Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients with WHIM Syndrome with Open-Label Extension

This research study is for people with a rare condition called WHIM syndrome. It's looking into a new medicine, Mavorixafor, to see if it can help improve the body's ability to fight off infections. For the first part of the study, some participants will receive Mavorixafor, while others will get a placebo (a dummy pill, with no medicine in it). This helps researchers see if Mavorixafor is truly effective. They will measure if the medicine helps increase certain infection-fighting white blood cells and looks at changes in warts and infections. After this, everyone will have the option to receive Mavorixafor directly to further check its safety and how well it's tolerated over time. The main goal is to find out if this new medicine can make a positive difference for people with WHIM syndrome.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

X4 Pharmaceuticals Inc.

Enrolment target

Start

09 Nov 2024

WHIM Syndrome

What is this study about?

This study is about a condition called WHIM syndrome. People with WHIM syndrome often have problems with their immune system, which makes them more likely to get infections and develop warts. This study is testing a new medicine called Mavorixafor, hoping it can help improve how the body fights off these issues.

In the first part of the study, some people will get the active medicine, Mavorixafor, and others will get a placebo. A placebo is a dummy pill that looks just like the real medicine but contains no active ingredient. This is done so researchers can fairly compare if the new medicine really works better than no treatment at all. Neither the participants nor the doctors will know who is getting which tablet during this initial phase. Throughout this period, the study team will regularly check your blood to see if the medicine is increasing important infection-fighting cells called neutrophils, and they'll also keep track of any changes in infections or warts.

After this initial comparison period, all participants will have the chance to receive the active medicine, Mavorixafor, if they choose. This longer-term part of the study will help researchers gather more information on the medicine's safety, how well people tolerate it, and if the positive effects continue or improve over time. The overall aim is to understand if Mavorixafor could be a helpful new treatment option for people living with WHIM syndrome.

Key takeaways

This study is for adults with WHIM syndrome.
It's testing a new medicine called Mavorixafor.
The first part compares Mavorixafor to a dummy pill (placebo).
It aims to see if the medicine improves immunity and reduces infections/warts.
Participants will have regular health checks and blood tests.
Safety will be closely monitored throughout the study.

Who may be eligible?

To be considered for this study, you must be at least 18 years old. People of any gender can take part.

Specific details about your health and medical history would be checked by the study team to make sure this study is right for you. They will need to confirm that you have WHIM syndrome.

It's important to discuss with your doctor if taking part in this study is suitable for your individual health situation.

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 WHIM syndrome?
Are you able to attend regular clinic visits?
Are you willing to potentially receive a placebo for the first 12 months?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first go through a screening process to make sure you meet all the requirements. This would involve medical checks and tests. If you qualify, you'd then be assigned to receive either the study medicine (Mavorixafor) or a placebo (dummy pill) for the first 12 months. You wouldn't know which one you're getting, and neither would your study doctor.

During this time, you would have regular visits to the clinic, likely every three months, for blood tests to check your infection-fighting cells, as well as physical exams and other checks like heart readings (ECGs) and eye exams. The study team will also ask about any infections you've had and look at any warts. After these first 12 months, if you wish, you would then receive the active medicine Mavorixafor in an 'open-label' period, meaning everyone knows they are getting the medicine. This period would continue for a longer time, with ongoing checks of your health, safety, and how you're responding to the treatment.

Potential risks and benefits

Taking part in a study like this could potentially offer benefits, such as access to a new medicine that might improve your WHIM syndrome symptoms before it's widely available. However, there's no guarantee the medicine will work for you, or that you'll even receive the active medicine during the first part of the study if you're assigned to the placebo group. Like all medicines, Mavorixafor may have side effects, which the study team will carefully monitor. You will always have the right to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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

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Unverified
Spain
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Unverified
France
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Unverified
Italy
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Unverified
Netherlands
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Unverified
Denmark

Common questions

What is WHIM syndrome?

WHIM syndrome is a rare condition that affects the immune system, making people more prone to infections and certain skin issues like warts.

What is Mavorixafor?

Mavorixafor is the name of the new medicine being tested in this study for people with WHIM syndrome.

What is a placebo?

A placebo is a 'dummy pill' that looks exactly like the study medicine but contains no active ingredients. It's used to fairly compare the new medicine against no treatment.

What does 'double-blind' mean?

Double-blind means that during part of the study, neither the participants nor their doctors will know whether they are receiving the actual medicine or the placebo. This helps ensure fair results.

How long will the study last?

The first comparative part of the study will last for 12 months. After that, there's an option to continue receiving the medicine in a longer-term open-label phase.