What side effects are associated with this type of intervention?

Common treatments for neutropenia include granulocyte colony-stimulating factors (G-CSFs) like filgrastim and pegfilgrastim, which can cause bone pain, headache, and injection site reactions. CXCR4 antagonists, such as mavorixafor, are being studied for their impact on chronic neutropenia. While specific side effects of mavorixafor are still under investigation, CXCR4 antagonists generally may cause gastrointestinal disturbances, fatigue, and dizziness. It is important to discuss potential side effects with a healthcare provider to tailor treatment to individual needs.

What prior FDA approvals exist?

The FDA has approved several treatments for neutropenia, primarily focusing on granulocyte colony-stimulating factors (G-CSFs) like filgrastim (Neupogen) and pegfilgrastim (Neulasta), which stimulate the bone marrow to produce more neutrophils. Another relevant drug is plerixafor (Mozobil), a CXCR4 antagonist like mavorixafor, which is used to mobilize hematopoietic stem cells for transplantation in patients with multiple myeloma and non-Hodgkin's lymphoma. These treatments aim to reduce the risk of infection by increasing neutrophil counts in patients with neutropenia.

What other types of research exist?

Promising novel alternatives to Mavorixafor for neutropenia patients include: 1) G-CSF (Granulocyte Colony-Stimulating Factor) analogs, which stimulate the production of neutrophils. 2) Plerixafor (Mozobil), which mobilizes hematopoietic stem cells and may be used in specific clinical circumstances. 3) Thrombopoietin receptor agonists, which have shown potential in promoting hematopoietic recovery. These alternatives have demonstrated efficacy in clinical trials and may offer viable options for neutropenia treatment in 2024.

← Back to Search

CXCR4 Inhibitor

Mavorixafor for Neutropenia

Phase 1 & 2

Waitlist Available

Research Sponsored by X4 Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants who are not on G-CSF must be off for ≥14 days prior to the Baseline visit and have an ANC ≤1000 cells/µL at the Screening visit.

Participant has a history of symptomatic chronic neutropenia confirmed by the Investigator.

Must not have

Known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immune deficiency syndrome.

- Systemic glucocorticoids (>5 mg prednisone equivalent per day).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing mavorixafor, a pill that may help people with chronic neutropenia by increasing their white blood cell counts. The study includes people with different types of chronic neutropenia who often have low levels of these important infection-fighting cells. Researchers want to see if taking mavorixafor daily for several months can safely and effectively raise their neutrophil counts. Mavorixafor has been shown to increase white blood cell counts in patients with WHIM syndrome in previous studies.

Who is the study for?

This trial is for people with chronic neutropenia, including congenital and idiopathic types. Eligible participants must have been diagnosed at least 6 months prior, weigh over 15 kg, can be on or off G-CSF treatment but with specific ANC levels, and agree to use effective contraception. Those with active infections or certain medical conditions are excluded.

What is being tested?

The study tests mavorixafor's effects on individuals with chronic neutropenia in two parts: Part 1 (Phase 1b) explores how it impacts patients; Part 2 (Phase 2) assesses safety of long-term dosing and its effect on neutropenia after six months.

What are the potential side effects?

While the side effects of mavorixafor will be closely studied during this trial, potential risks may include allergic reactions to the drug or its ingredients, digestive issues from capsule ingestion, and possible interactions with other medications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I haven't taken G-CSF for 14 days and my white blood cell count is low.

Select...

I have a long-term low white blood cell count that causes symptoms.

Select...

I showed improvement after the first part of the treatment.

Select...

I have been on a stable dose of G-CSF for at least 14 days and my white blood cell count is not above 10,000.

Select...

I have had low white blood cell counts for over 6 months not due to medications, infections, or cancer.

Select...

I weigh at least 15 kilograms.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have tested positive for HIV or have been diagnosed with AIDS.

Select...

I am taking more than 5 mg of prednisone or its equivalent daily.

Select...

My kidney function, measured by eGFR, is 60 mL/min/1.73m^2 or less.

Select...

I cannot swallow mavorixafor capsules.

Select...

I currently have COVID-19 or tested positive recently.

Select...

I am not taking medications that interact with specific body enzymes or transporters.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MavorixaforExperimental Treatment1 Intervention

Part 1: Adult participants and adolescent participants who weigh more than 50 kilograms (kg) will receive mavorixafor 400 milligrams (mg) (4 capsules of 100 mg each), orally once on Day 1. Adolescents weighing less than or equal to 50 kg will receive mavorixafor 200 mg (2 capsules of 100 mg each), orally once on Day 1. Part 2: Eligible participants from Part 1 will receive once daily dosing of mavorixafor for 6 months.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mavorixafor

2021

Completed Phase 2

~50

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for neutropenia include granulocyte colony-stimulating factors (G-CSFs) like filgrastim, which stimulate the bone marrow to produce more neutrophils, and antibiotics to prevent or treat infections. Mavorixafor, a CXCR4 antagonist, works by blocking the CXCR4 receptor, which is involved in the retention of neutrophils in the bone marrow. By inhibiting this receptor, mavorixafor promotes the release of neutrophils into the bloodstream, thereby increasing neutrophil counts. This mechanism is crucial for neutropenia patients as it helps to reduce the risk of infections and improve overall immune function.

Thrombotic microangiopathy (TMA) in adult patients with solid tumors: a challenging complication in the era of emerging anticancer therapies.Cluster-Like Headache Revealing Polycythemia Vera: A Case Report.[The blood-simulating properties of a recombinant colony-stimulating factor and glycyram during cytostatic myelosuppression].