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Treatment of Ectopic Calcification in Fahr's Disease or Syndrome

NCT05662111 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 98

Last updated 2026-03-02

No results posted yet for this study

Summary

Fahr's disease or syndrome are neurodegenerative diseases in which patients present with bilateral vessel associated calcifications in the basal ganglia. The clinical penetration of Fahr's disease or syndrome is incomplete and heterogeneous comprising of neuropsychiatric signs, cognitive decline, movement disorders, and various other signs (migraine, speech disorders, pain, seizures). The symptoms start between 30 and 50 years and are (slowly) progressive. Symptomatic patients have an increased risk for dependence in activities of daily living and impaired quality of life.

Currently, disease-modifying therapies are not available for patients with Fahr's disease or syndrome. However, in a small case series it was shown that alendronate was effective in the clinical treatment of several patients with Fahr's disease or syndrome. Now the time has come to investigate the effectiveness of treatment with bisphosphonates in patients with Fahr's disease or syndrome in a randomized controlled trial.

Conditions

  • Fahr Disease
  • Fahr Syndrome
  • Primary Familial Brain Calcification

Interventions

DRUG

Etidronate

The dosage of etidronate is 20 mg/kg for twee weeks and ten weeks off. Etidronate is given in capsules of 200 mg. Etidronate capsules are administered orally. During the study, participants will receive etidronate in four periods of two weeks during the twelve months of follow-up.

DRUG

Placebo

Placebo is given in capsules and are administered orally. During the study, participants will receive placebo in four periods of two weeks during the twelve months of follow-up.

Sponsors & Collaborators

  • Netherlands Brain Foundation

    collaborator OTHER

  • UMC Utrecht

    lead OTHER

Principal Investigators

  • Huiberdina L Koek, MD, PhD · UMC Utrecht

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-04-03
Primary Completion
2027-12-31
Completion
2027-12-31

Countries

  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05662111 on ClinicalTrials.gov