AuthorisedTherapeutic exploratory (Phase II)Interventional

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

This research, called the CALCIFADE trial, is investigating a drug called etidronate for people who have Fahr's disease or Fahr's syndrome. In this study, some participants will receive etidronate capsules, while others will receive a dummy capsule (placebo). Neither the participants nor their doctors will know who is getting which. The main goal is to understand if etidronate can improve or maintain thinking and memory skills in people with Fahr's disease or syndrome over a 12-month period. Researchers will also look at how it affects movement, mood, daily activities, quality of life, and any changes in brain calcification. This is an early-stage study to explore the drug's potential effects.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Universitair Medisch Centrum Utrecht

Enrolment target

Start

03 Oct 2024

Fahr's disease Fahr's syndrome

What is this study about?

Fahr's disease and Fahr's syndrome are rare conditions where calcium deposits build up in certain parts of the brain. This can lead to a variety of symptoms, including problems with thinking, movement, and mood. Currently, there isn't a specific treatment to stop or reverse these calcium deposits.

This study, called CALCIFADE, is looking into whether a drug called etidronate can help people with Fahr's disease or syndrome. Etidronate is a type of medicine that affects how the body handles calcium, and it's already used for other conditions where calcium builds up in the wrong places. The researchers want to see if this medicine can make a difference for people with these brain calcium deposits.

The main aim of the study is to find out if etidronate can improve or at least stabilise thinking and memory in people with Fahr's disease or syndrome over a year. The study will also explore whether etidronate can help with other important aspects of daily life, such as how well people can move around, their mood, how they manage daily activities, and their overall quality of life. They will also check if the medicine has any effect on the calcium build-up in the brain itself.

Key takeaways

This study investigates etidronate for Fahr's disease/syndrome.
Aims to see if it improves thinking skills, movement, and mood.
Compares etidronate to a dummy pill (placebo).
Participation involves taking capsules for 12 months and clinic visits.
Open to adults aged 18 and over.
You can stop participating at any time.

Who may be eligible?

To take part in this study, you must be 18 years old or older. There is no upper age limit, meaning people of all adult ages can be considered. The study is open to both men and women.

More specific details about your health and medical history would be discussed with you by the study team. This is to make sure the study is a good fit for you and that taking part would be safe.

If you have Fahr's disease or Fahr's syndrome and are interested, it's always best to chat with your doctor or the study team to see if you meet all the requirements.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of Fahr's disease or Fahr's syndrome?
Are you able to take capsules by mouth?
Are you able to attend regular clinic visits and assessments for 12 months?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be asked to take capsules by mouth every day for 12 months. Some people will receive the active study medicine, Etidronate, while others will receive a dummy capsule (placebo) that looks exactly the same but contains no medicine. Neither you nor your doctor will know which one you are taking.

You would have several visits to the clinic over the 12 months, including at the beginning and at the end of the study. During these visits, the study team would assess your thinking skills, how you move, your mood, and how you manage everyday tasks. You might also be asked to complete questionnaires about your quality of life, and there will likely be scans to check for calcium in your brain. After the 12-month treatment period, there will be follow-up checks to see how you are doing.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as closely monitored care and access to a new treatment that isn't widely available yet, which *might* help improve your symptoms. However, there's no guarantee the treatment will work for you, and it's also possible you could receive the placebo. All medicines have potential side effects, and etidronate is no exception. The study team will carefully explain any known risks. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands

Common questions

What is Fahr's disease/syndrome?

It's a rare condition where calcium builds up in parts of the brain, which can affect thinking, movement, and mood.

What is etidronate?

It's a medicine that affects how the body handles calcium. This study is testing if it can help with calcium deposits in the brain.

What does 'placebo-controlled' mean?

It means some people get the active medicine, and others get a dummy pill, so researchers can compare the effects accurately.

How long will the study last for me?

You'll be taking capsules for 12 months, with visits and checks before, during, and after this period.

Can I stop participating if I want to?

Yes, you have the right to withdraw from the study at any time without giving a reason, and it won't affect your medical care.