RecruitingPHASE3INTERVENTIONAL

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

This research study is testing a new medicine called Mavorixafor for people who have neutropenia, a condition where your body doesn't produce enough white blood cells called neutrophils. Neutrophils are very important for fighting off infections. People with neutropenia often get frequent and serious infections. The study wants to see if Mavorixafor can help by increasing the number of these infection-fighting cells and, in turn, reduce how often people get sick. Some people will receive Mavorixafor, while others will receive a placebo (a dummy pill with no medicine) to compare the effects. The study will also carefully check if the medicine is safe and if people can tolerate it well.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

X4 Pharmaceuticals

Enrolment target

176

Start

06 Jun 2024

Estimated completion

01 Nov 2027

Neutropenia

What is this study about?

This study is for individuals diagnosed with neutropenia, which is a condition where your body has a lower-than-normal count of white blood cells called neutrophils. These neutrophils are a crucial part of your immune system, acting like tiny soldiers that fight off infections. Because of this low count, people with neutropenia are more prone to getting frequent and serious infections.

The main goal of this study is to investigate a new medication called Mavorixafor. Researchers want to find out if Mavorixafor can effectively increase the number of neutrophils in your blood and, as a result, help reduce the number and severity of infections you experience. They will also be carefully monitoring how safe the medicine is and if people taking it have any side effects.

Participants in the study will continue to receive their usual treatments for neutropenia, such as G-CSF injections (a growth factor that helps the body make more neutrophils), immunoglobulin therapy, or antibiotics. This new medicine, Mavorixafor, would be added to their existing care. By comparing those who take Mavorixafor with those who take a placebo (an inactive tablet that looks just like the study drug), the researchers can determine if Mavorixafor truly makes a difference.

Key takeaways

This study is for people with certain types of neutropenia.
It's testing a new drug, Mavorixafor, to boost infection-fighting cells.
The goal is to reduce frequent and serious infections.
Participants will continue their current neutropenia treatments.
It involves regular clinic visits for checks and blood tests.
You might receive the new drug or a dummy pill (placebo).

Who may be eligible?

To join this study, you need to have a specific type of neutropenia – either a condition you were born with (congenital) or one you developed later (acquired primary autoimmune and idiopathic chronic neutropenia). This diagnosis needs to have been made at least six months ago, and your neutropenia should not be caused by other health issues like certain medicines, active infections, or cancer.

You would also need to have a low level of neutrophils in your blood (less than 1000 cells/µL) and a history of infections. This means you would have had at least two infections in the past year that required antibiotics (either by mouth or injection) or a visit to a healthcare facility like an emergency room or your GP. Importantly, your doctor must believe these infections are related to your neutropenia, and you shouldn't have any active widespread infections when you join the study.

The study is open to anyone aged 12 years and older. Your doctor will carefully review your medical history and conduct tests to confirm if you meet all the necessary requirements to participate safely in the study.

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.

Have I been diagnosed with a congenital or acquired primary autoimmune/idiopathic chronic neutropenic disorder for at least 6 months?
Do I have low neutrophil counts (ANC less than 1000 cells/µL)?
Have I had at least two infections requiring antibiotics or a hospital visit in the last year?
Am I at least 12 years old?
Is my neutropenia NOT caused by medicines, other infections, or cancer?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will continue with your current neutropenia treatments, such as G-CSF, immunoglobulin therapy, or antibiotics. On top of this, you will either receive the study medicine, Mavorixafor, or a placebo (a dummy pill with no active drug). You won't know which one you are receiving. You'll need to visit the study clinic regularly for appointments. These visits will involve various assessments, including blood tests to check your neutrophil levels and overall health, physical examinations, and discussions about any infections you've had or any side effects you might be experiencing. The total duration of your participation in the study, including follow-up, will be decided by the researchers and explained to you in detail.

Potential risks and benefits

Participating in this study might offer potential benefits, such as a possible improvement in your neutrophil counts and a reduction in infections if you receive the active study drug. However, there's also a chance you could receive the placebo, meaning you wouldn't get the potential benefits of the new medicine. As with any medication, Mavorixafor could have side effects, and some tests and procedures in the study might carry minor risks or discomfort. All potential risks and discomforts will be explained fully before you decide to join. Remember, taking part in a clinical trial is always voluntary, and you are free to withdraw at any time without affecting your medical care.

Locations (112)

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

Phoenix Children's Hospital
Verified postcode
Phoenix, United States· Recruiting
UC Irvine
Verified postcode
Irvine, United States· Recruiting
Orso Health
Verified postcode
La Jolla, United States· Recruiting
University of California, Los Angeles-UCLA
Verified postcode
Los Angeles, United States· Recruiting
Orso Health
Verified postcode
Torrance, United States· Recruiting
University of Southern Florida
Verified postcode
St. Petersburg, United States· Recruiting
Children's Healthcare of Atlanta (CHOA)
Verified postcode
Atlanta, United States· Recruiting
University of Iowa
Verified postcode
Iowa City, United States· Recruiting
Boston Children's Cancer Center
Verified postcode
Boston, United States· Recruiting
University of Michigan
Verified postcode
Ann Arbor, United States· Recruiting
Washington University
Verified postcode
St Louis, United States· Recruiting
Duke University Medical Center
Verified postcode
Durham, United States· Recruiting

Common questions

What are neutrophils and why are they important?

Neutrophils are a type of white blood cell that play a key role in your immune system, helping your body fight off infections from germs like bacteria and fungi.

What does 'placebo' mean in this study?

A placebo is a 'dummy pill' that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the effects of the actual medicine.

Will I still take my regular neutropenia medicine if I join?

Yes, you will continue all your regular treatments for neutropenia, such as G-CSF injections, while participating in the study. Mavorixafor is added on top of your existing care.

Who is funding this research?

This study is sponsored by X4 Pharmaceuticals, the company that developed Mavorixafor.

Can I leave the study if I change my mind?

Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

How to find out more

Patient Affairs and Advocacy

clinicaltrialinfo@x4pharma.com 857-529-5779 Official trial record

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