The 4WHIM Trial is a phase 3 clinical research study to see if mavorixafor, an investigational treatment, can lower the number of infections and warts in people living with WHIM syndrome. Enrollment to this study is now closed. Sign up for updates on trial outcomes.

What is WHIM syndrome?

WHIM syndrome is a rare and difficult-to-diagnose primary immunodeficiency in which the body’s immune system does not function properly and has trouble fighting infections. Some doctors also call primary immunodeficiencies inborn errors of immunity, which are genetic mutations that cause immunodeficiencies. WHIM syndrome is caused by mutations in the CXCR4 gene which causes white blood cells to get trapped in the bone marrow.

WHIM syndrome was named after four symptoms that most diagnosed patients have experienced: Warts, Hypogammaglobulinemia, Infections, Myelokathexis. There are no existing treatments that specifically target the underlying problem of WHIM syndrome—new treatments are needed.

4WHIM trial overview

The goals of the new phase 3 study are to find out if the investigational oral drug mavorixafor can safely reduce bacterial infections and warts in people with WHIM syndrome.

The 4WHIM Trial is a phase 3, randomized, double-blind, placebo-controlled study in patients with WHIM syndrome with an open-label extension study.

About mavorixafor

There are no approved therapies for WHIM syndrome. Doctors can treat symptoms but not the cause. For example, G-CSF is an injection given twice a day that stimulates neutrophil production. IVIG is an infusion of antibodies given every few weeks to help your immune system fight off germs and disease. Unfortunately, neither of these fully resolves all symptoms.

One new potential medication that specifically focuses on the underlying problem of WHIM syndrome is mavorixafor. Mavorixafor is an oral investigational medication (in capsules) which is taken once a day. This medication turns on the “green light” for white blood cells to leave the bone marrow and be available in the bloodstream to fight infections in WHIM syndrome patients.

What did we learn from earlier studies?

Researchers learned in phase 1 and phase 2 studies that mavorixafor may help people with WHIM syndrome by targeting the way the disease manifests.

Study Sponsor

The 4WHIM Trial is sponsored by X4 Pharmaceuticals, Inc., a global pharmaceutical company developing treatments that may have a profound impact for patients with rare diseases (such as WHIM syndrome) and cancer.

To learn more about X4 Pharmaceuticals, visit www.X4Pharma.com.

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