NCT04388254

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

A 12-Month, Open-Label Safety Study of Simufilam Followed by a 6-Month Randomized Withdrawal and 6 Additional Months Open-Label in Mild-to-moderate Alzheimer's Disease Patients

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 220 (actual)
• Sponsor: Cassava Sciences, Inc. · Industry
• Sex: All
• Age: 50 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The objectives of this study are to build the safety database for simufilam (PTI-125) and to investigate its effects on biomarkers, cognition and neuropsychiatric symptoms during 12-month twice-daily administration in mild-to-moderate AD patients. Additional objectives are to assess differences in cognition and neuropsychiatric symptoms between active and placebo arms in the 6-month randomized period. All subjects will undergo lumbar puncture at screening for baseline testing of cerebrospinal fluid (CSF) total tau and Abeta42, and the first 50 subjects will also provide a CSF sample at Month 6 or Month 12 for evaluation of change from baseline in CSF biomarkers. CSF will not be required of subjects with prior CSF, PET or MRI evidence of Alzheimer's disease. Plasma biomarkers will be evaluated in all subjects. Safety will be assessed by blood tests, electrocardiograms, adverse event monitoring and, at Months 12 and 24, full physical examinations.

Conditions

• Alzheimer Disease

Interventions

Type	Name	Description
DRUG	Simufilam 100 mg oral tablet	Simufilam 100 mg oral tablet for b.i.d. administration
DRUG	Placebo	Matching placebo oral tablets

Timeline

• Start date: 2020-03-24
• Primary completion: 2023-11-09
• Completion: 2023-11-09
• First posted: 2020-05-14
• Last updated: 2025-04-22
• Results posted: 2025-04-22

Locations

17 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study