Trial Outcomes & Findings for Open-Label Extension of the PTI-125-04 Study in Mild-to-Moderate Alzheimer's Disease (NCT NCT05352763)

Last Updated: 2025-08-15

Results Overview

Number of Participants with Adverse Events

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

90 participants

Primary outcome timeframe

Baseline to 96 Weeks

Results posted on

2025-08-15

Participant milestones
Measure	Simufilam 100 mg simufilam 100 mg oral tablet, twice daily simufilam: simufilam 100 mg oral tablets
Overall Study STARTED	90
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	69

Withdrawal data not reported

Open-Label Extension of the PTI-125-04 Study in Mild-to-Moderate Alzheimer's Disease

Baseline characteristics by cohort
Measure	Simufilam 100 mg n=90 Participants simufilam 100 mg oral tablet, twice daily simufilam: simufilam 100 mg oral tablets
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=5 Participants
Age, Categorical >=65 years	71 Participants n=5 Participants
Age, Continuous	73.2 years STANDARD_DEVIATION 8.78 • n=5 Participants
Sex: Female, Male Female	55 Participants n=5 Participants
Sex: Female, Male Male	35 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	83 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	87 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

Timeframe: Baseline to 96 Weeks

Population: Safety Analysis Population

Number of Participants with Adverse Events

Outcome measures
Measure	Simufilam 100 mg n=90 Participants simufilam 100 mg oral tablet, twice daily simufilam: simufilam 100 mg oral tablet, twice daily
Adverse Event Monitoring	90 Participants

Serious events: 15 serious events

Other events: 43 other events

Deaths: 0 deaths

Serious adverse events
Measure	Simufilam 100 mg n=90 participants at risk simufilam 100 mg oral tablets, b.i.d. simufilam: simufilam 100 mg oral tablets
Cardiac disorders Atrial fibrillation	1.1% 1/90 • Up to 2 years
Gastrointestinal disorders Abdominal hernia perforation	1.1% 1/90 • Up to 2 years
Gastrointestinal disorders Intestinal obstruction	1.1% 1/90 • Up to 2 years
Gastrointestinal disorders Pelvic mass	1.1% 1/90 • Up to 2 years
Infections and infestations Sepsis	1.1% 1/90 • Up to 2 years
Infections and infestations COVID-19	2.2% 2/90 • Up to 2 years
Infections and infestations Cellulitis	1.1% 1/90 • Up to 2 years
Infections and infestations Pyelonephritis	1.1% 1/90 • Up to 2 years
Infections and infestations Influenza	1.1% 1/90 • Up to 2 years
Infections and infestations Pneumonia	2.2% 2/90 • Up to 2 years
Injury, poisoning and procedural complications Rib fracture	1.1% 1/90 • Up to 2 years
Nervous system disorders Cerebrovascular accident	1.1% 1/90 • Up to 2 years
Nervous system disorders Dizziness	1.1% 1/90 • Up to 2 years
Nervous system disorders Syncope	2.2% 2/90 • Up to 2 years
Psychiatric disorders Agitation	1.1% 1/90 • Up to 2 years
Social circumstances Immobile	1.1% 1/90 • Up to 2 years
Vascular disorders Deep vein thrombosis	1.1% 1/90 • Up to 2 years

Other adverse events
Measure	Simufilam 100 mg n=90 participants at risk simufilam 100 mg oral tablets, b.i.d. simufilam: simufilam 100 mg oral tablets
Infections and infestations Urinary tract infection	16.7% 15/90 • Up to 2 years
Injury, poisoning and procedural complications Fall	15.6% 14/90 • Up to 2 years
Infections and infestations COVID-19	11.1% 10/90 • Up to 2 years
Psychiatric disorders Anxiety	8.9% 8/90 • Up to 2 years
Infections and infestations Upper respiratory tract infection	5.6% 5/90 • Up to 2 years

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