RecruitingPHASE2INTERVENTIONAL

Treatment of Ectopic Calcification in Fahr's Disease or Syndrome

This research is investigating a condition called Fahr's disease or syndrome, where calcium deposits build up in certain parts of the brain. This can lead to various symptoms like memory problems, movement difficulties, and mood changes, usually starting in adulthood. Currently, there isn't a specific treatment to stop the disease from getting worse. However, there's a small hint that a type of drug used for bones, called alendronate (a bisphosphonate), might help. This new study aims to properly test if these bisphosphonate drugs can make a real difference for people with Fahr's disease, or if they have no effect compared to a dummy treatment (placebo).

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

UMC Utrecht

Enrolment target

Start

03 Apr 2023

Estimated completion

01 Dec 2027

Fahr Disease Fahr Syndrome Primary Familial Brain Calcification

What is this study about?

Imagine tiny specks of calcium building up in a very important part of your brain called the basal ganglia. This is what happens in a condition called Fahr's disease or syndrome. When doctors don't know why it's happening, they usually call it Fahr's disease. If they find another reason for the calcium build-up, they might call it Fahr's syndrome instead. These calcium deposits can affect how the brain works, leading to a variety of symptoms like problems with memory, thinking, movement, speech, or even mood changes. These symptoms usually appear when a person is between 30 and 50 years old and can slowly get worse over time, making everyday tasks more difficult and affecting their quality of life.

At the moment, there aren't any treatments that can stop Fahr's disease from progressing or reverse its effects. However, doctors have noticed in a few patients that a medicine usually used for bones, called alendronate, seemed to help. This medicine belongs to a group of drugs called bisphosphonates. This new study is being done to see if this type of medicine can actually be an effective treatment for a wider group of people with Fahr's disease or syndrome.

The main goal of this study is to properly test if bisphosphonates can improve the symptoms and quality of life for people with Fahr's disease. Participants will either receive the active drug (etidronate, another bisphosphonate) or a dummy treatment (placebo). By comparing the two groups, researchers hope to understand if bisphosphonates can play a role in managing this condition and potentially offer a new treatment option in the future.

Key takeaways

The study is for Fahr's disease or syndrome.
It's testing a drug called Etidronate against a placebo.
Symptoms of Fahr's can include memory, movement, and mood changes.
Currently, there are no specific treatments for Fahr's disease.
The study aims to see if bisphosphonate drugs can help manage the condition.

Who may be eligible?

This study is looking for adults aged 18 or older.

To be considered for the study, you must have a formal diagnosis of Fahr's disease or syndrome. This diagnosis is usually made based on the symptoms you experience and a special head scan (CT scan) that clearly shows calcium build-ups in both sides of a specific part of your brain called the basal ganglia. The amount of calcium seen on the scan must be higher than what would be expected for your age to help confirm it's Fahr's disease, not just age-related changes.

Additionally, having a family history of Fahr's disease (meaning close relatives have it) or having certain genetic changes linked to the condition can also support the diagnosis and your eligibility for this study.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with Fahr's disease or syndrome?
Do you have calcium build-ups in specific parts of your brain visible on a CT scan?
Are these calcium levels higher than what's expected for your age?
Do you have a family history of Fahr's disease or known genetic changes linked to it (this helps, but might not be required)?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

The information provided does not detail the specifics of what taking part will involve, such as the number of visits, specific assessments, or the total duration of the study. However, it mentions that participants will either receive the study drug (Etidronate) or a placebo (a dummy treatment). This usually means you would be taking medication regularly according to the study's instructions. There would likely be regular appointments to monitor your health, how you are responding to the treatment, and to check for any side effects. You would also have check-ups to assess your symptoms and overall well-being. Detailed information about what you would need to do will be provided if you are considered for the study.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as receiving a new investigational treatment that might improve your condition, or contributing to medical knowledge that could help others in the future. However, there are also potential risks. The study treatment (Etidronate) may have side effects, which would be explained to you. There's also a chance you could receive a placebo, meaning you wouldn't get the active drug. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

University Medical Center Utrecht
Utrecht, Netherlands· Recruiting
University College London Hospital
London, United Kingdom· Not yet recruiting

Common questions

What is Fahr's disease?

It's a condition where calcium deposits build up in parts of the brain, which can lead to various symptoms like memory or movement problems.

Why are you studying this particular drug?

Early observations suggest that medicines like Etidronate, used for bone conditions, might help with Fahr's disease, and we want to test this properly.

Will I definitely get the active drug?

No, participants will either receive the study drug (Etidronate) or a placebo (a dummy treatment), chosen randomly, so we can compare their effects.

Who is funding this research?

The information provided does not specify the funding source for this trial.

Where is this study taking place?

The provided summary doesn't state the specific locations where this trial is being conducted.

How to find out more

Huiberdina L Koek, MD PhD

h.l.koek@umcutrecht.nl 0031 88 75 55555 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.