Alzheimer DiseasePhase 3NCT05575076
Open-label Extension for Phase 3 Clinical Trials of Simufilam
A Phase 3 study testing Simufilam in people with Alzheimer Disease, run by Cassava Sciences, Inc. at 134 sites in 5 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 51 to 89 years
- Conditions the study is for
- Alzheimer Disease
- What is being tested?
- Simufilam
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1,081 participants
- Where
- 134 sitesin 5 countries
- Study dates
- 7 Nov 2022to 31 Jan 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 51 to 89 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
134 sites in 5 countries.
134 sites in 5 countries
United States107 sites
Albany Medical Center
Albany, New York, 12208
Neurological Associates of Albany
Albany, New York, 12208
Albuquerque Neuroscience, Inc
Albuquerque, New Mexico, 87109
Neuro Medical Clinic of Central Louisiana, LLC
Alexandria, Louisiana, 71301
Dent Neurologic Institute
Amherst, New York, 14226
Advanced Research Center, Inc.
Anaheim, California, 92805
JEM Research Institute
Atlantis, Florida, 33462
Senior Adults Specialty Research, Inc
Austin, Texas, 78757
Mountain Neurological Research Center
Basalt, Colorado, 81621
Insight Clinical Trials LLC
Beachwood, Ohio, 44122
Northwest Clinical Research Center
Bellevue, Washington, 98007
Neurology Offices of South Florida
Boca Raton, Florida, 33428
Integrative Clinical Trials, LLC
Brooklyn, New York, 11229
SPRI Clinical Trials Brooklyn
Brooklyn, New York, 11235
NeuroScience Research Center, LLC
Canton, Ohio, 44718
North County Neurology Associates
Carlsbad, California, 92011
Dayton Center for Neurological Disorders
Centerville, Ohio, 45459
MDFirst Research
Chandler, Arizona, 85286
Clinical Research Professionals
Chesterfield, Missouri, 63005
K2 Medical Research- Ocoee
Clermont, Florida, 34711
Axiom Research, LLC
Colton, California, 92324
Columbus Memory Center, PC
Columbus, Georgia, 31909
the Ohio State University
Columbus, Ohio, 43221
ATP Clinical Research, Inc.
Costa Mesa, California, 92626
Texas Neurology, PA
Dallas, Texas, 75206
Baylor Scott & White Research Institute
Dallas, Texas, 75231
Neurology Diagnostics
Dayton, Ohio, 45459
Arrow Clinical Trials
Daytona Beach, Florida, 32117
Accel Research Sites - NeuroStudies
Decatur, Georgia, 30030
Brain Matters Research
Delray Beach, Florida, 33445
Colorado Neurological Research Center, PC
Denver, Colorado, 80210
Rhode Island Mood & Memory Research Institute
East Providence, Rhode Island, 02914
Velocity Clinical Research, Formerly Clarity Clinical Research
East Syracuse, New York, 13057
Re:Cognition Health
Fairfax, Virginia, 22031
Neuropsychiatric Research Center of Southwest Florida
Fort Myers, Florida, 33912
Neurology Center of New England
Foxborough, Massachusetts, 02035
Neuro-Pain Medical Center
Fresno, California, 93710
Neurology Center of North Orange County
Fullerton, California, 92835
CCT Research - Gilbert Neurology Partners
Gilbert, Arizona, 85297
Triad Clinical Trials, LLC
Greensboro, North Carolina, 27410
Velocity Clinical Research, Hallandale Beach
Hallandale, Florida, 33009
Galiz Research
Hialeah, Florida, 33016
Infinity Clinical Research - Sunrise
Hollywood, Florida, 33024
Luminous Clinical Research
Homestead, Florida, 33030
Sun Valley Research Center, Inc.
Imperial, California, 92251
CNS Healthcare - Jacksonville
Jacksonville, Florida, 32256
Mt. Olympus Medical Research, LLC
Katy, Texas, 77450
Charter Research
Lady Lake, Florida, 32792
Senior Clinical Trials
Laguna Hills, California, 92653
University of Kentucky
Lexington, Kentucky, 40504
ClinCloud
Maitland, Florida, 32751
Alzheimer's Memory Center
Matthew, North Carolina, 28105
Velocity Clinical Research - Boise
Meridian, Idaho, 83642
Merritt Island Medical Research, LLC
Merritt Island, Florida, 32952
ActivMed Practices & Research, LLC
Methuen, Massachusetts, 01844
Central Miami Medical Institute (GMI)
Miami, Florida, 33125
Mind Institute at Miami Jewish Health
Miami, Florida, 33137
New Horizon Research Center
Miami, Florida, 33165
Brainstorm Research
Miami, Florida, 33176
South Florida Research Phase I-IV INC
Miami Springs, Florida, 33166
Patient First MD
Middletown, New Jersey, 07748
Advanced Clinical Institute, Inc
Neptune City, New Jersey, 07753
Parker Jewish Institute for Health Care & Rehabilitation
New Hyde Park, New York, 11040-1433
Suncoast Clinical Research, Inc.
New Port Richey, Florida, 34652
Mid Hudson Medical Research
New Windsor, New York, 12553
NY Neurology Associates
New York, New York, 10003
Shankle Clinic and Hoag Memorial Hospital Presbyterian
Newport Beach, California, 92663
Boston Center for Memory
Newton, Massachusetts, 02459
Neuro-Behavioral Clinical Research
North Canton, Ohio, 44720
Boston Neuro Research Center
North Dartmouth, Massachusetts, 02747
Renstar Medical Research
Ocala, Florida, 34470
Health Synergy Clinical Research
Okeechobee, Florida, 34972
Clinical Neuroscience Solutions, Inc. dba CNS Healthcare
Orlando, Florida, 32801
CCT Research - Papillion Research Center
Papillion, Nebraska, 68046
Advocate Aurora Health - Advocate Memory Center
Park Ridge, Illinois, 60068
Xenoscience, Inc.
Phoenix, Arizona, 85004
Banner Alzheimer's Institute
Phoenix, Arizona, 85006
Headlands Eastern MA LLC
Plymouth, Massachusetts, 02360
Keystone Clinical Studies, LLC
Plymouth Meeting, Pennsylvania, 19462
Medical Research
Port Orange, Florida, 32127
Summit Research Network, LLC
Portland, Oregon, 97210
Center for Cognitive Health - Portland
Portland, Oregon, 97225
Global Medical Institutes, LLC
Princeton, New Jersey, 08540
Artemis Institute for Clinical Research
Riverside, California, 92503
University of Rochester Medical Center - Alzheimer's Disease Care, Research and Education Program
Rochester, New York, 14620
Pacific Research Network, LLC
San Diego, California, 92103
Syrentis Clinical Research
Santa Ana, California, 92705
Intercoastal Medical Group - Sarasota
Sarasota, Florida, 34239
Memory and Brain Wellness Center at Harborview
Seattle, Washington, 98104
The Cognitive and Research Center of New Jersey (CRCNJ)
Springfield, New Jersey, 07081
Ki Health Partners, LLC
Stamford, Connecticut, 06905
Richmond Behavioral Associates
Staten Island, New York, 10312
Richmond Behavioral Associates
Staten Island, New York, 10314
Alzheimer's Research & Treatment Center
Stuart, Florida, 34997
Palmetto Clinical Research
Summerville, South Carolina, 29485
Banner Sun Health Research Institute
Sun City, Arizona, 85351
Clinical Research of Brandon, LLC (Tampa)
Tampa, Florida, 33603
Stedman Clinical Trials
Tampa, Florida, 33613
Advanced Memory Research Institute of NJ
Toms River, New Jersey, 08755
MedVadis Research
Waltham, Massachusetts, 02451
Alzheimer's Research & Treatment Center
Wellington, Florida, 33414
Neurology Specialists of Monmouth County
West Long Branch, New Jersey, 07764
Premier Research Institute at Palm Beach Neurology
West Palm Beach, Florida, 33407
Ascension Via Christi Research
Wichita, Kansas, 67214
Grayline Research Center
Wichita Falls, Texas, 76309
Accellacare Research of Winston-Salem
Winston-Salem, North Carolina, 27103
Charter Research
Winter Park, Florida, 32792
Canada12 sites
True North Clinical Research - Halifax/Nova Scotia
Halifax, Nova Scotia, B3S 1N2
OCT Research ULC DBA Okanagan Clinical Trials
Kelowna, British Columbia, V1Y 1Z9
St. Joseph's Health Care London
London, Ontario, N6C 5J1
Dr. George Dumont University Hospital Centre
Moncton, New Brunswick, E1C 2Z3
True North Clinical Research - New Minas
New Minas, Nova Scotia, B4N 3R7
Ottawa Memory Clinic
Ottawa, Ontario, K1Z 1G3
Kawartha Centre - Redefining Healthy Aging
Peterborough, Ontario, K9H2P4
Alpha Recherche Clinique
Québec, G3K 2P8
Q & T Research
Sherbrooke, Quebec, J1J 2G2
Diex Research - Sherbrooke
Sherbrooke, Quebec, J1L 0H8
Toronto Memory Program
Toronto, Ontario, M3B 2S7
The Centre for Memory and Aging
Toronto, Ontario, M4G 3E8
Australia7 sites
Delmont Consulting Suites
Glen Iris, Victoria, 3146
Austin Health
Heidelberg, Victoria, 3084
The University of Queensland
Herston, Queensland, 4029
KaRa Institute of Neurological Diseases Pty. Ltd.
Macquarie Park, New South Wales, 2113
The Alfred Hospital
Melbourne, Victoria, 3004
Eastern Health
Melbourne, Victoria, 3128
Alzheimer's Research Australia
Nedlands, Western Australia, 6009
South Korea5 sites
Kyungpook National University Chilgok Hospital
Daegu, Buk-gu, 41404
Chonnam National University Hospital
Gwangju, Dong-gu, 61469
Inha Unviersity Medical Center
Incheon, Jung-gu, 22332
Gachon University Gil Hospital
Namyangju, Incheon, 21565
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, 13620
Puerto Rico3 sites
Santa Cruz Behavioral PSC
Bayamón, 00961
Barbara Diaz Hernandez Md Research, Inc.
San Juan, PR, 00926
Inspira Clinical Research
San Juan, 00918
About this study
Official title: 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.
Read the 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).
- Conditions
- Alzheimer Disease
- Being tested
- Simufilam
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SimufilamDrug
Also called PTI-125
simufilam 100 mg oral tablet, twice daily
| Study group | Role | What they receive |
|---|---|---|
| Simufilam 100 mg | Experimental | simufilam 100 mg oral tablet, twice daily Receives: Drug: Simufilam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events
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.
Measured: Baseline to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2022
- Study start7 Nov 2022
- Primary completion31 Jan 2025
- Study completion31 Jan 2025
Why it stopped: sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Alzheimer DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label extension
- Masking
- None (open label)
- Enrolment
- 1,081 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cassava Sciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- James Kupiec, MD, Study director, Cassava Sciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05575076
- Sponsor's protocol ID
- PTI-125-10
- Status last verified
- Feb 2025
- First submitted
- 7 Oct 2022
- Last updated
- 23 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05575076
- Acronym / sponsor ID
- PTI-125-10
- Conditions
- Alzheimer Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: sponsor decision
- Enrolment
- 1,081 participants (actual)
- Lead sponsor
- Cassava Sciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Nov 2022 (actual)
- Primary completion
- 31 Jan 2025 (actual)
- Study completion
- 31 Jan 2025 (actual)
- First posted
- 12 Oct 2022 (actual)
- Results
- Posted 14 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label extension
| Arm | Type | Receives |
|---|---|---|
| Simufilam 100 mg | Experimental | Drug: Simufilamsimufilam 100 mg oral tablet, twice daily |
Who may qualify
- Ages
- 51 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SimufilamAlso: PTI-125simufilam 100 mg oral tablet, twice daily | Drug | Simufilam 100 mg |
Primary outcome
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Kupiec, MD study director
Cassava Sciences, Inc.
134 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Simufilam in people with Alzheimer Disease, run by Cassava Sciences, Inc. at 134 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 134 sites in United States, Canada, Australia, South Korea, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (time frame: Baseline to 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2025.