The effects of etidronate on brain calcifications in Fahr's disease or syndrome: rationale and design of the...

Area of Science:

• Neurology
• Pharmacology
• Radiology

Background:

• Fahr's disease and syndrome involve basal ganglia calcification, leading to cognitive, motor, and psychiatric issues.
• Currently, no therapies exist to modify the progression of Fahr's disease and syndrome.
• Bisphosphonates show potential for treating ectopic vascular calcifications.

Purpose of the Study:

• To evaluate the efficacy of etidronate in treating Fahr's disease and syndrome.
• To assess the impact of etidronate on cognitive function, mobility, and neuropsychiatric symptoms.

Main Methods:

• The CALCIFADE trial is a randomized, double-blind, placebo-controlled study.
• Participants (aged ≥18) receive either etidronate or placebo for 12 months.
• Primary endpoint: change in cognitive functioning; secondary endpoints include mobility, neuropsychiatric symptoms, brain calcification volume, daily living activities, and quality of life.

Main Results:

• Patient recruitment commenced in April 2023.
• Study results are anticipated in 2026.
• Findings will be published in peer-reviewed journals and presented at conferences.

Conclusions:

• Fahr's disease and syndrome are progressive, impacting health outcomes.
• Etidronate presents a potential new therapeutic option for patients diagnosed with Fahr's disease or syndrome.