Actively Recruiting
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Experiencing Recurrent or Serious Infections
Led by X4 Pharmaceuticals · Updated on 2026-06-05
176
Participants Needed
114
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the effects of mavorixafor in people aged 12 and older who have congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These disorders cause low levels of neutrophils, a type of white blood cell, leading to recurrent or serious infections. The study aims to show if mavorixafor can increase neutrophil levels and reduce infections, while also evaluating the drugs safety and how well participants tolerate it. Participants will continue their current treatments, which may include granulocyte-colony stimulating factor G-CSF, immunoglobulin replacement therapy, prophylactic antibiotics, or no active treatment. They will be randomly assigned to receive either mavorixafor or a placebo once daily by mouth for 52 weeks. The study is double-blind, meaning neither participants nor researchers will know which treatment is given until the study ends. Throughout the study, participants will be monitored for infections and neutrophil counts, with assessments including infection rates reviewed by a blinded committee. Other measures include infection severity, duration, antibiotic use, presence of oral ulcers, and fatigue levels from patient questionnaires. The research team will track safety and tolerability during the 52-week treatment period, with follow-up assessments to observe outcomes and any side effects.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of congenital or acquired primary autoimmune or idiopathic chronic neutropenic disorder for at least 6 months not due to medications, infections, or malignancy
- Congenital neutropenia including isolated permanent forms, those with extra-hematologic manifestations, metabolic disorders, or Shwachman-Diamond syndrome
- Acquired primary neutropenia including chronic idiopathic or primary autoimmune neutropenia
- Absolute neutrophil count (ANC) below 1000 cells/µL during screening and confirmed low ANC at baseline without systemic infection
- History of at least 2 infections in the past 12 months requiring antibiotics or medical visits linked to neutropenia
- Stable use of granulocyte-colony stimulating factor (G-CSF) or other background therapy for at least 4 weeks prior to screening and willingness to maintain stable dosing during the study
You will not qualify if you...
- Diagnosis of secondary neutropenia caused by hypersplenism, infection, malignancy, autoimmune diseases, nutritional deficiency, or drug-induced causes
- Diagnosis of aplastic anemia, WHIM syndrome, or certain cyclic or immune dysregulation-associated congenital neutropenias
- Neutropenia linked to a Duffy-null phenotype unless accompanied by a pathogenic variant
- Medical or personal conditions that may affect safety or study completion
- Prior receipt of more than one dose of mavorixafor or other CXCR4 antagonists within 6 months
- Use of pegylated-G-CSF unless congenital neutropenia diagnosis is confirmed
- Use of other investigational drugs within 30 days or five half-lives before screening
Research Team
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Patient Affairs and Advocacy
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