Active Brain & Nervous System NIHR-supported project

A randomized, placebo-controlled, double-blind trial to study the effects of Etidronate on ectopic CALCIfication in FAhr’s Disease or syndrome: CALCIFADE trial

Funder NIHR (part of DHSC)
Recipient organisation NIHR University College London Hospitals Biomedical Research Centre
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Dec 2026

In plain English

AI plain-English summary

A daily capsule of etidronate, a drug normally used for osteoporosis, is being tested to see if it can slow or reverse the abnormal calcium buildup in the brains of people with Fahr’s disease. Fahr’s disease and syndrome are rare, incurable conditions in which calcium deposits form in the small arteries deep inside the brain, steadily destroying movement, cognition, and mental health. No treatment currently exists to stop or shrink these deposits. Previous studies have suggested that bisphosphonates—drugs that regulate calcium in bone—might also work on these brain calcifications. The CALCIFADE trial is the first rigorous, placebo-controlled test of this idea in adults. If etidronate proves effective, it would become the first targeted therapy for Fahr’s disease, potentially preserving patients’ independence, mobility, and cognitive function for years longer than current supportive care allows. The trial measures not only brain calcification volume on scans but also real-world outcomes: how well patients walk, think, manage daily tasks, and rate their own quality of life. A positive result would transform a condition that currently has no medical recourse into one that can be actively managed.

View original technical description
Fahr's disease and syndrome are rare disorders leading to calcification of the small arteries in deep regions of the brain (basal ganglia), resulting in a wide range of symptoms, including cognitive decline, movement disorders and neuropsychiatric symptoms. No cure is available. Studies have shown the potential of treatment of these abnormal vascular calcifications with bisphosphonates, a group of medications used to treat osteoporosis and similar diseases. The CALCIFADE trial is a randomised, placebo-controlled, double-blind trial which evaluates the effects of etidronate 20 mg/kg during 12 months follow-up in patients aged ? 18 years with Fahr's disease or syndrome. Etidronate and placebo are administered in capsules daily for two weeks on followed by ten weeks off. The CALCIFADE trial has started in April 2023 and is currently being conducted at the outpatient clinic of the University Medical Center Utrecht, The Netherlands. The University College London Hospital, United Kingdom, will be added as a second study site. The primary endpoint is the change in cognitive functioning after 12 months of treatment. Secondary endpoints are the change in mobility, neuropsychiatric symptoms, volume of brain calcifications, dependence in activities of daily living, and quality of life.

Researchers

Amit Batla (Principal Investigator)

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Original classification

Translational Neuroscience

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