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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04388254
Recruitment Status : Recruiting
First Posted : May 14, 2020
Last Update Posted : January 22, 2021
National Institute on Aging (NIA)
Information provided by (Responsible Party):
Cassava Sciences, Inc.

Brief Summary:
An Open-label study for patients who completed the previous studies, PTI-125-02 or PTI-125-03. Additional new patients will be included for a total of 100 patients enrolled for this study.

Condition or disease Intervention/treatment Phase
Alzheimer Disease Drug: Simufilam (PTI-125), 100 mg tablet Phase 2

Detailed Description:
The objectives of this study are to establish 1-year safety and to investigate the effect of Sumifilam (PTI-125) on biomarkers, cognition and neuropsychiatric symptoms during 12-month repeat-dose oral administration in mild-to-moderate AD patients, 50-85 years of age.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Approximately one hundred (100) patients will be enrolled into the study. The patients will receive Sumifilam (PTI-125), 100 mg b.i.d.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A 12-Month, Open-Label Safety Study of PTI-125 in Mild-to-moderate Alzheimer's Disease Patients
Actual Study Start Date : March 24, 2020
Estimated Primary Completion Date : March 31, 2022
Estimated Study Completion Date : April 30, 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Simafilam (PTI-125), 100 mg tablets
Simufilam (PTI-125), 100 mg oral tablets administered twice daily (BID)
Drug: Simufilam (PTI-125), 100 mg tablet
Sumifilam (PTI-125), 100 mg oral tablet
Other Names:
  • PTI-125
  • Simufilam

Primary Outcome Measures :
  1. Safety and Tolerability [ Time Frame: Day 1 to Month 12 ]
    Safety and tolerability of Sumifilam (PTI-125)

  2. Cerebrospinal fluid P-tau, neurofilament light chain, neurogranin, Total Tau, YKL-40, Abeta42 (pg/mL) [ Time Frame: Screening to Month 12 ]
    Biomarkers of AD pathology, neurodegeneration and neuroinflammation

  3. Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog-11) [ Time Frame: Day 1 to Month 12 ]
    Assess cognitive symptoms of dementia

  4. Neuropsychiatric Index (NPI) [ Time Frame: Day 1 to Month 12 ]
    Assess behavioral symptoms

Information from the National Library of Medicine

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Ages Eligible for Study:   50 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Informed consent form (ICF) signed by the subject or legally acceptable representative.
  • Patient has a caregiver or legal respresentative responsible for administering the drug and recording the time.
  • Ages >= 50 and <= 85 years (upper limit waived for prior PTI-125 study participants)
  • Clinical diagnosis of dementia due to possible or probable Alzheimer's disease
  • 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. If receiving donepezil, 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, laboratory assessments and for new patients or patients starting < 30 days form the last PTI-125 study, with a lumbar puncture and the drawing of cerebrospinal fluid samples for biomarker assessments.

Additional Criteria for NEW patients:

  • The patient has a ratio of total tau/Aβ42 in cerebrospinal fluid >= 0.28.
  • MMSE score >= 16 and <=26 at screening, OR if > 26, must have evidence of AD pathology such as a prior CSF total tau/AB42 ratio >=0.28, an amyloid positive PET scan or hippocampal volume loss consistent with AD.

Exclusion Criteria:

  • Anything in the opinion of the Investigator would preclude participation in a 1-year study.
  • Positive urine drug test at screening
  • Positive HIV, HCV or HbsAg screen
  • Suicidality on C-SSRS
  • Exposure to an experimental drug other than PTI-125, experimental biologic or experimental medical device within the longer of 5 half-lives or 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
  • Insufficiently controlled diabetes mellitus
  • Renal insufficiency (serum creatinine > ULN and clinically signigicant in the opinion of PI and/or Sponsor)
  • 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 corrected QT interval 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
  • Specific degenerative Central Nervous System disease diagnosis other than Alzheimer's disease (eg, Huntington's disease, Creutzfeld-Jacob disease, Down's syndrome, Frontotemporal Dementia, Parkinson's disease)
  • Wernicke's encephalopathy
  • Active acute or chronic Central Nervous System infection
  • Donepezil 23 mg or greater QD currently or within 3 months prior to randomization
  • Discontinued AChEI < 30 days prior to randomization
  • Antipsychotics; low doses are allowed only if given for sleep disturbances, agitation and/or aggression, and only if the subject has received a stable dose for at least 3 months before randomization
  • Tricyclic antidepressants and monoamine oxidase inhibitors; all other antidepressants are allowed only if the subject has received a stable dose for at least 3 months before randomization.
  • Anxiolytics or sedative-hypnotics, including barbiturates (unless given in low doses for benign tremor); low doses of benzodiazepines and zolpidem are allowed only if given for insomnia/sleep disturbance, and only if the subject has received a stable dose for at least 3 months before randomization.
  • Immunosuppressants, including systemic corticosteroids, if taken in clinically immunosuppressive doses (Steroid use for allergy or other inflammation is permitted.)
  • Antiepileptic medications if taken for control of seizures
  • Chronic intake of opioid-containing analgesics
  • Sedating H1 antihistamines
  • Nicotine therapy (all dosage forms including a patch), varenicline (Chantix), or similar therapeutic agent within 30 days before screening
  • Clinically significant illness within 30 days of enrollment
  • History of significant neurological, hepatic, renal, endocrine, cardiovascular, gastrointestinal, pulmonary, or metabolic disease
  • Loss of a significant volume of blood (> 450 mL) within 4 weeks prior to the study
  • COVID-19 infection

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04388254

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Contact: Lindsay Burns, PhD 512-501-2484
Contact: Antonio Hernandez, PsyD 512-582-2172

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Sponsors and Collaborators
Cassava Sciences, Inc.
National Institute on Aging (NIA)
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Study Chair: Lindsay Burns, PhD Cassava Sciences
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Responsible Party: Cassava Sciences, Inc. Identifier: NCT04388254    
Other Study ID Numbers: PTI-125-04
R44AG065152 ( U.S. NIH Grant/Contract )
First Posted: May 14, 2020    Key Record Dates
Last Update Posted: January 22, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alzheimer Disease
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders