament light chain, neurogranin, YKL-40, soluble TREM2 and HMGB1 during first 6 months of open-label period 1
+1 more
Secondary study objectives
Change from baseline in CSF P-tau, Total Tau, Abeta42, neurofilament light chain, neurogranin, YKL-40, soluble TREM2 and HMGB1 during open-label period 1
Change from baseline in plasma P-tau181 during open-label period 1
Change from baseline in plasma SavaDx during open-label period 1
+3 more

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Simufilam 100 mg oral tablets throughoutExperimental Treatment1 Intervention
Simufilam 100 mg oral tablets administered twice daily (BID) for the full 24 months (including the randomized period Month 12 to Month 18)
Group II: Simufilam 100 mg oral tablets / Placebo / Simufilam 100 mg oral tabletsPlacebo Group2 Interventions
This placebo arm is only for Month 12 to Month 18. Day 1 to Month 12, as well as Month 18 to Month 24 are open-label treatment periods of simufilam 100 mg b.i.d. for all subjects.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Simufilam 100 mg oral tablet
2020
Completed Phase 2
~220

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Alzheimer's Disease (AD) include cholinesterase inhibitors (e.g., donepezil, rivastigmine) and NMDA receptor antagonists (e.g., memantine). Cholinesterase inhibitors work by preventing the breakdown of acetylcholine, a neurotransmitter important for learning and memory, thereby improving communication between nerve cells. Memantine regulates glutamate activity to prevent excitotoxicity, which can damage neurons. Simufilam, a newer treatment under study, stabilizes filamin A to reduce neuroinflammation and synaptic dysfunction, addressing underlying pathological processes in AD. These mechanisms are crucial as they aim to alleviate cognitive symptoms and slow disease progression, improving the quality of life for AD patients.
A Risk-Benefit Assessment of Dementia Medications: Systematic Review of the Evidence.

Find a Location

Who is running the clinical trial?

Cassava Sciences, Inc.Lead Sponsor
8 Previous Clinical Trials
3,653 Total Patients Enrolled
National Institute on Aging (NIA)NIH
1,766 Previous Clinical Trials
28,184,620 Total Patients Enrolled
Lindsay Burns, PhDStudy ChairCassava Sciences
2 Previous Clinical Trials
77 Total Patients Enrolled

Media Library

Simufilam (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04388254 — Phase 2
Alzheimer's Disease Research Study Groups: Simufilam 100 mg oral tablets throughout, Simufilam 100 mg oral tablets / Placebo / Simufilam 100 mg oral tablets
Alzheimer's Disease Clinical Trial 2023: Simufilam Highlights & Side Effects. Trial Name: NCT04388254 — Phase 2
Simufilam (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04388254 — Phase 2
Alzheimer's Disease Patient Testimony for trial: Trial Name: NCT04388254 — Phase 2
~40 spots leftby Oct 2025