NCT05575076

Open-label Extension for Phase 3 Clinical Trials of Simufilam

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1,081 (actual)
• Sponsor: Cassava Sciences, Inc. · Industry
• Sex: All
• Age: 51 Years – 89 Years
• Healthy volunteers: Not accepted

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.

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

Interventions

Type	Name	Description
DRUG	Simufilam	simufilam 100 mg oral tablet, twice daily

Timeline

• Start date: 2022-11-07
• Primary completion: 2025-01-31
• Completion: 2025-01-31
• First posted: 2022-10-12
• Last updated: 2025-05-23
• Results posted: 2025-05-14

Locations

134 sites across 5 countries: United States, Australia, Canada, Puerto Rico, South Korea

Regulatory

• FDA-regulated drug study