Ongoing, recruitingTherapeutic use (Phase IV)Interventional

Treating Leg Symptoms in Women with X-linked Adrenoleukodystrophy: A Key to Improving Sleep and Gait Performance

This study is for women living with X-linked adrenoleukodystrophy (X-ALD), a rare genetic condition. Many women with X-ALD experience uncomfortable leg symptoms, sometimes called 'restless legs,' which can make it hard to sleep and affect how they get around. The research team wants to find out if a common medicine called Pramipexole (often used for restless legs) can help ease these leg problems. They will be looking at how the medicine might improve sleep, walking ability, mood, anxiety levels, and overall quality of life for these women. This is a Phase IV study, meaning the medicine is already approved for other uses but researchers are now studying its effects in this specific group of patients, aiming to find better ways to manage their symptoms.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic use (Phase IV)

Sponsor

Amsterdam UMC Stichting

Enrolment target

Start

17 Dec 2024

X-linked adrenoleukodystrophy Restless legs

What is this study about?

This study is for women who have a rare genetic condition called X-linked adrenoleukodystrophy, or X-ALD. While X-ALD can affect many parts of the body, some women with the condition get uncomfortable feelings in their legs, often described as 'restless legs.' These feelings can make it hard to relax, fall asleep, and stay asleep. This lack of good sleep can then make them feel tired, affect their mood, and make daily activities, including walking, more difficult.

The research team wants to explore if a medicine called Pramipexole, which is already used to treat restless legs in general, could help improve these leg symptoms specifically for women with X-ALD. By easing the leg discomfort, the study hopes to see if women sleep better, walk more easily, and generally feel better in themselves day-to-day. It’s important to find ways to help with these issues as they can significantly impact someone's life.

This is a 'Phase IV' study. This means the medicine (Pramipexole) is already approved and available for use in other conditions. However, scientists are now studying if it can also help women with X-ALD. The goal is to gather more information about how this medicine works in this specific group of people, to potentially offer a new way to manage their symptoms and improve their overall quality of life.

Key takeaways

Targets leg symptoms in women with X-ALD.
Investigates if Pramipexole improves sleep and walking.
Also checks effects on mood, anxiety, and quality of life.
Open to women aged 18 and over with X-ALD.
Medicine (Pramipexole) is already approved for other conditions.

Who may be eligible?

This study is looking for women aged 18 years and older. There is no upper age limit, so women of all adult ages can be considered. The main requirement is that you have been diagnosed with X-linked adrenoleukodystrophy (X-ALD).

If you are a woman living with X-ALD and are experiencing leg symptoms that bother you, this study might be suitable for you. The research team will carefully check other details about your health to make sure the study is a good fit and safe for you to take part.

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 a woman?
Are you 18 years old or older?
Do you have a diagnosis of X-linked adrenoleukodystrophy (X-ALD)?
Do you experience leg symptoms (like restless legs) as part of your condition?

Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the study clinic over a period of time. During these visits, you would receive the study medicine, Pramipexole. The study team will help you understand how to take it.

You would have several assessments to check how the medicine is affecting your sleep (how long you sleep, how long it takes to fall asleep, and if you wake up during the night). They will also look at your mood and anxiety levels using simple questionnaires. Your overall well-being and daily life will be checked through quality of life surveys. To see how your movement is affected, you might be asked to do some simple walking tests, like timing how quickly you walk a certain distance or stand up and go. The full duration of your involvement will be clearly explained by the study team.

Potential risks and benefits

Potential benefits of taking part could include an improvement in your leg symptoms, leading to better sleep, improved walking, and a better mood. However, there's no guarantee that the medicine will work for you. As with any medicine, Pramipexole can have side effects, which the study team will explain in detail. You will be closely monitored for any reactions. You are completely 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 X-linked adrenoleukodystrophy (X-ALD)?

X-ALD is a rare genetic condition that mainly affects the brain and spinal cord, leading to various health problems. It is inherited through families.

What are 'restless legs'?

Restless legs is a feeling of uncomfortable sensations in the legs, often an irresistible urge to move them, especially at night or when resting. It can make sleeping difficult.

What is Pramipexole and how does it work?

Pramipexole is a medicine that helps to balance certain chemicals in the brain. It's commonly used to treat restless legs and Parkinson's disease. In this study, we're seeing if it can help women with X-ALD.

Will I receive the actual medicine or a dummy pill?

This study uses Pramipexole Viatris 0.088 mg tablets. The study is open-label, meaning everyone receives the active medicine. There is no dummy pill (placebo) involved in this particular trial.

How long will the study last for each individual?

The full length of your participation will be discussed by the research team if you are eligible and interested.