Open-label Extension for Phase 3 Clinical Trials of Simufilam

May 13, 2025 updated by: Cassava Sciences, Inc.

An Open-label, Long-term Extension Study to Evaluate the Safety and Tolerability of Simufilam 100 mg Tablets in Participants With Mild to Moderate Alzheimer's Disease

The goal of this open-label extension study is to assess long-term safety and tolerability of simufilam 100 mg in subjects who have completed the RETHINK-ALZ or REFOCUS-ALZ Phase 3 clinical trials.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

This is a multi-national, multi-center, fixed-dose, 52-week, open-label extension study. After completing participation in either RETHINK-ALZ (PTI-125-06) or REFOCUS-ALZ (PTI-125-07), subjects will have the option to participate in this study. After the subject provides consent and the Investigator confirms the subject satisfies both inclusion and exclusion criteria, the study drug will be administered at the research site on Study Day 1 and subsequent visits will be scheduled.

We anticipate up to 1600 subjects may enroll in this study. Approximately up to 150 clinical sites in the USA, Canada, South Korea, and Australia will have the option to participate in this collaborative research effort.

For subjects electing to participate, the clinical and laboratory assessments from the Week 76 (REFOCUS-ALZ) or Week 52 (RETHINK-ALZ) End-of-Treatment visit will serve as Baseline Visit assessments for the open-label study on Study Day 1. All subjects will return in 4 weeks and every 12 weeks thereafter for safety assessments. At all post-baseline visits, subjects will report any adverse events since their last visit. In addition to adverse event monitoring, safety will be evaluated at every visit by vital signs, brief examinations, clinical laboratory tests (biochemistry, hematology, and urinalysis) and the Columbia Suicide Severity Rating Scale (C-SSRS). Study drug use since the last visit will be assessed and a new bottle of study drug will be dispensed.

The emerging subject safety assessments will be monitored throughout the study by an independent Data Safety Monitoring Board (DSMB).

Study Type

Interventional

Enrollment (Actual)

1081

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Macquarie Park, New South Wales, Australia, 2113
        • KaRa Institute of Neurological Diseases Pty. Ltd.
    • Queensland
      • Herston, Queensland, Australia, 4029
        • The University of Queensland
    • Victoria
      • Glen Iris, Victoria, Australia, 3146
        • Delmont Consulting Suites
      • Heidelberg, Victoria, Australia, 3084
        • Austin Health
      • Melbourne, Victoria, Australia, 3004
        • The Alfred Hospital
      • Melbourne, Victoria, Australia, 3128
        • Eastern Health
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Alzheimer's Research Australia
  • Canada
      • Québec, Canada, G3K 2P8
        • ALPHA Recherche Clinique
    • British Columbia
      • Kelowna, British Columbia, Canada, V1Y 1Z9
        • OCT Research ULC DBA Okanagan Clinical Trials
    • New Brunswick
      • Moncton, New Brunswick, Canada, E1C 2Z3
        • Dr. George Dumont University Hospital Centre
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3S 1N2
        • True North Clinical Research - Halifax/Nova Scotia
      • New Minas, Nova Scotia, Canada, B4N 3R7
        • True North Clinical Research - New Minas
    • Ontario
      • London, Ontario, Canada, N6C 5J1
        • St. Joseph's Health Care London
      • Ottawa, Ontario, Canada, K1Z 1G3
        • Ottawa Memory Clinic
      • Peterborough, Ontario, Canada, K9H2P4
        • Kawartha Centre - Redefining Healthy Aging
      • Toronto, Ontario, Canada, M3B 2S7
        • Toronto Memory Program
      • Toronto, Ontario, Canada, M4G 3E8
        • The Centre for Memory and Aging
    • Quebec
      • Sherbrooke, Quebec, Canada, J1J 2G2
        • Q & T Research
      • Sherbrooke, Quebec, Canada, J1L 0H8
        • Diex Research - Sherbrooke
  • Korea, Republic of
    • Buk-gu
      • Daegu, Buk-gu, Korea, Republic of, 41404
        • Kyungpook National University Chilgok Hospital
    • Dong-gu
      • Gwangju, Dong-gu, Korea, Republic of, 61469
        • Chonnam National University Hospital
    • Gyeonggi-do
      • Seongnam-si, Gyeonggi-do, Korea, Republic of, 13620
        • Seoul National University Bundang Hospital
    • Incheon
      • Namyangju, Incheon, Korea, Republic of, 21565
        • Gachon University Gil Hospital
    • Jung-gu
      • Incheon, Jung-gu, Korea, Republic of, 22332
        • Inha Unviersity Medical Center
  • Puerto Rico
      • Bayamón, Puerto Rico, 00961
        • Santa Cruz Behavioral PSC
      • San Juan, Puerto Rico, 00918
        • INSPIRA Clinical Research
      • San Juan, Puerto Rico, 00926
        • Barbara Diaz Hernandez MD Research, Inc.
  • United States
    • Arizona
      • Chandler, Arizona, United States, 85286
        • MDFirst Research
      • Gilbert, Arizona, United States, 85297
        • CCT Research - Gilbert Neurology Partners
      • Phoenix, Arizona, United States, 85006
        • Banner Alzheimer's Institute
      • Phoenix, Arizona, United States, 85004
        • Xenoscience, Inc.
      • Sun City, Arizona, United States, 85351
        • Banner Sun Health Research Institute
    • California
      • Anaheim, California, United States, 92805
        • Advanced Research Center, Inc.
      • Carlsbad, California, United States, 92011
        • North County Neurology Associates
      • Colton, California, United States, 92324
        • Axiom Research, LLC
      • Costa Mesa, California, United States, 92626
        • ATP Clinical Research, Inc.
      • Fresno, California, United States, 93710
        • Neuro-Pain Medical Center
      • Fullerton, California, United States, 92835
        • Neurology Center of North Orange County
      • Imperial, California, United States, 92251
        • Sun Valley Research Center, Inc.
      • Laguna Hills, California, United States, 92653
        • Senior Clinical Trials
      • Newport Beach, California, United States, 92663
        • Shankle Clinic and Hoag Memorial Hospital Presbyterian
      • Riverside, California, United States, 92503
        • Artemis Institute for Clinical Research
      • San Diego, California, United States, 92103
        • Pacific Research Network, LLC
      • Santa Ana, California, United States, 92705
        • Syrentis Clinical Research
    • Colorado
      • Basalt, Colorado, United States, 81621
        • Mountain Neurological Research Center
      • Denver, Colorado, United States, 80210
        • Colorado Neurological Research Center, PC
    • Connecticut
      • Stamford, Connecticut, United States, 06905
        • KI Health Partners, LLC
    • Florida
      • Atlantis, Florida, United States, 33462
        • JEM Research Institute
      • Boca Raton, Florida, United States, 33428
        • Neurology Offices of South Florida
      • Clermont, Florida, United States, 34711
        • K2 Medical Research- Ocoee
      • Daytona Beach, Florida, United States, 32117
        • Arrow Clinical Trials
      • Delray Beach, Florida, United States, 33445
        • Brain Matters Research
      • Fort Myers, Florida, United States, 33912
        • Neuropsychiatric Research Center of Southwest Florida
      • Hallandale Beach, Florida, United States, 33009
        • Velocity Clinical Research, Hallandale Beach
      • Hialeah, Florida, United States, 33016
        • Galiz Research
      • Hollywood, Florida, United States, 33024
        • Infinity Clinical Research - Sunrise
      • Homestead, Florida, United States, 33030
        • Luminous Clinical Research
      • Jacksonville, Florida, United States, 32256
        • CNS Healthcare - Jacksonville
      • Lady Lake, Florida, United States, 32792
        • Charter Research
      • Maitland, Florida, United States, 32751
        • ClinCloud
      • Merritt Island, Florida, United States, 32952
        • Merritt Island Medical Research, LLC
      • Miami, Florida, United States, 33165
        • New Horizon Research Center
      • Miami, Florida, United States, 33176
        • Brainstorm Research
      • Miami, Florida, United States, 33125
        • Central Miami Medical Institute (GMI)
      • Miami, Florida, United States, 33137
        • Mind Institute at Miami Jewish Health
      • Miami Springs, Florida, United States, 33166
        • South Florida Research Phase I-IV INC
      • New Port Richey, Florida, United States, 34652
        • Suncoast Clinical Research, Inc.
      • Ocala, Florida, United States, 34470
        • Renstar Medical Research
      • Okeechobee, Florida, United States, 34972
        • Health Synergy Clinical Research
      • Orlando, Florida, United States, 32801
        • Clinical Neuroscience Solutions, Inc. dba CNS Healthcare
      • Port Orange, Florida, United States, 32127
        • Medical Research
      • Sarasota, Florida, United States, 34239
        • Intercoastal Medical Group - Sarasota
      • Stuart, Florida, United States, 34997
        • Alzheimer's Research & Treatment Center
      • Tampa, Florida, United States, 33613
        • Stedman Clinical Trials
      • Tampa, Florida, United States, 33603
        • Clinical Research of Brandon, LLC (Tampa)
      • Wellington, Florida, United States, 33414
        • Alzheimer's Research & Treatment Center
      • West Palm Beach, Florida, United States, 33407
        • Premier Research Institute at Palm Beach Neurology
      • Winter Park, Florida, United States, 32792
        • Charter Research
    • Georgia
      • Columbus, Georgia, United States, 31909
        • Columbus Memory Center, PC
      • Decatur, Georgia, United States, 30030
        • Accel Research Sites - Neurostudies
    • Idaho
      • Meridian, Idaho, United States, 83642
        • Velocity Clinical Research - Boise
    • Illinois
      • Park Ridge, Illinois, United States, 60068
        • Advocate Aurora Health - Advocate Memory Center
    • Kansas
      • Wichita, Kansas, United States, 67214
        • Ascension via Christi Research
    • Kentucky
      • Lexington, Kentucky, United States, 40504
        • University of Kentucky
    • Louisiana
      • Alexandria, Louisiana, United States, 71301
        • Neuro Medical Clinic of Central Louisiana, LLC
    • Massachusetts
      • Foxboro, Massachusetts, United States, 02035
        • Neurology Center of New England
      • Methuen, Massachusetts, United States, 01844
        • ActivMed Practices & Research, LLC
      • Newton, Massachusetts, United States, 02459
        • Boston Center for Memory
      • North Dartmouth, Massachusetts, United States, 02747
        • Boston Neuro Research Center
      • Plymouth, Massachusetts, United States, 02360
        • Headlands Eastern MA LLC
      • Waltham, Massachusetts, United States, 02451
        • MedVadis Research
    • Missouri
      • Chesterfield, Missouri, United States, 63005
        • Clinical Research Professionals
    • Nebraska
      • Papillion, Nebraska, United States, 68046
        • CCT Research - Papillion Research Center
    • New Jersey
      • Middletown, New Jersey, United States, 07748
        • Patient First MD
      • Neptune, New Jersey, United States, 07753
        • Advanced Clinical Institute, Inc
      • Princeton, New Jersey, United States, 08540
        • Global Medical Institutes, LLC
      • Springfield, New Jersey, United States, 07081
        • The Cognitive and Research Center of New Jersey (CRCNJ)
      • Toms River, New Jersey, United States, 08755
        • Advanced Memory Research Institute of NJ
      • West Long Branch, New Jersey, United States, 07764
        • Neurology Specialists of Monmouth County
    • New Mexico
      • Albuquerque, New Mexico, United States, 87109
        • Albuquerque Neuroscience, INC
    • New York
      • Albany, New York, United States, 12208
        • Albany Medical Center
      • Albany, New York, United States, 12208
        • Neurological Associates of Albany
      • Amherst, New York, United States, 14226
        • Dent Neurologic Institute
      • Brooklyn, New York, United States, 11229
        • Integrative Clinical Trials, Llc
      • Brooklyn, New York, United States, 11235
        • SPRI Clinical Trials Brooklyn
      • East Syracuse, New York, United States, 13057
        • Velocity Clinical Research, Formerly Clarity Clinical Research
      • New Hyde Park, New York, United States, 11040-1433
        • Parker Jewish Institute for Health Care & Rehabilitation
      • New Windsor, New York, United States, 12553
        • Mid Hudson Medical Research
      • New York, New York, United States, 10003
        • NY Neurology Associates
      • Rochester, New York, United States, 14620
        • University of Rochester Medical Center - Alzheimer's Disease Care, Research and Education Program
      • Staten Island, New York, United States, 10312
        • Richmond Behavioral Associates
      • Staten Island, New York, United States, 10314
        • Richmond Behavioral Associates
    • North Carolina
      • Greensboro, North Carolina, United States, 27410
        • Triad Clinical Trials, LLC
      • Matthew, North Carolina, United States, 28105
        • Alzheimer's Memory Center
      • Winston-Salem, North Carolina, United States, 27103
        • Accellacare Research of Winston-Salem
    • Ohio
      • Beachwood, Ohio, United States, 44122
        • Insight Clinical Trials Llc
      • Canton, Ohio, United States, 44718
        • Neuroscience Research Center, LLC
      • Centerville, Ohio, United States, 45459
        • Dayton Center for Neurological Disorders
      • Columbus, Ohio, United States, 43221
        • The Ohio State University
      • Dayton, Ohio, United States, 45459
        • Neurology Diagnostics
      • North Canton, Ohio, United States, 44720
        • Neuro-Behavioral Clinical Research
    • Oregon
      • Portland, Oregon, United States, 97210
        • Summit Research Network, LLC
      • Portland, Oregon, United States, 97225
        • Center for Cognitive Health - Portland
    • Pennsylvania
      • Plymouth Meeting, Pennsylvania, United States, 19462
        • Keystone Clinical Studies, LLC
    • Rhode Island
      • East Providence, Rhode Island, United States, 02914
        • Rhode Island Mood & Memory Research Institute
    • South Carolina
      • Summerville, South Carolina, United States, 29485
        • Palmetto Clinical Research
    • Texas
      • Austin, Texas, United States, 78757
        • Senior Adults Specialty Research, Inc
      • Dallas, Texas, United States, 75206
        • Texas Neurology, PA
      • Dallas, Texas, United States, 75231
        • Baylor Scott & White Research Institute
      • Katy, Texas, United States, 77450
        • Mt. Olympus Medical Research, LLC
      • Wichita Falls, Texas, United States, 76309
        • Grayline Research Center
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Re:Cognition Health
    • Washington
      • Bellevue, Washington, United States, 98007
        • Northwest Clinical Research Center
      • Seattle, Washington, United States, 98104
        • Memory and Brain Wellness Center at Harborview

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

51 years to 89 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criterion:

  • Completed RETHINK-ALZ (PTI-125-07) or REFOCUS-ALZ (PTI-125-06).
  • Clinical presentation continues to be consistent with Alzheimer's disease.
  • Availability of a study partner.

Exclusion Criteria:

  • Residence in a skilled nursing facility requiring 24-hour care.
  • Evidence of a neurologic condition other than AD that significantly contributes to the subject's dementia.
  • Current clinically significant psychiatric diagnosis other than AD.
  • Unstable, clinically significant medical condition other than AD.
  • Any medical condition that in the opinion of the investigator may affect safety or ability to complete the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Simufilam 100 mg
simufilam 100 mg oral tablet, twice daily
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
Number of Participants With Adverse Events
Time Frame: Baseline to 52 weeks
To evaluate the long-term safety and tolerability of simufilam 100 mg tablets in subjects who have completed one of the two double-blind simufilam phase 3 studies.
Baseline to 52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cassava Sciences, Inc.

Investigators

  • Study Director: James Kupiec, MD, Cassava Sciences, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 7, 2022

Primary Completion (Actual)

January 31, 2025

Study Completion (Actual)

January 31, 2025

Study Registration Dates

First Submitted

October 7, 2022

First Submitted That Met QC Criteria

October 7, 2022

First Posted (Actual)

October 12, 2022

Study Record Updates

Last Update Posted (Actual)

May 23, 2025

Last Update Submitted That Met QC Criteria

May 13, 2025

Last Verified

February 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PTI-125-10

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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