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New treatment option for Neutropenia
Official 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
ClinicalTrials.gov ID: NCT06056297
What this study is testing
What is Mavorixafor?
Mavorixafor is an investigational medicine, being studied as a potential treatment for neutropenia.
Also referred to as X4P-001.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not...
- Congenital Neutropenia, including but not limited to these classifications:
- Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor...
- Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3)...
- Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
You likely can't join if
- A diagnosis of secondary neutropenia including those due to:
- Hypersplenism
- Infection
- Malignancy
- Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid...
- Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
See the full eligibility criteria
- Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or...
- Congenital Neutropenia, including but not limited to these classifications:
- Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor (CSF3R), C-X-C chemokine receptor 2 (CXCR2), Wiskott-Aldrich syndrome (WAS)
- Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease
- Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
- Shwachman-Diamond syndrome
- Acquired Primary Neutropenia
- Chronic idiopathic neutropenia
- Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with study Medical Monitor.
- Have an ANC \<1000 cells/µL during screening (single ANC value from hematology) and confirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) at baseline visit, with no clinical evidence of...
- Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12...
- Infection requiring the use of antibiotics (intravenous [IV]/oral); OR
- Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization); AND for all potential...
- Infections considered by the Investigator to be likely related to the potential participant's CN disorder.
- Participants who are on G-CSF or other active background therapy must have been receiving these therapies during the previous 12 months while continuing to suffer from infections, be on a stable dose and dosing schedule...
- Participants must be willing to keep their G-CSF or other background therapy doses/regimens stable (other than for safety reasons) for the duration of the study. Key
- A diagnosis of secondary neutropenia including those due to:
- Hypersplenism
- Infection
- Malignancy
- Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease
- Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
- Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.
- A diagnosis of any of the following:
- Aplastic anemia
- Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome
- Certain CNs, including but not limited to these classifications are excluded:
- Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE)
- Associated with immune dysregulation, for example, common variable immunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS), familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome...
- Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia
- Neutropenia associated with a Duffy-null phenotype (formerly known as benign ethnic neutropenia). However, a participant with an autosomal dominant pathogenic variant in a gene associated with CN on a Duffy-null...
- A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or...
- Received more than 1 dose of mavorixafor in the past.
- Received C-X-C chemokine receptor 4 (CXCR4) antagonist (other than mavorixafor) in the past 6 months.
- Participants taking pegylated-G-CSF unless they have a diagnosis of congenital neutropenia confirmed at screening.
- Participant is currently taking or has taken other investigational drug \<30 days prior to the screening visit or 5 half-lives, whichever is longer. Note: Other protocol-defined inclusion and exclusion criteria may...
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Irvine, California, United States
- La Jolla, California, United States
- Los Angeles, California, United States
- Torrance, California, United States
- St. Petersburg, Florida, United States
- Atlanta, Georgia, United States
- Iowa City, Iowa, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- St Louis, Missouri, United States
- Durham, North Carolina, United States
- Cincinnati, Ohio, United States
- Cleveland, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Dallas, Texas, United States
- Fort Worth, Texas, United States
- Houston, Texas, United States
- Woodland, Texas, United States
+ 70 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Irvine, California, United States; La Jolla, California, United States; Los Angeles, California, United States; Torrance, California, United States; St. Petersburg, Florida, United States and 84 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.