Open-label Extension for Phase 3 Clinical Trials of Simufilam

NCT ID: NCT05575076

Last Updated: 2025-05-23

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

1081 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-07

Study Completion Date

2025-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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).

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Alzheimer Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open-label extension
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Simufilam 100 mg

simufilam 100 mg oral tablet, twice daily

Group Type EXPERIMENTAL

Simufilam

Intervention Type DRUG

simufilam 100 mg oral tablet, twice daily

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Simufilam

simufilam 100 mg oral tablet, twice daily

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

PTI-125

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* 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.
Minimum Eligible Age

51 Years

Maximum Eligible Age

89 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Cassava Sciences, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

James Kupiec, MD

Role: STUDY_DIRECTOR

Cassava Sciences, Inc.

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

MDFirst Research

Chandler, Arizona, United States

Site Status

CCT Research - Gilbert Neurology Partners

Gilbert, Arizona, United States

Site Status

Xenoscience, Inc.

Phoenix, Arizona, United States

Site Status

Banner Alzheimer's Institute

Phoenix, Arizona, United States

Site Status

Banner Sun Health Research Institute

Sun City, Arizona, United States

Site Status

Advanced Research Center, Inc.

Anaheim, California, United States

Site Status

North County Neurology Associates

Carlsbad, California, United States

Site Status

Axiom Research, LLC

Colton, California, United States

Site Status

ATP Clinical Research, Inc.

Costa Mesa, California, United States

Site Status

Neuro-Pain Medical Center

Fresno, California, United States

Site Status

Neurology Center of North Orange County

Fullerton, California, United States

Site Status

Sun Valley Research Center, Inc.

Imperial, California, United States

Site Status

Senior Clinical Trials

Laguna Hills, California, United States

Site Status

Shankle Clinic and Hoag Memorial Hospital Presbyterian

Newport Beach, California, United States

Site Status

Artemis Institute for Clinical Research

Riverside, California, United States

Site Status

Pacific Research Network, LLC

San Diego, California, United States

Site Status

Syrentis Clinical Research

Santa Ana, California, United States

Site Status

Mountain Neurological Research Center

Basalt, Colorado, United States

Site Status

Colorado Neurological Research Center, PC

Denver, Colorado, United States

Site Status

Ki Health Partners, LLC

Stamford, Connecticut, United States

Site Status

JEM Research Institute

Atlantis, Florida, United States

Site Status

Neurology Offices of South Florida

Boca Raton, Florida, United States

Site Status

K2 Medical Research- Ocoee

Clermont, Florida, United States

Site Status

Arrow Clinical Trials

Daytona Beach, Florida, United States

Site Status

Brain Matters Research

Delray Beach, Florida, United States

Site Status

Neuropsychiatric Research Center of Southwest Florida

Fort Myers, Florida, United States

Site Status

Velocity Clinical Research, Hallandale Beach

Hallandale, Florida, United States

Site Status

Galiz Research

Hialeah, Florida, United States

Site Status

Infinity Clinical Research - Sunrise

Hollywood, Florida, United States

Site Status

Luminous Clinical Research

Homestead, Florida, United States

Site Status

CNS Healthcare - Jacksonville

Jacksonville, Florida, United States

Site Status

Charter Research

Lady Lake, Florida, United States

Site Status

ClinCloud

Maitland, Florida, United States

Site Status

Merritt Island Medical Research, LLC

Merritt Island, Florida, United States

Site Status

Central Miami Medical Institute (GMI)

Miami, Florida, United States

Site Status

Mind Institute at Miami Jewish Health

Miami, Florida, United States

Site Status

New Horizon Research Center

Miami, Florida, United States

Site Status

Brainstorm Research

Miami, Florida, United States

Site Status

South Florida Research Phase I-IV INC

Miami Springs, Florida, United States

Site Status

Suncoast Clinical Research, Inc.

New Port Richey, Florida, United States

Site Status

Renstar Medical Research

Ocala, Florida, United States

Site Status

Health Synergy Clinical Research

Okeechobee, Florida, United States

Site Status

Clinical Neuroscience Solutions, Inc. dba CNS Healthcare

Orlando, Florida, United States

Site Status

Medical Research

Port Orange, Florida, United States

Site Status

Intercoastal Medical Group - Sarasota

Sarasota, Florida, United States

Site Status

Alzheimer's Research & Treatment Center

Stuart, Florida, United States

Site Status

Clinical Research of Brandon, LLC (Tampa)

Tampa, Florida, United States

Site Status

Stedman Clinical Trials

Tampa, Florida, United States

Site Status

Alzheimer's Research & Treatment Center

Wellington, Florida, United States

Site Status

Premier Research Institute at Palm Beach Neurology

West Palm Beach, Florida, United States

Site Status

Charter Research

Winter Park, Florida, United States

Site Status

Columbus Memory Center, PC

Columbus, Georgia, United States

Site Status

Accel Research Sites - NeuroStudies

Decatur, Georgia, United States

Site Status

Velocity Clinical Research - Boise

Meridian, Idaho, United States

Site Status

Advocate Aurora Health - Advocate Memory Center

Park Ridge, Illinois, United States

Site Status

Ascension Via Christi Research

Wichita, Kansas, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

Neuro Medical Clinic of Central Louisiana, LLC

Alexandria, Louisiana, United States

Site Status

Neurology Center of New England

Foxborough, Massachusetts, United States

Site Status

ActivMed Practices & Research, LLC

Methuen, Massachusetts, United States

Site Status

Boston Center for Memory

Newton, Massachusetts, United States

Site Status

Boston Neuro Research Center

North Dartmouth, Massachusetts, United States

Site Status

Headlands Eastern MA LLC

Plymouth, Massachusetts, United States

Site Status

MedVadis Research

Waltham, Massachusetts, United States

Site Status

Clinical Research Professionals

Chesterfield, Missouri, United States

Site Status

CCT Research - Papillion Research Center

Papillion, Nebraska, United States

Site Status

Patient First MD

Middletown, New Jersey, United States

Site Status

Advanced Clinical Institute, Inc

Neptune City, New Jersey, United States

Site Status

Global Medical Institutes, LLC

Princeton, New Jersey, United States

Site Status

The Cognitive and Research Center of New Jersey (CRCNJ)

Springfield, New Jersey, United States

Site Status

Advanced Memory Research Institute of NJ

Toms River, New Jersey, United States

Site Status

Neurology Specialists of Monmouth County

West Long Branch, New Jersey, United States

Site Status

Albuquerque Neuroscience, Inc

Albuquerque, New Mexico, United States

Site Status

Albany Medical Center

Albany, New York, United States

Site Status

Neurological Associates of Albany

Albany, New York, United States

Site Status

Dent Neurologic Institute

Amherst, New York, United States

Site Status

Integrative Clinical Trials, LLC

Brooklyn, New York, United States

Site Status

SPRI Clinical Trials Brooklyn

Brooklyn, New York, United States

Site Status

Velocity Clinical Research, Formerly Clarity Clinical Research

East Syracuse, New York, United States

Site Status

Parker Jewish Institute for Health Care & Rehabilitation

New Hyde Park, New York, United States

Site Status

Mid Hudson Medical Research

New Windsor, New York, United States

Site Status

NY Neurology Associates

New York, New York, United States

Site Status

University of Rochester Medical Center - Alzheimer's Disease Care, Research and Education Program

Rochester, New York, United States

Site Status

Richmond Behavioral Associates

Staten Island, New York, United States

Site Status

Richmond Behavioral Associates

Staten Island, New York, United States

Site Status

Triad Clinical Trials, LLC

Greensboro, North Carolina, United States

Site Status

Alzheimer's Memory Center

Matthew, North Carolina, United States

Site Status

Accellacare Research of Winston-Salem

Winston-Salem, North Carolina, United States

Site Status

Insight Clinical Trials LLC

Beachwood, Ohio, United States

Site Status

NeuroScience Research Center, LLC

Canton, Ohio, United States

Site Status

Dayton Center for Neurological Disorders

Centerville, Ohio, United States

Site Status

the Ohio State University

Columbus, Ohio, United States

Site Status

Neurology Diagnostics

Dayton, Ohio, United States

Site Status

Neuro-Behavioral Clinical Research

North Canton, Ohio, United States

Site Status

Summit Research Network, LLC

Portland, Oregon, United States

Site Status

Center for Cognitive Health - Portland

Portland, Oregon, United States

Site Status

Keystone Clinical Studies, LLC

Plymouth Meeting, Pennsylvania, United States

Site Status

Rhode Island Mood & Memory Research Institute

East Providence, Rhode Island, United States

Site Status

Palmetto Clinical Research

Summerville, South Carolina, United States

Site Status

Senior Adults Specialty Research, Inc

Austin, Texas, United States

Site Status

Texas Neurology, PA

Dallas, Texas, United States

Site Status

Baylor Scott & White Research Institute

Dallas, Texas, United States

Site Status

Mt. Olympus Medical Research, LLC

Katy, Texas, United States

Site Status

Grayline Research Center

Wichita Falls, Texas, United States

Site Status

Re:Cognition Health

Fairfax, Virginia, United States

Site Status

Northwest Clinical Research Center

Bellevue, Washington, United States

Site Status

Memory and Brain Wellness Center at Harborview

Seattle, Washington, United States

Site Status

KaRa Institute of Neurological Diseases Pty. Ltd.

Macquarie Park, New South Wales, Australia

Site Status

The University of Queensland

Herston, Queensland, Australia

Site Status

Delmont Consulting Suites

Glen Iris, Victoria, Australia

Site Status

Austin Health

Heidelberg, Victoria, Australia

Site Status

The Alfred Hospital

Melbourne, Victoria, Australia

Site Status

Eastern Health

Melbourne, Victoria, Australia

Site Status

Alzheimer's Research Australia

Nedlands, Western Australia, Australia

Site Status

OCT Research ULC DBA Okanagan Clinical Trials

Kelowna, British Columbia, Canada

Site Status

Dr. George Dumont University Hospital Centre

Moncton, New Brunswick, Canada

Site Status

True North Clinical Research - Halifax/Nova Scotia

Halifax, Nova Scotia, Canada

Site Status

True North Clinical Research - New Minas

New Minas, Nova Scotia, Canada

Site Status

St. Joseph's Health Care London

London, Ontario, Canada

Site Status

Ottawa Memory Clinic

Ottawa, Ontario, Canada

Site Status

Kawartha Centre - Redefining Healthy Aging

Peterborough, Ontario, Canada

Site Status

Toronto Memory Program

Toronto, Ontario, Canada

Site Status

The Centre for Memory and Aging

Toronto, Ontario, Canada

Site Status

Q & T Research

Sherbrooke, Quebec, Canada

Site Status

Diex Research - Sherbrooke

Sherbrooke, Quebec, Canada

Site Status

Alpha Recherche Clinique

Québec, , Canada

Site Status

Barbara Diaz Hernandez Md Research, Inc.

San Juan, PR, Puerto Rico

Site Status

Santa Cruz Behavioral PSC

Bayamón, , Puerto Rico

Site Status

Inspira Clinical Research

San Juan, , Puerto Rico

Site Status

Kyungpook National University Chilgok Hospital

Daegu, Buk-gu, South Korea

Site Status

Chonnam National University Hospital

Gwangju, Dong-gu, South Korea

Site Status

Seoul National University Bundang Hospital

Seongnam-si, Gyeonggi-do, South Korea

Site Status

Gachon University Gil Hospital

Namyangju, Incheon, South Korea

Site Status

Inha Unviersity Medical Center

Incheon, Jung-gu, South Korea

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Australia Canada Puerto Rico South Korea

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

PTI-125-10

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.