RecruitingRare disease

Study on the Effects of Etidronate on Brain Calcification in Patients with Fahr's Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Etidronate Disodium on a rare condition known as Fahr's Disease or Fahr's Syndrome. Fahr's Disease is characterized by abnormal calcium deposits in certain areas of the brain, which can lead to problems with movement, thinking, and behavior. The trial aims to determine if Etidronate Disodium can help slow down or reduce the worsening of cognitive functions, which are related to thinking and understanding, in patients with this condition.

Participants in the study will receive either the medication Etidronate Disodium or a placebo, which is a substance with no active medication. The study is designed to be "double-blind," meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This approach helps ensure that the results are not influenced by expectations. The trial will last for a period of 12 months, during which changes in cognitive functioning, mobility, psychiatric symptoms, daily functioning, quality of life, and brain calcification will be monitored and compared between the two groups.

The primary goal of the study is to see if Etidronate Disodium can effectively halt or lessen the decline in cognitive abilities in those with Fahr's Disease. Additionally, the study will observe any changes in other aspects of the participants' health and well-being, such as their ability to move, their mental health, and their overall quality of life. By the end of the study, researchers hope to gather valuable information that could lead to better treatment options for individuals affected by this rare condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on specific criteria, including age and clinical diagnosis of Fahr's disease or syndrome.
A CT scan of the head is performed to assess calcifications in the brain, which are characteristic of Fahr's disease.
Step 2
Randomization and medication
Participants are randomly assigned to receive either etidronate disodium oral capsules 400 mg or a placebo. This process is double-blind, meaning neither the participant nor the researchers know which treatment is being administered.
The medication is taken orally in capsule form.
Step 3
Treatment period
The treatment period lasts for 12 months. During this time, the participant continues to take the assigned medication as directed.
Regular assessments are conducted to monitor changes in cognitive functioning, mobility, psychiatric symptoms, daily functioning, quality of life, and brain calcification.
Step 4
End of study assessments
At the end of the 12-month period, final assessments are conducted to evaluate the effects of the treatment.
The primary focus is on any changes in cognitive functioning, with secondary assessments on mobility, psychiatric symptoms, daily functioning, quality of life, and brain calcification.

Who can join the trial?

5 criteria

Must be **18 years or older**.
Must have a **clinical diagnosis** of Fahr's disease or syndrome. This means the condition is identified based on symptoms and specific findings on a CT scan of the head.
Must show **bilateral calcifications** of the basal ganglia on a CT scan. This means there are calcium deposits on both sides of a specific area in the brain.
A **family history** that suggests the condition is inherited in an autosomal dominant way can support the diagnosis. This means the condition may be passed down from one generation to the next.
The presence of a **pathogenic mutation** in one of the genes related to PFBC (Primary Familial Brain Calcification) can support the diagnosis. This means there is a change in the DNA that is known to cause the condition.

Who cannot join the trial?

4 criteria

Patients who do not have a diagnosis of Fahr's syndrome or Fahr's disease cannot participate. These are rare conditions that affect the brain.
Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
Patients who are part of a vulnerable population, such as those who cannot give consent or are in a dependent situation, are not eligible.
Patients who do not meet the specific health criteria set by the study cannot participate. These criteria ensure the safety and relevance of the study for participants.

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Universitair Medisch Centrum Utrecht
Utrecht, The Netherlands

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Investigated drugs

Etidronate is a medication being studied to see if it can stop or slow down the worsening of cognitive functions in patients with Fahr's disease or syndrome. This condition involves abnormal calcium deposits in the brain, and the trial aims to find out if etidronate can help manage these deposits and improve or maintain brain function.

Etidronate Disodium

What is already known about the treatment

Etidronate – Etidronate is administered orally in tablet form. It is currently being studied in clinical trials for its potential to treat Fahr's disease or syndrome, a rare condition characterized by abnormal calcium deposits in the brain. The main therapeutic indication for Etidronate in this context is to prevent or slow down cognitive decline associated with these calcium deposits. At the molecular level, Etidronate works by inhibiting the activity of cells that break down bone tissue, which may help reduce abnormal calcification. It is classified as a bisphosphonate, a type of medication commonly used to treat bone disorders.

Investigated diseases

Fahr's Syndrome
Fahr's syndrome is a rare neurological disorder characterized by abnormal calcium deposits in areas of the brain that control movement. These deposits can lead to a variety of symptoms, including movement disorders, cognitive decline, and psychiatric issues. The condition often begins in adulthood, but symptoms can vary widely among individuals. As the disease progresses, patients may experience difficulties with coordination, speech, and balance. The exact cause of Fahr's syndrome is not well understood, but it is believed to have a genetic component. The progression of symptoms can be gradual, affecting daily functioning and quality of life over time.
Fahr's Disease
Fahr's disease is a genetic disorder that results in abnormal calcium buildup in the brain, particularly in the basal ganglia and other areas responsible for motor control. This condition can lead to a range of neurological symptoms, such as tremors, muscle stiffness, and involuntary movements. Cognitive impairment and psychiatric symptoms, such as mood swings or depression, may also occur. The onset of symptoms typically occurs in adulthood, but the severity and progression can vary. Over time, individuals may experience increasing difficulty with movement and coordination. The underlying genetic mutations associated with Fahr's disease are still being studied to better understand its development.