Open-Label Extension of the PTI-125-04 Study in Mild-to-Moderate Alzheimer's Disease

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.

Study Overview

• Status: Terminated
• Conditions: Alzheimer's Disease
• Intervention / Treatment: Drug: simufilam

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 Type: Interventional
• Enrollment (Actual): 90
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Ottawa, Ontario, Canada, K1Z1G3
      • Recherches Neuro-Hippocampe Inc., d/b/a Ottawa Memory Clinic
    • Toronto, Ontario, Canada, M3B 2S7
      • Toronto Memory Program ULC
• United States
  • Arizona
    • Gilbert, Arizona, United States, 85296
      • Cognitive Clinical Trials
  • California
    • Imperial, California, United States, 92251
      • Valley Research Center, Inc.
  • Florida
    • Delray Beach, Florida, United States, 33445
      • Brain Matters Research
    • Fort Myers, Florida, United States, 33912
      • Neuropsychiatric Research Center of Southwest Florida
    • Palmetto Bay, Florida, United States, 33157
      • IMIC Research
  • Nebraska
    • Papillion, Nebraska, United States, 68046
      • Cognitive Clinical Trials
  • New Jersey
    • Toms River, New Jersey, United States, 08755
      • Advanced Memory Research Institute of NJ
  • Ohio
    • Columbus, Ohio, United States, 43221
      • The Ohio State University
  • Texas
    • Austin, Texas, United States, 78757
      • Senior Adults Specialty Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Simufilam 100 mg; simufilam 100 mg oral tablets, b.i.d.	Drug: simufilam; simufilam 100 mg oral tablet, twice daily; Other Names: PTI-125

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Event Monitoring	Number of Participants with Adverse Events	Baseline to 96 Weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
NPI	Baseline to 96 Weeks

Collaborators and Investigators

• Sponsor: Cassava Sciences, Inc.
• Investigators: Study Chair: Chris Cook, JD, Chief Operating and Legal Officer

Study record dates

Study Major Dates

• Study Start (Actual): May 12, 2022
• Primary Completion (Actual): December 31, 2024
• Study Completion (Actual): December 31, 2024

Study Registration Dates

• First Submitted: April 4, 2022
• First Submitted That Met QC Criteria: April 26, 2022
• First Posted (Actual): April 29, 2022

Study Record Updates

• Last Update Posted (Estimated): August 15, 2025
• Last Update Submitted That Met QC Criteria: July 30, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Neurocognitive Disorders; Dementia; Tauopathies; Neurodegenerative Diseases; Alzheimer Disease
• Other Study ID Numbers: PTI-125-09

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No