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
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter 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 join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 12 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests whether mavorixafor helps people with long-term neutropenia who have repeated or serious infections. Neutropenia means having too few neutrophils, a type of white blood cell that fights infection. The study checks whether the drug raises these cell counts, provides health benefits, and is safe and tolerable.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Diagnosed at least 6 months before screening with an eligible long-term neutropenia disorder, not caused by medicines, recent or active infection, or cancer.
- Have an eligible condition present from birth, primary autoimmune neutropenia (the immune system attacks neutrophils), or idiopathic neutropenia (no known cause). Some other types need approval from the study medical monitor.
- Have a neutrophil count below 1,000 cells per microliter at screening, confirmed by repeated blood tests over 6 hours at the baseline visit.
- Have no signs of an infection affecting the whole body when these blood counts are checked.
- Have had at least 2 infections in the past 12 months that needed antibiotics or a visit to a healthcare facility.
- The study doctor must think these infections were likely linked to your neutropenia.
- If taking G-CSF (a medicine that raises neutrophil counts) or another treatment for neutropenia, have used it for the past 12 months while still having infections. The dose and schedule must be stable for at least 4 weeks before screening.
- Be willing to keep your usual treatment dose and schedule stable during the study, except for safety reasons or allowed changes for high neutrophil counts.
Who can't join
- Neutropenia caused by an overactive spleen, infection, cancer, another autoimmune disease, poor nutrition, or medicines.
- Aplastic anemia (bone marrow makes too few blood cells), WHIM syndrome, neutropenia with counts that rise and fall in cycles, or certain disorders affecting immune control or bone marrow function.
- Neutropenia linked to the inherited Duffy-null trait. Some people with this trait and a specific gene change causing neutropenia may still qualify.
- A medical or personal condition that could make the study unsafe, prevent completion, or affect the study’s goals.
- More than 1 past dose of mavorixafor, or use of another CXCR4 blocker (a similar type of drug) in the past 6 months.
- Use of pegylated G-CSF, a long-acting medicine that raises neutrophil counts, unless neutropenia present from birth is confirmed at screening.
- Current or recent use of another study drug: within 30 days before screening, or longer if needed for 5 drug half-lives (the time for the drug level to drop by half each time).
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Key Inclusion 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 malignancy. * Congenital Neutropenia, including but not limited to these classifications: 1. 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) 2. Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease 3. Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b) 4. Shwachman-Diamond syndrome * Acquired Primary Neutropenia 1. Chronic idiopathic neutropenia 2. 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 systemic infection. * 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 months that meet the following criteria: * 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 participants: * 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 for ≥4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC \>10,000 cells/µL for ≥4 weeks). * 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 Exclusion Criteria: * A diagnosis of secondary neutropenia including those due to: 1. Hypersplenism 2. Infection 3. Malignancy 4. Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease 5. Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition 6. Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies. * A diagnosis of any of the following: 1. Aplastic anemia 2. Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome 3. Certain CNs, including but not limited to these classifications are excluded: 1. Isolated with a cyclic presentation, for example, elastase, neutrophil expressed (ELANE) 2. Associated with immune dysregulation, for example, common variable immunodeficiency (CVID), autoimmune lymphoproliferative syndrome (ALPS), familial hemophagocytic lymphohistiocytosis, Chédiak-Higashi syndrome, GATA-binding protein 2 (GATA2) deficiency syndrome 3. Associated with bone marrow failure, for example, Fanconi anemia, Diamond-Blackfan anemia 4. 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 background may be eligible for inclusion * 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 the Medical Monitor, interfere with the objectives of the study. * 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 apply.
Study sites (22)
AZ
- Phoenix Children's Hospital · Phoenix, AZ 85016More trials in Phoenix
CA
- UC Irvine · Irvine, CA 92697More trials in Irvine
- Orso Health · La Jolla, CA 92037More trials in La Jolla
- University of California, Los Angeles-UCLA · Los Angeles, CA 90095More trials in Los Angeles
- Orso Health · Torrance, CA 90505More trials in Torrance
FL
- University of Southern Florida · St. Petersburg, FL 33701More trials in St. Petersburg
GA
- Children's Healthcare of Atlanta (CHOA) · Atlanta, GA 30322More trials in Atlanta
IA
- University of Iowa · Iowa City, IA 52242More trials in Iowa City
MA
- Boston Children's Cancer Center · Boston, MA 02143More trials in Boston
MI
- University of Michigan · Ann Arbor, MI 48109More trials in Ann Arbor
MO
- Washington University · St Louis, MO 63130More trials in St Louis
NC
- Duke University Medical Center · Durham, NC 27710More trials in Durham
OH
- Cincinnati Children's Hospital Medical Center · Cincinnati, OH 45229More trials in Cincinnati
- Cleveland Clinic · Cleveland, OH 44195More trials in Cleveland
PA
- The Children's Hospital of Philadelphia · Philadelphia, PA 19104More trials in Philadelphia
TN
- St. Jude Childrens; Research Hospital · Memphis, TN 38105More trials in Memphis
TX
- UT Southwestern Medical Center · Dallas, TX 75390More trials in Dallas
- Cook Children's Medical Center · Fort Worth, TX 76104More trials in Fort Worth
- Texas Children's Hospital/ Baylor College of Medicine · Houston, TX 77030More trials in Houston
- Sarah Cannon Research Institute · Woodland, TX 77380More trials in Woodland
UT
- University of Utah · Salt Lake City, UT 84112More trials in Salt Lake City
WI
- Froedtert And Medical College Of Wisconsin · Milwaukee, WI 53226More trials in Milwaukee