NCT05564169

Masitinib in Patients With Mild Alzheimer's Disease

A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 3 Clinical Trial to Evaluate the Safety and Efficacy of Masitinib as add-on Therapy in Patients With Mild Alzheimer's Disease, Treated With Standard of Care

• Status: Not Yet Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 600 (estimated)
• Sponsor: AB Science · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

Masitinib is an orally administered tyrosine kinase inhibitor that targets activated cells of the neuroimmune system (mast cells and microglia). Study AB21004 will evaluate masitinib as an adjunct to cholinesterase inhibitor and/or memantine in patients with mild-to-moderate Alzheimer's disease.

Detailed description

Masitinib is an oral tyrosine kinase inhibitor that has demonstrated neuroprotective action in neurodegenerative diseases via inhibition of mast cell and microglia/macrophage activity, and which is capable of accumulating within the central nervous system (CNS) at a therapeutically relevant concentration. There is a growing body of evidence implicating mast cells and microglia (types of innate immune cells that are present in the CNS), with the pathophysiology of Alzheimer's disease. Masitinib has been shown to restore normal spatial learning performance and promote recovery of synaptic markers in a mouse model of Alzheimer's disease, with its synapto-protective action being directly linked to mast cell inhibition. The potential benefit of masitinib in the treatment of patients with mild to moderate Alzheimer's disease has been previously demonstrated in a phase 2 study (AB04024; NCT00976118) and a positive phase 2B/3 study (AB09004; NCT01872598) that showed masitinib (4.5 mg/kg/day) was associated with a statistically significant slowing of cognitive deterioration. The objective of study AB21004 is to confirm treatment effect with masitinib as an adjunct to cholinesterase inhibitor and/or memantine in patients with mild-to-moderate Alzheimer's disease.

Conditions

• Alzheimer Disease

Interventions

Type	Name	Description
DRUG	Placebo	treatment per os
DRUG	Masitinib (4.5)	Masitinib (titration to 4.5 mg/kg/day)
DRUG	Standard of care	Cholinesterase inhibitors (donepezil, rivastigmine or galantamine) and/or memantine

Timeline

• Start date: 2026-06-01
• Primary completion: 2028-12-01
• Completion: 2029-12-01
• First posted: 2022-10-03
• Last updated: 2025-10-03

Locations

9 sites across 2 countries: France, Spain

Regulatory

• FDA-regulated drug study