Study on Mavorixafor for Patients with Chronic Neutropenia Experiencing Recurrent or Serious Infections

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What is this study about?

This clinical trial is focused on studying a group of conditions known as neutropenias. Neutropenias are disorders where the body has a lower than normal number of a type of white blood cell called neutrophils, which are important for fighting infections. The study is particularly interested in people with chronic forms of these conditions, which can be either congenital (present from birth) or acquired later in life. These conditions can lead to frequent and serious infections.

The treatment being tested in this study is a medication called mavorixafor, which is taken as a capsule by mouth. The purpose of the study is to evaluate how effective mavorixafor is in reducing the rate of infections and improving the levels of neutrophils in the blood. Participants in the study will be randomly assigned to receive either mavorixafor or a placebo, which looks like the medication but does not contain the active ingredient. The study will last for about a year, during which participants will have regular check-ups to monitor their health and the effects of the treatment.

Throughout the study, participants will be closely monitored for any changes in their condition, including the frequency and severity of infections. The study aims to provide valuable information on the safety and tolerability of mavorixafor for people with chronic neutropenia. This research could potentially lead to new treatment options for managing these challenging conditions.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age, weight, and medical history related to chronic neutropenia.

A bone marrow biopsy or aspirate is required to ensure no evidence of blood-related cancer.

2 screening and baseline assessment

During the screening phase, a blood test is conducted to measure the absolute neutrophil count (ANC).

The baseline visit occurs at least two weeks before the first dose of the study medication, ensuring the ANC is below 1500 cells/µL without signs of infection.

3 medication administration

Participants receive either mavorixafor or a placebo in the form of hard capsules taken orally.

The dosage and frequency are determined by the study protocol, and participants must adhere to the prescribed schedule.

4 treatment period

The treatment period lasts for 12 months, during which the effectiveness of mavorixafor is evaluated based on infection rates and ANC levels.

Participants continue any existing therapies for chronic neutropenia, maintaining stable doses unless safety concerns arise.

5 monitoring and assessments

Regular visits are scheduled to monitor health status, including blood tests to assess ANC and check for infections.

Additional assessments may include questionnaires to evaluate fatigue and the presence of oral ulcers.

6 end of study

At the end of the 12-month period, a final assessment is conducted to evaluate the overall health and response to the treatment.

Participants may be required to continue using contraception for a specified period after the last dose.

Who Can Join the Study?

  • Participants must be at least 12 years old when they sign the consent form.
  • A test called a bone marrow biopsy or aspirate must show no signs of blood cancer.
  • Participants must weigh at least 15 kilograms.
  • Men and women must use birth control methods that follow local rules. Abstinence is not considered a reliable method.
  • Participants must be able to give consent, or have a parent or guardian who can do so.
  • Participants must have a type of low white blood cell count called chronic neutropenia for at least 6 months. This condition should not be caused by medications, infections, or cancer.
  • Participants must have a confirmed low level of a type of white blood cell called ANC (absolute neutrophil count) below 1500 cells per microliter, with no signs of infection.
  • Participants must have had at least two serious infections in the past year that required antibiotics or a visit to a healthcare facility.
  • If participants are on a treatment called G-CSF or other therapies, they must have been on a stable dose for at least 4 weeks before the study and keep it stable during the study.
  • Participants must avoid certain drugs that affect how the body processes medications, starting 14 days before the study.
  • Participants must avoid grapefruit and Seville orange products, as they can affect medication processing.

Who Cannot Join the Study?

  • Patients who are currently receiving chronic G-CSF treatment cannot participate. G-CSF is a medication that helps the body make more white blood cells.
  • Patients with chronic neutropenia who are not receiving chronic G-CSF treatment are eligible, but those who are receiving it are excluded.
  • Patients who do not meet the age requirements for the study cannot participate. The study has specific age ranges that are eligible.
  • Patients who are part of a vulnerable population may not be eligible. A vulnerable population includes groups that might need special protection, like children or the elderly.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
University General Hospital Of Thessaloniki Ahepa Thessaloniki Greece
Unidade Local De Saúde De Santa Maria, E.P.E. Lisbon Portugal
Virgen del Rocío University Hospital Sevilla Spain
Fakultni nemocnice Motol a Homolka Prague Czechia
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l. Meldola Italy
Fakultni Nemocnice Brno Brno Czechia
Semmelweis University Budapest Hungary
Hospital Universitario Fundacion Jimenez Diaz Madrid Spain
Centro Hospitalar E Universitario De Coimbra E.P.E. Coimbra Portugal
Azienda Ospedaliera Universitaria Citta’ Della Salute E Della Scienza Di Torino Turin Italy
Centre Hospitalier Universitaire De Rennes Rennes France
Oncopole Claudius Regaud Toulouse France
Technische Universitaet Dresden Dresden Germany
Katholieke Universiteit te Leuven Leuven Belgium
Hopital Necker Enfants Malades Paris France
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Hospital Universitario De La Princesa Madrid Spain
Hospital Universitario Marques De Valdecilla Santander Spain
Hospital Del Mar Barcelona Spain
Hospital Universitario Y Politecnico La Fe Valencia Spain
Institut Catala D’oncologia L'hospitalet De Llobregat Spain
Universita Degli Studi Di Brescia Brescia Italy
Servei De Salut De Les Illes Balears Palma Spain
University General Hospital Of Heraklion Heraklion Greece
Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Azienda Ospedaliero Universitaria Ospedali Riuniti Umberto I G M Lancisi G Salesi Ancona Italy
Children’s Health Ireland Dublin Ireland
Assistance Publique Hopitaux De Paris Paris France
Centro Hospitalar Do Baixo Vouga E.P.E. (CHBV E.P.E.) Aveiro Portugal
IRCCS Istituto Giannina Gaslini Genoa Italy
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi Lodz Poland
Hospital Universitario Infanta Leonor Madrid Spain
El Hospital Universitario De Gran Canaria Dr. Negrin Las Palmas De Gran Canaria Spain
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA Rome Italy

Other Sites

Site Name City Country Status
Fundeni Clinical Institute Bucharest Romania
Ustav Hematologie A krevni Transfuze Prague Czechia
Uhloxiqlze Tijqvsbx Hpeawkjw Mmbfqqckiejb Szombathely Hungary
Svteitzr Cezjkf Cvrwoomaj Bqueohbau Bucharest Romania
Cdipyro Mkncjlg Cqmvtu Dlik Kmnfflx Budapest Hungary
2ei Arw Fqsmf Gppgfno Hwqalapr Athens Greece
Enrqjklyt Ieiliepxc Fkx Cfljwflxozalyd Doyricrr Aax Tlrkzjmxnd Targu Mures Romania
Oxxuzbsj Tjlbacprsscyt Gkzanhn Ctjqwc Om Pkmwln Scer Patras Greece

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
19.06.2024
Czechia Czechia
Recruiting
19.06.2024
France France
Recruiting
19.06.2024
Germany Germany
Recruiting
19.06.2024
Greece Greece
Recruiting
19.06.2024
Hungary Hungary
Recruiting
19.06.2024
Ireland Ireland
Not yet recruiting
19.06.2024
Italy Italy
Recruiting
19.06.2024
Poland Poland
Not yet recruiting
19.06.2024
Portugal Portugal
Recruiting
19.06.2024
Romania Romania
Recruiting
19.06.2024
Spain Spain
Recruiting
19.06.2024

Trial locations

Investigated Drugs:

Mavorixafor is a medication being studied for its ability to help people with certain types of chronic neutropenia, a condition where the body has a lower than normal number of neutrophils, a type of white blood cell important for fighting infections. This trial is looking at how well mavorixafor can reduce the rate of infections and improve the levels of neutrophils in the blood of participants who have chronic neutropenia and are not receiving ongoing treatment with G-CSF, a common therapy for this condition.

Neutropenias – Neutropenias are conditions characterized by an abnormally low count of neutrophils, a type of white blood cell essential for fighting off infections. This reduction in neutrophils can make individuals more susceptible to infections, as their immune system is compromised. Neutropenias can be chronic or acute, with chronic forms persisting over a longer period. The condition can arise from various causes, including bone marrow disorders, certain medications, or autoimmune diseases. Symptoms may include frequent infections, fever, and mouth ulcers. The severity of neutropenia can vary, influencing the risk and frequency of infections.

Trial ID:
2023-508482-32-00
Protocol code:
X4P-001-110
NCT ID:
NCT06056297
Trial Phase:
Therapeutic confirmatory (Phase III)

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