Active not recruitingPHASE3INTERVENTIONAL

Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

This research study is looking into a new medication called mavorixafor for people living with WHIM syndrome. WHIM syndrome is a rare condition that affects the immune system. The main goals are to find out how well mavorixafor works at increasing certain white blood cells (called neutrophils), which are important for fighting infections, and to check if the medicine is safe and well-tolerated. The study will involve two main parts: a period where some participants receive mavorixafor and others receive a dummy medicine (placebo) without knowing which they have, and then an open-label period where everyone taking part can receive mavorixafor. It's hoped this study will provide important information about future treatment options for WHIM syndrome.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

X4 Pharmaceuticals

Enrolment target

Start

24 Oct 2019

Estimated completion

01 Dec 2025

WHIM Syndrome

What is this study about?

This study is designed to learn more about a new medicine called mavorixafor, specifically for people who have a rare condition called WHIM syndrome. WHIM syndrome affects your immune system, making it harder for your body to fight off infections. People with WHIM syndrome often have low levels of certain white blood cells, called neutrophils, which are crucial for staying healthy and preventing infections.

The main purpose of this study is to see if mavorixafor can help increase the number of these important neutrophils in the blood. Researchers also want to make sure the medicine is safe and doesn't cause too many side effects. This is a "Phase 3" study, which means it's one of the final steps in testing a new medicine before it might be made available to everyone.

The study has two main parts. In the first part, participants will be randomly assigned to either receive mavorixafor or a placebo (a dummy medicine with no active ingredients). Neither you nor your study doctor will know which you are receiving, which helps make the results fair. In the second part, called the open-label period, everyone who continues in the study will receive mavorixafor. This part focuses on checking the long-term safety of the medicine.

Key takeaways

This study is testing a new medicine for WHIM syndrome.
The new medicine aims to boost important infection-fighting cells.
Participation involves taking medication and regular check-ups.
Some participants will initially receive a dummy medicine (placebo).
Safety and effectiveness are the main focus of this research.
You can discuss any concerns with your study team or doctor.

Who may be eligible?

To join the first part of this study, you would need to be at least 12 years old and have a confirmed diagnosis of WHIM syndrome through a genetic test. It's important that your blood tests show low levels of neutrophils, even when you aren't fighting an infection. You also need to agree to use effective contraception if you're able to become pregnant, and be willing to follow all the study instructions.

If you successfully complete the first part of the study, or if you needed to leave the first part early for certain reasons, you could potentially join the second, open-label part.

However, you would not be able to join the study if you are pregnant or breastfeeding, or if you have ever had a serious allergic reaction to mavorixafor or any of its ingredients. The study doctor would also review your overall health to make sure that taking part would be safe for you and that you could complete all the study 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 at least 12 years old?
Do you have a genetically confirmed diagnosis of WHIM syndrome?
Do your blood tests show low levels of certain white blood cells (neutrophils) when you're not infected?
Are you not pregnant or breastfeeding?
Are you willing and able to follow all study instructions and attend appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will involve regular visits to a clinic. During these visits, the study team will take blood samples, measure your vital signs, and ask you questions about your health and any side effects you might be experiencing. You would be given either mavorixafor or a dummy medicine (placebo) to take regularly, following specific instructions.

The first part of the study, where some people get the active medicine and others get the placebo, will last for a set period. After that, if you choose, you may be able to continue receiving mavorixafor in the open-label part of the study. This open-label period could last until the medicine becomes widely available, or until the study officially ends. The total duration of your involvement would depend on several factors, including how long the study continues and your individual journey through it.

Potential risks and benefits

Participating in this study might offer some potential benefits. You would receive close medical attention and monitoring from study doctors and nurses, and you might be among the first to receive this new potential treatment for WHIM syndrome. However, there are also potential risks involved. Like any medicine, mavorixafor could cause side effects, and some might be serious. It's also possible that the medicine may not work for you, or you might receive the placebo during the first part of the study. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (23)

University of California San Diego Health/Rady Children's Hospital
Verified postcode
San Diego, United States
California Dermatology Institute
Verified postcode
Thousand Oaks, United States
University of Iowa
Verified postcode
Iowa City, United States
Johns Hopkins University Medical Center
Verified postcode
Baltimore, United States
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States
University of Washington Medical Center
Verified postcode
Seattle, United States
Wesley Hospital
Verified postcode
Auchenflower, Australia
Children's Health Queensland Hospital
Verified postcode
South Brisbane, Australia
Medical University of Vienna - Medizinische Universität Wien
Verified postcode
Vienna, Austria
Aarhus University Hospital
Verified postcode
Aarhus, Denmark
CHU de Lyon, Institut d'Hematologie et d'Oncologie Pediatrique
Verified postcode
Lyon, France
CHU Paris Est, Hôpital d'Enfants Armand-Trousseau
Verified postcode
Paris, France

Common questions

What is WHIM syndrome?

WHIM syndrome is a rare condition that affects your immune system, making it harder for your body to fight off infections. It's linked to changes in a specific gene.

What are neutrophils and why are they important?

Neutrophils are a type of white blood cell. They're like your body's tiny soldiers, crucial for finding and destroying germs, helping to prevent infections.

What does a 'placebo' mean?

A placebo is a dummy medicine that looks exactly like the real medicine but contains no active ingredients. It helps researchers understand if the real medicine is truly working.

Will I know if I'm getting the real medicine or the placebo?

During the first part of the study, neither you nor your doctor will know if you're receiving mavorixafor or the placebo. This is called 'double-blind' and helps ensure fair results.

How long will the study last?

The study has two parts. The first part has a set duration. If you continue into the second part, it could last until the medicine becomes available or the study ends, so the total time can vary.