What is the mechanism of action of this treatment?

Common treatments for Alzheimer's Disease include cholinesterase inhibitors and NMDA receptor antagonists. Cholinesterase inhibitors, such as donepezil, rivastigmine, and galantamine, increase acetylcholine levels in the brain by inhibiting its breakdown, thereby improving memory and learning. NMDA receptor antagonists like memantine protect neurons by blocking excessive glutamate signaling, which can cause neuronal damage. These treatments are important for Alzheimer's patients as they aim to enhance cognitive function and slow disease progression, similar to the potential benefits being studied for Simufilam (PTI-125).

What side effects are associated with this type of intervention?

Common treatments for Alzheimer's Disease include cholinesterase inhibitors (donepezil, rivastigmine, galantamine) and the NMDA receptor antagonist memantine. Cholinesterase inhibitors can cause side effects such as nausea, vomiting, diarrhea, muscle cramps, and loss of appetite. Memantine is generally well-tolerated but can cause dizziness, headache, confusion, and constipation. These treatments aim to improve cognitive function and slow disease progression, similar to the goals of Simufilam.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Alzheimer's Disease that aim to improve cognitive function and slow disease progression. Notably, acetylcholinesterase inhibitors like donepezil and galantamine have shown promise in improving cognitive symptoms. Memantine, an NMDA receptor antagonist, has also been approved for its potential to moderate symptoms and slow progression. Additionally, aducanumab, a monoclonal antibody targeting amyloid-beta plaques, was approved for its disease-modifying potential, despite mixed clinical trial results. These treatments share a common goal with Simufilam (PTI-125) in targeting cognitive decline and disease progression in Alzheimer's patients.

What other types of research exist?

Promising alternatives to Simufilam for Alzheimer's disease include: <ol> <li>Aducanumab (Aduhelm): An FDA-approved monoclonal antibody that targets amyloid-beta plaques, potentially slowing cognitive decline.</li> <li>Lecanemab: Another monoclonal antibody targeting amyloid-beta, currently in late-stage clinical trials.</li> <li>Donanemab: Targets a modified form of amyloid-beta and has shown promise in slowing cognitive decline in clinical trials.</li> <li>Gantenerumab: An investigational monoclonal antibody targeting amyloid-beta, with ongoing clinical trials showing potential benefits.</li> <li>Blarcamesine (Anavex 2-73): A sigma-1 receptor agonist that has shown potential in improving cognitive function and reducing neuroinflammation in clinical trials.</li> </ol>

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Simufilam for Alzheimer's Disease (REFOCUS-ALZ Trial)

Phase 3

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

Do you have early symptoms of dementia or Alzheimer's?

Do you have a study partner who is willing to attend screening with you?

Must not have

Meets National Institute on Aging and Alzheimer's Association Research Framework criteria for individuals in clinical Stage 4 or 5 of the Alzheimer's continuum.

Evidence for AD pathophysiology, confirmed prior to or during screening

Timeline

Screening 30 days

Treatment 12 months

Follow Up 12 months

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called simufilam to see if it can help people with mild-to-moderate Alzheimer's disease by improving their brain function and slowing down the progression of their symptoms.

Who is the study for?

This trial is for people with mild-to-moderate Alzheimer's who've had consistent Alzheimer's medication doses for 3+ months and haven't smoked in 3+ years. Participants need a study partner to attend screenings.

What is being tested?

The trial tests the safety and effectiveness of Simufilam, at either 50 mg or 100 mg doses, compared to a placebo over a period of 76 weeks. The goal is to see if it improves cognition or slows decline in Alzheimer's patients.

What are the potential side effects?

While specific side effects are not listed here, common ones associated with Alzheimer’s treatments include nausea, diarrhea, insomnia, muscle cramps, fatigue and possibly confusion.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

Are you experiencing the initial signs of memory loss or Alzheimer's disease?

Select...

You need to bring someone with you to the screening appointment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

My Alzheimer's is in the moderate to moderately severe stages.

Select...

, and not meeting the study inclusion criteria, was found in 96 of the 377 (25%) individuals screened

Select...

My Alzheimer's medication dose has been the same for the last 12 weeks.

Select...

I have not smoked for at least 3 years.

Timeline

Screening ~ 30 days8 visits

Treatment ~ 12 months8 visits

Follow Up ~ 12 months8 visits

Screening ~ 30 days

Treatment ~ 12 months

Follow Up ~12 months

This trial's timeline: 30 days for screening, 12 months for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in the 12-item Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog12)

Change from baseline in the Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL)

Secondary study objectives

Change from baseline in plasma biomarker SavaDx

Change from baseline in the Clinical Dementia Rating Sum of Boxes (CDR-SB)

Change from baseline in the MMSE

+7 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Simufilam 50 mgExperimental Treatment1 Intervention

Simufilam 50 mg, supplied by Cassava as coated tablets, and taken b.i.d. for 76 weeks

Group II: Simufilam 100 mgExperimental Treatment1 Intervention

Simufilam 100 mg, supplied by Cassava as coated tablets, and taken b.i.d. for 76 weeks

Group III: PlaceboPlacebo Group1 Intervention

Matching placebo, supplied by Cassava as coated tablets, and taken twice daily (b.i.d.) for 76 weeks

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Simufilam

2021

Completed Phase 3

~810

0 / 6Eligibility criteria met

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 include cholinesterase inhibitors and NMDA receptor antagonists. Cholinesterase inhibitors, such as donepezil, rivastigmine, and galantamine, increase acetylcholine levels in the brain by inhibiting its breakdown, thereby improving memory and learning. NMDA receptor antagonists like memantine protect neurons by blocking excessive glutamate signaling, which can cause neuronal damage. These treatments are important for Alzheimer's patients as they aim to enhance cognitive function and slow disease progression, similar to the potential benefits being studied for Simufilam (PTI-125).

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