Treatment of patients with Primary Familial Brain Calcification with etidronate
Research type
Research Study
Full title
A randomized, placebo-controlled, double-blind trial to study the effects of Etidronate on ectopic CALCIfication in FAhr’s Disease or syndrome: CALCIFADE trial
IRAS ID
1009530
Contact name
Birgitta Snijders
Contact email
Sponsor organisation
University Medical Center (UMC) Utrecht
Eudract number
2022-003299-17
ISRCTN Number
ISRCTN10492452
Clinicaltrials.gov Identifier
Research summary
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.
REC name
London - Central Research Ethics Committee
REC reference
24/LO/0870
Date of REC Opinion
5 Feb 2025
REC opinion
Further Information Favourable Opinion