Ongoing Clinical Trials for Neutropenia
Currently, there are 2 ongoing clinical trials investigating new treatments for neutropenia, a condition characterized by abnormally low levels of neutrophils—white blood cells essential for fighting infections. These trials are testing medications designed to increase neutrophil counts and reduce the frequency of infections in patients with chronic forms of this condition.
Clinical trial locations
- Belgium
- Czechia
- France
- Germany
- Greece
- Hungary
- Ireland
- Italy
- Poland
- Portugal
- Romania
- Spain
Study on Mavorixafor for Patients with Chronic Neutropenia Experiencing Recurrent or Serious Infections
This international trial is evaluating mavorixafor, an oral medication taken as capsules, for people with chronic neutropenia who experience frequent or serious infections. The study includes patients with both congenital (present from birth) and acquired forms of the condition.
Main inclusion criteria: Participants must be at least 12 years old and weigh at least 15 kilograms. They must have chronic neutropenia lasting at least 6 months, with confirmed low neutrophil counts (below 1500 cells per microliter) that are not caused by medications, infections, or cancer. Participants must have experienced at least two serious infections in the past year requiring antibiotics or healthcare visits. A bone marrow test must show no signs of blood cancer. If participants are already receiving G-CSF treatment or other therapies, they must maintain stable doses for at least 4 weeks before and during the study. Both men and women must use effective birth control methods according to local regulations.
Main exclusion criteria: Patients currently receiving chronic G-CSF treatment are excluded from this study. Additionally, individuals who do not meet the age requirements or who belong to vulnerable populations requiring special protection may not be eligible.
Study focus: The trial aims to evaluate how effective mavorixafor is in reducing infection rates and improving neutrophil levels in the blood. Participants will be randomly assigned to receive either mavorixafor or a placebo over a 12-month treatment period. Regular check-ups will monitor health status, including blood tests to assess neutrophil counts and infection frequency, as well as questionnaires evaluating fatigue and oral ulcers.
Investigational drug: Mavorixafor works at the molecular level by blocking the CXCR4 receptor, which plays a role in neutrophil movement and function. This medication represents a different approach to treating chronic neutropenia compared to traditional therapies.
Study on Pegfilgrastim for Patients with Severe Chronic Neutropenia
This Italian trial is studying pegfilgrastim (marketed as Neulasta), a long-acting medication that helps the body produce more neutrophils. The study focuses on patients with severe chronic neutropenia who are already receiving daily G-CSF treatment and anti-infective therapy.
Main inclusion criteria: Patients of any age and either sex can participate if they have congenital or acquired chronic neutropenia. Eligibility requires an absolute neutrophil count of less than 500 per cubic millimeter, confirmed by three consecutive tests conducted at least one week apart over a minimum three-month period. Participants must already be receiving daily G-CSF treatment.
Main exclusion criteria: Patients not currently being treated with G-CSF or anti-infective therapy are excluded. Those who do not have severe chronic neutropenia or who fall outside the specified age range for the study are also ineligible.
Study focus: The primary goal is to evaluate whether pegfilgrastim can maintain neutrophil levels above 1000 per cubic millimeter from the second to the sixth month of treatment. The study will monitor the number, type, and duration of infections, antibiotic usage, and hospitalizations during this period. Pegfilgrastim is administered as a 6 mg subcutaneous injection (under the skin) according to the study protocol.
Investigational drugs: In addition to pegfilgrastim, participants continue receiving G-CSF (a medication that stimulates bone marrow to produce neutrophils) and anti-infective therapy (medications to prevent or treat infections). Pegfilgrastim is a granulocyte colony-stimulating factor that provides longer-lasting effects compared to standard daily G-CSF treatments. The trial is expected to continue until 2030, providing valuable long-term data on this treatment approach.
Summary
These two ongoing trials represent different therapeutic approaches to managing chronic neutropenia. The mavorixafor study is being conducted across 12 European countries, including Portugal, Romania, Hungary, Spain, Czechia, Greece, Italy, Germany, France, Belgium, Poland, and Ireland, making it a large international effort. In contrast, the pegfilgrastim study is currently limited to Italy.
The trials target different patient populations: the mavorixafor study focuses on patients with chronic neutropenia who may or may not be receiving G-CSF, while the pegfilgrastim trial specifically enrolls patients already on daily G-CSF treatment. Mavorixafor represents a novel approach by blocking the CXCR4 receptor, whereas pegfilgrastim is a well-established colony-stimulating factor medication. Both trials share the common goal of reducing infection rates and maintaining adequate neutrophil levels to improve quality of life for patients living with these challenging conditions.
