The 4WARD Study

The 4WARD Study is a phase 3 clinical research study now enrolling people living with chronic neutropenia. Researchers aim to learn if mavorixafor, the investigative study medicine, may increase neutrophil counts and decrease the chance of getting infections.

What is chronic neutropenia?

Chronic neutropenia encompasses a group of rare blood disorders that can be:

Inherited (called congenital or cyclic)
Immune mediated (called acquired primary autoimmune)
Occur for no known or identifiable reason (called idiopathic)

Neutrophils are a type of white blood cell that helps the body defend itself against infections. People with neutropenia have a low number of neutrophils in the blood. When neutrophil counts are low, there is a greater risk of infections such as ear infections, pneumonia, gingivitis, skin infections, and more.

Diagnosis

Chronic neutropenia is diagnosed through a blood test. Genetic testing can be used to help identify or rule out congenital neutropenia.

Treatments

Chronic neutropenia is a disease with few approved treatments, so more treatment options are needed. Current treatments include granulocyte colony-stimulating factor (G-CSF), an injection that helps the body make neutrophils, and intravenous immunoglobulin (IVIG), an infusion of antibodies to help your immune system fight off germs and disease.

This is a phase 3, randomized, double-blind, placebo controlled, multicenter study of mavorixafor in participants with congenital and acquired primary autoimmune and idiopathic chronic neutropenia disorders who are experiencing recurrent and/or serious infections.

Phase 3: A larger study that compares the safety and effectiveness of a new treatment against a current standard treatment while checking side effects.
Randomized: To ensure there is no bias or unfairness in the study results, study participants are assigned by chance to receive either the investigative medicine or a placebo.
Double-blind: A type of study where the patient as well as the study team do not know who is assigned to take the investigative medicine and who is assigned to take the placebo. Double-blinding helps make sure that opinion, bias and behavior do not influence the interpretation of study results.
Placebo-controlled: A type of study that has one group of study participants who take the investigative medicine with active ingredients, and another group that gets the placebo, which has no active ingredients.
Multicenter: A study conducted at many clinics.

Interested in learning more about the 4WARD Study?

Talk to your doctor to see if joining a clinical study is a good option for you.

You may be eligible to join the study if you:

Are age 12 and older
Are diagnosed with chronic neutropenia (including congenital, acquired primary autoimmune and idiopathic chronic neutropenia)
Are taking or not taking any treatment for your chronic neutropenia, including granulocyte colony-stimulating factor (G-CSF)

For more details, visit https://clinicaltrials.gov/ct2/show/NCT06056297

Interested in joining? Please contact ClinicalTrialInfo@x4pharma.com .

What to expect if you participate in the study

Potential study participants will meet with the study team for a screening visit to determine if they meet the eligibility criteria and ensure that the risks and benefits are understood. This is called the informed consent process. Deciding to enroll in a study is a personal decision.

This study to learn about the investigational study medicine, mavorixafor, will last about 12 months.

Study participants will be randomly assigned to get either:

Mavorixafor, the investigative study medicine, along with their current treatment.

Placebo, a substance made to look like the investigative study medicine that has no active ingredients, along with their current treatment

Study-related treatments and procedures are provided at no cost to participants. If needed, approved travel, lodging, and meals will be reimbursed by the study sponsor, X4 Pharmaceuticals.

At study appointments, the study team measures the effects and the safety of the study medicine on your neutropenia by drawing blood and administering short questionnaires about your symptoms. Phase 3 study participants will undergo an ophthalmology (eye) exam and other exams and procedures.

Study Sponsor

The 4WARD Study is sponsored by X4 Pharmaceuticals, Inc., a pharmaceutical company developing treatments for diseases of the immune system, with a focus on rare diseases and those with limited treatment options.

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References

“A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenia Disorders Who Are Experiencing Recurrent and/or Serious Infections.” ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT06056297?term=NCT06056297&rank=1. Accessed June 1, 2024.
Dale DC, et al. An update on the diagnosis and treatment of chronic idiopathic neutropenia. Current Opinion in Hematology. 2017;24(1):46-53.
Donadieu J, et al. Chronic neutropenia: How best to assess severity and approach management? Expert Rev Hematol. 2021;14:945-960.
“Glossary of Common Terms.” National Institutes of Health (NIH). Apr. 2023. https://www.nih.gov/health-information/nih-clinical-research-trials-you/glossary-common-terms. Accessed June 1, 2024.
“Glossary of Terms.” U.S. Food and Drug Administration, FDA. https://www.fda.gov/patients/clinical-trials-what-patients-need-know/glossary-terms. Accessed June 1, 2024.
Kolaczkowska E, et al. Neutrophil recruitment and function in health and inflammation. Nature Reviews Immunology. 2013;13(3):159-175.
Methods and Applications of Statistics in Clinical Trials: Concepts, Principles, Trials, and Design, Volume 1. https://onlinelibrary.wiley.com/doi/abs/10.1002/9781118596005.ch45 Accessed June 1, 2024.