Simufilam for Alzheimer's Disease

No longer recruiting at 10 trial locations
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Cassava Sciences, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests simufilam, a new treatment for individuals with mild-to-moderate Alzheimer's disease. The goal is to determine the long-term safety of simufilam and monitor any side effects. Participants must have completed a previous study with this medication to join. This trial is crucial for understanding simufilam's effects over an extended period. As a Phase 2 trial, it measures the treatment's effectiveness in an initial, smaller group.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that simufilam is likely to be safe for humans?

Research has shown that simufilam is well-tolerated by people with mild-to-moderate Alzheimer's disease. One study found that simufilam maintained a good safety record. An independent board reviewed the safety data and recommended that the ongoing trials continue without changes. This suggests that simufilam is generally safe for participants. However, while simufilam has a good safety record, it did not achieve key goals for improving memory or daily functioning in some trials. This indicates that while the treatment seems safe, its effectiveness in treating Alzheimer's symptoms is still under investigation.12345

Why do researchers think this study treatment might be promising for Alzheimer's?

Unlike the standard treatments for Alzheimer's disease, which often focus on managing symptoms, simufilam is unique because it targets an underlying biological process. Specifically, simufilam works by stabilizing a protein called filamin A, which researchers believe plays a key role in the dysfunction of neurons in Alzheimer's. This novel approach could potentially slow or even halt the progression of the disease, offering hope beyond the typical symptom relief provided by current medications like donepezil or memantine. Researchers are excited about simufilam because it represents a promising shift towards addressing the root causes of Alzheimer's, rather than just its symptoms.

What evidence suggests that simufilam might be an effective treatment for Alzheimer's?

Research shows that simufilam, which participants in this trial will receive, might help slow memory and thinking problems in people with mild-to-moderate Alzheimer's disease. One study found that people taking simufilam experienced a 38% slower decline in memory and thinking skills over six months compared to those not taking the drug. However, another study did not find significant improvements in memory or daily activities. Despite these mixed results, ongoing trials have found simufilam to be safe, with no major safety concerns. While its effectiveness is still under study, simufilam could potentially help some patients.23467

Who Is on the Research Team?

CC

Chris Cook, JD

Principal Investigator

Chief Operating and Legal Officer

Are You a Good Fit for This Trial?

This clinical trial is open to individuals with mild-to-moderate Alzheimer's Disease who have completed the PTI-125-04 study. It's designed to assess long-term treatment effects and safety over a period of 96 weeks.

Inclusion Criteria

Subjects must have completed the PTI-125-04 study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants receive simufilam 100 mg b.i.d. for long-term treatment and safety evaluation

96 weeks
Clinic visits every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • simufilam
Trial Overview The trial is testing simufilam, given at a dose of 100 mg twice daily (b.i.d.). The focus is on understanding its safety and effectiveness when used over an extended time in those previously involved in the initial Phase 2 study.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Simufilam 100 mgExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Cassava Sciences, Inc.

Lead Sponsor

Trials
9
Recruited
3,400+

Citations

Oral Simufilam in Mild-to-moderate Alzheimer's Disease... Alzheimer's disease dementia; the safety or efficacy of simufilam in people with Alzheimer's disease ... All efficacy data remains blinded; no interim analyses.
Simufilam Fails to Meet Key End Points in Phase 3 ...Topline data from the REFOCUS-ALZ trial showed no significant cognitive or functional benefits with simufilam in patients with mild-to-moderate AD.
Study Details | NCT04994483 | Simufilam 100 mg for Mild- ...A 52-week safety and efficacy study of simufilam (PTI-125) given twice daily to participants with mild-to-moderate Alzheimer's disease (AD) for 52 weeks.
Oral Simufilam Slowed Cognitive Decline in a Randomized ...Simufilam treatment for 6 months slowed cognitive decline by 38% compared to placebo in mild-to-moderate Alzheimer's disease (MMSE 16-26).
Simufilam found safe in review of ongoing Alzheimer's ...After a third review of safety data, an independent board recommended two trials of simufilam for Alzheimer's continue without modification.
Cassava Sciences Announces Completion of an Interim ...Final Clinical Safety Data for Simufilam Are Expected at the Conclusion of the Phase 3 Program. ... simufilam in Alzheimer's disease. This ...
Cassava Sciences Reports Topline Phase 3 REFOCUS- ...Simufilam continued to demonstrate an overall favorable safety profile. “We are disappointed that the results of REFOCUS-ALZ and RETHINK-ALZ ...
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