Simufilam (PTI-125), 100 mg, for Mild-to-moderate Alzheimer's Disease Patients

Completed · Phase 2 · Has a placebo group

Conditions studied: Alzheimer Disease

In brief

A two-year safety study of simufilam (PTI-125) 100 mg oral tablets twice daily for participants of the previous simufilam studies as wells as additional new mild-to-moderate Alzheimer's disease subjects for a total of 200 participants. All participants will receive simufilam 100 mg tablets twice daily for one year, followed by a 6-month randomized, double-blind period where subjects will either continue on active treatment or be switched to placebo. The study concludes with an additional 6-month open-label treatment period. Clinic visits are every month or month and a half in the first year, and every 3 months in the second year with an additional visit at Month 13. Cognition and neuropsychiatric symptoms are evaluated.

Key facts

Study ID

NCT04388254

Run by

Cassava Sciences, Inc.

People needed

220

Starts

2020-03-24

Expected to finish

2023-11-09

Last updated by the study team

2025-04-22

Who can join

Age: 50 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Informed consent form (ICF) signed by the subject or legally acceptable representative.
• Patient has a caregiver or legal representative responsible for administering the drug and recording the time.
• Ages ≥ 50 and ≤ 85 years
• Clinical diagnosis of dementia due to possible or probable AD consistent with criteria established by a workgroup of the National Institute on Aging and the Alzheimer's Disease Association.
• If female, postmenopausal for at least 1 year
• Patient living at home, senior residential setting, or an institutional setting without the need for continuous (i.e. 24-h) nursing care
• General health status acceptable for participation in the study
• Fluency (oral and written) in English or Spanish
• If receiving memantine, rivastigmine, galantamine or an AChEI, receiving a stable dose for at least 3 months (90 days) before screening. If receiving donepezil, receiving any dose lower than 23 mg once daily. Multiple medications are allowed.
• The patient is a non-smoker for at least 3 years.
• The patient or legal representative must agree to comply with the drawing of blood samples for the PK assessments, laboratory assessments and SavaDx.
• MMSE-2 score ≥ 16 and ≤ 26 at screening, OR if > 26, must have evidence of AD pathology such as a prior CSF total tau/Aβ42 ratio ≥ 0.28, an amyloid positive PET scan or hippocampal volume loss consistent with AD.

You may not qualify if…

• Anything that in the opinion of the Investigator would preclude participation in a 2-year study.
• BMI < 18.5
• Positive urine drug screen.
• Positive HIV, HCV or HbsAg screen.
• Suicidality on C-SSRS
• Exposure to an experimental drug other than simufilam, experimental biologic or experimental medical device within 3 months before screening
• A medical condition that would interfere with a lumbar puncture
• Residence in a skilled nursing facility and requiring 24 h care.
• Clinically significant laboratory test results
• Clinically significant untreated hypothyroidism (if treated, thyroid-stimulating hormone level and thyroid supplementation dose must be stable for at least 6 months before screening)
• Insufficiently controlled diabetes mellitus, including requiring insulin or metformin >1000 mg/day.
• Renal insufficiency (serum creatinine > ULN and clinically significant in the opinion of PI and/or Sponsor OR eGFR <60 ml/min/m2 as estimated by either the MDRD or CKD-EPI equation)
• Malignant tumor within 3 years before screening (except squamous and basal cell carcinoma or cervical carcinoma in situ or localized prostate cancer or localized stage 1 bladder cancer)
• History of ischemic colitis or ischemic enterocolitis
• Unstable medical condition that is clinically significant in the judgment of the investigator
• Alanine transaminase (ALT) or aspartate transaminase (AST) > ULN or total bilirubin > ULN and clinically significant in the opinion of PI and/or Sponsor.
• History of myocardial infarction or unstable angina within 6 months before screening
• History of more than 1 myocardial infarction within 5 years before screening
• Clinically significant cardiac arrhythmia (including atrial fibrillation), cardiomyopathy, or cardiac conduction defect (patients with a pacemaker are acceptable)
• Symptomatic hypotension, or uncontrolled hypertension
• Clinically significant abnormality on screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QTc (Fridericia correction method) value ≥ 450 msec for males or ≥ 470 msec for females.
• Stroke within 18 months before screening, or history of a stroke concomitant with onset of dementia
• History of brain tumor or other clinically significant space-occupying lesion on CT or MRI
• Head trauma with clinically significant loss of consciousness within 12 months before screening or concurrent with the onset of dementia
• Onset of dementia secondary to cardiac arrest, surgery with general anesthesia, or resuscitation

Where it is running

• Cognitive Clinical Trials — Gilbert, Arizona, United States
• Cognitive Clinical Trials — Surprise, Arizona, United States
• Sun Valley Research Center, Inc. — Imperial, California, United States
• Brain Matters Research — Delray Beach, Florida, United States
• Neuropsychiatric Research Center of Southwest Florida — Fort Myers, Florida, United States
• Optimus U — Miami, Florida, United States
• Adaptive Clinical Research, Inc — Miami Lakes, Florida, United States
• IMIC, Inc. — Palmetto Bay, Florida, United States
• Cognitive Clinical Trials — Bellevue, Nebraska, United States
• Cognitive Clinical Trials — Omaha, Nebraska, United States
• Advanced Memory Research Institute — Toms River, New Jersey, United States
• Neuro-Behavioral Clinical Research — North Canton, Ohio, United States
• Senior Adults Specialty Research — Austin, Texas, United States
• Centex Studies, Inc. — Houston, Texas, United States
• Centex Studies — McAllen, Texas, United States
• Ottawa Memory Clinic — Ottawa, Ontario, Canada
• Toronto Memory Program — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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