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Alzheimer DiseasePhase 2NCT04079803

PTI-125 for Mild-to-moderate Alzheimer's Disease Patients

A Phase 2 study testing Simufilam 100 mg tablet and Simufilam 50 mg oral tablet in people with Alzheimer Disease, run by Cassava Sciences, Inc. at 9 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a Phase 2b, Randomized, Double-blind, Placebo-controlled, multiple dose study of PTI-125 in mild-to-moderate Alzheimer's disease patients. Read more about this study

Who is it for?
Ages 50 to 85 years
Conditions the study is for
Alzheimer Disease
What is being tested?
Simufilam 100 mg tablet and Simufilam 50 mg oral tablet
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
64 participants
Where
9 sitesUnited States
Study dates
9 Sep 2019to 31 Mar 2020
Phase
Phase 2
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 50 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Ages >= 50 and <= 85 years
  • Informed consent form (ICF) signed by the subject or legally acceptable representative.
  • Clinical diagnosis of dementia due to possible or probable Alzheimer's disease
  • Mini-Mental State Examination score >= 16 and <= 26 at screening
  • 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
Show all 13Show fewer
  • 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.
  • 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 and with a lumbar puncture and the drawing of cerebrospinal fluid samples.
  • The patient has a ratio of total tau/Aβ42 in cerebrospinal fluid >= 0.28.
  • Patient has a caregiver or legal representative responsible for administering the drug and recording the time.

Who cannot join

  • Exposure to an experimental drug, experimental biologic or experimental medical device within the longer of 5 half-lives or 3 months before screening
  • Enrollment in the previous PTI-125 trial
  • 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
Show all 41Show fewer
  • Insufficiently controlled diabetes mellitus
  • Renal insufficiency (serum creatinine > ULN)
  • 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.
  • 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 quaque die currently or within 3 months prior to randomization
  • Discontinued AChEI < 30 days prior to randomization
  • Antipsychotics; low doses are allowed only if the subject has received a stable dose for at least 3 months before randomization
  • Tricyclic antidepressants and monoamine oxidase inhibitors
  • Anxiolytics or sedative-hypnotics, including barbiturates (unless given in low doses for benign tremor); low doses of benzodiazepines and zolpidem are allowed
  • 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
  • Positive serum hepatitis B surface antigen (HBsAg) or positive hepatitis C virus HCV antibody test at screening
  • Positive HIV test at screening
  • Positive urine drug test at screening
  • Loss of a significant volume of blood (> 450 mL) within 4 weeks prior to the study
  • Suicidality on C-SSRS at screening

Trial locations and contacts

9 sites in 1 country.

9 sites in 1 country

United States9 sites

  • Cognitive Clinical Trials

    Bellevue, Nebraska, 68005

  • Cognitive Clinical Trials

    Gilbert, Arizona, 85296

  • Centex Studies, Inc.

    Houston, Texas, 77058

  • Centex Studies, Inc.

    McAllen, Texas, 78504

  • Optimus U

    Miami, Florida, 33125

  • Cognitive Clinical Trials

    Omaha, Nebraska, 68116

  • IMIC, Inc.

    Palmetto Bay, Florida, 33157

  • Cognitive Clinical Trials

    Surprise, Arizona, 85374

  • Advanced Memory Research Institute

    Toms River, New Jersey, 08755

Other trials in United States

About this study

Official title: A Phase 2b, Randomized, Double-blind, Placebo-controlled, Multiple Dose, Biomarker and Safety Study of PTI-125 in Mild-to-moderate Alzheimer's Disease Patients

This is a Phase 2b, Randomized, Double-blind, Placebo-controlled, multiple dose study of PTI-125 in mild-to-moderate Alzheimer's disease patients.

Read the detailed description

This is a Phase 2b, Randomized, Double-blind, Placebo-controlled, multiple dose study of PTI-125 in mild-to-moderate Alzheimer's disease patients. A total of sixty (60) patients will be enrolled in the study. Patients will receive Placebo, 50 mg or 100 mg b.i.d. of PTI-125. The objective of this study are to investigate the safety, and biomarkers of PTI-125 following 28-day repeat oral administration.

Being tested
Placebo oral tablet, Simufilam 100 mg tablet, Simufilam 50 mg oral tablet
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Placebo oral tabletDrug

    Oral placebo tablet

  • Simufilam 100 mg tabletDrug

    Also called PTI-125

    Simufilam 100 mg oral tablet

  • Simufilam 50 mg oral tabletDrug

    Also called PTI-125

    Simufilam 50 mg oral tablet

Study groups in NCT04079803
Study groupRoleWhat they receive
Placebo CohortPlacebo comparatorSubjects administered placebo oral tablets twice daily (BID)
Receives: Drug: Placebo oral tablet
Simufilam (PTI-125) 100 mg tablets CohortExperimentalSubjects administered simufilam (PTI-125) 100 mg oral tablets twice daily (BID)
Receives: Drug: Simufilam 50 mg oral tablet
Simufilam (PTI-125) 50 mg tablets CohortExperimentalSubjects administered simufilam (PTI-125) 50 mg oral tablets twice daily (BID)
Receives: Drug: Simufilam 100 mg tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in CSF Abeta42

    Change from Baseline (screening sample) to Day 28 in cerebrospinal fluid levels of Amyloid beta42

    Measured: Screening to Day 28

  2. Change From Baseline in CSF Total Tau.

    Change from Baseline (screening sample) to Day 28 in cerebrospinal fluid total tau.

    Measured: Screening to Day 28

  3. Change From Baseline in CSF P-tau181

    Change from Baseline (screening) to Day 28 in cerebrospinal fluid P-tau181

    Measured: Screening to Day 28

  4. Change From Baseline in CSF Neurogranin

    Change from Baseline (screening) to Day 28 in cerebrospinal fluid neurogranin

    Measured: Screening to Day 28

  5. Change From Baseline in CSF Neurofilament Light Chain

    Change from Baseline (screening) to Day 28 in cerebrospinal fluid neurofilament light chain

    Measured: Screening to Day 28

  6. Change From Baseline in CSF YKL-40

    Change from Baseline (screening) in cerebrospinal fluid YKL-40

    Measured: Screening to Day 28

Secondary outcomes (3)

  1. Paired Associates Learning Test

    Cognitive test assessing episodic memory. Boxes are displayed on the screen and are "opened" in a randomized order. One or more of them contains a pattern. The patterns are then displayed in the middle of the screen, one at a time and the participant must select the box in which the pattern was originally located. If the participant makes an error, the boxes are opened in sequence again to remind the participant of the locations of the patterns. The number of boxes increases progressively to a total of 8.

    Measured: Day 1 to Day 28

  2. Spatial Working Memory Test

    Cognitive assessment of spatial working memory: A number of colored squares (boxes) are shown on the screen. By selecting the boxes and using a process of elimination, the subject should find one yellow 'token' in each of a number of boxes and use them to fill up an empty column on the right-hand side of the screen. The number of boxes is gradually increased to a total of 8 for the subjects to search. The colors and positions of the boxes are changed from trial to trial to discourage stereotyped search strategies.

    Measured: Day 1 to Day 28

  3. CSF IL-6, sTREM2, HMGB1, Albumin, IgG

    Change from Baseline (screening sample) to Day 28 in secondary CSF biomarkers of neuroinflammation and blood-brain barrier integrity

    Measured: Screening to Day 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Sep 2019
  2. Study start9 Sep 2019
  3. Primary completion31 Mar 2020
  4. Study completion31 Mar 2020

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Approximately sixty (60) patients will be enrolled into the study and randomized to one of three cohorts. Cohorts will receive placebo or PTI-125 at 50 or 100 mg b.i.d. (n=20 per group)
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
64 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04079803
Sponsor's protocol ID
PTI-125-02
Other IDs
R44AG060878
Status last verified
Sep 2021
First submitted
26 Aug 2019
Last updated
29 Sep 2021
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Sever S. Role of actin cytoskeleton in podocytes. Pediatr Nephrol. 2021 Sep;36(9):2607-2614. doi: 10.1007/s00467-020-04812-z. Epub 2020 Nov 13.

    PubMed 33188449

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Simufilam 100 mg tablet and Simufilam 50 mg oral tablet in people with Alzheimer Disease, run by Cassava Sciences, Inc. at 9 sites in United States.

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 13 inclusion and 41 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: finished, as of the sponsor's last update on 29 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 1 Jun 2021). 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?

Change From Baseline in CSF Abeta42 (time frame: Screening to Day 28). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04079803, which also lists 1 linked publication.

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 29 Sep 2021.

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