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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

PHASE3RECRUITING

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.

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Study details:

All participants will continue their pre-study background therapy, defined as the participant's current treatment regimen. Options include, but are not limited to granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or "watchful waiting".

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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.

Have a confirmed trough ANC <1500 cells/µL during the screening visit (single ANC measurement) and at baseline visit (mean ANC over 6 hours) held at least 2 weeks prior to Day 1 dosing, with no clinical evidence of 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 at least 1 of the following criteria: Infection requiring the use of antibiotics (intravenous [IV]/oral/topical) 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).

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.

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, irritable 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 chronic neutropenias (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 Sponsor, interfere with the objectives of the study.

Received more than 1 dose of mavorixafor in the past.

Received 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 have taken other investigational drug at least 30 days prior to the screening visit.

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Eligibility

Age eligible for study : 12 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2024-06-06

Primary completion: 2026-07-01

Study completion finish: 2026-08-01

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT06056297

Intervention or treatment

DRUG: Mavorixafor

DRUG: Placebo

Conditions

• Neutropenia

Condition: Neutropenia
DRUG: Mavorixafor and other drugs
Southport, Not Specified, Australia
Sponsor: X4 Pharmaceuticals

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

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Find a site

Closest Location:

Icon Cancer Centre Southport

Research sites nearby

Select from list below to view details:

Icon Cancer Centre Southport
Southport, Not Specified, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Mavorixafor Participants will receive mavorixafor orally once daily starting from Day 1 through Week 52.	DRUG: Mavorixafor Mavorixafor will be administered per schedule specified in the arm description.
PLACEBO_COMPARATOR: Placebo Participants will receive placebo to match mavorixafor orally once daily starting from Day 1 through Week 52.	DRUG: Placebo Placebo will be administered per schedule specified in the arm description.

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Annualized Infection Rate Based on Infections Adjudicated by Blinded, Independent Adjudication Committee (BIAC) During the Treatment Period	Not Specified	Up to 52 Weeks
Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response	Positive ANC response: ANC ≥1500 cells/microliter (µL), with the exception of participants with Baseline ANC \<500 cells/µL; and ≥2-fold increase in ANC from baseline, for participants with baseline ANC \< 500 cells/μL.	Baseline, Week 52

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) as Assessed by a BIAC During the Treatment Period	Not Specified	Up to 52 Weeks
Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections	Not Specified	Up to 52 Weeks
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period	Not Specified	Up to 52 Weeks
Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period	Not Specified	Up to 52 Weeks
Change From Baseline in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score	Not Specified	Baseline, Week 52

Frequently Asked Questions

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References

Clinical Trials Gov: 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