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Mavorixafor

Clinical trials are investigating Mavorixafor in people with WHIM syndrome and chronic neutropenic disorders. These studies look at whether it can improve blood cell counts, reduce infections, and stay safe over time. The trials include adults and other eligible participants in Phase 3 studies.

Table of contents

Clinical trials overview

These studies are testing Mavorixafor in people with blood disorders that cause low neutrophil counts and repeated infections.[1][2] The trial data show two Phase 3 interventional studies, one completed and one authorised.[1][2]

In clinical research, interventional means the study gives a treatment and then measures what happens.[1][2] Both studies compare Mavorixafor with placebo in at least part of the trial, which helps researchers see whether the study drug performs better than no active drug.[1][2]

WHIM syndrome study

NCT03995108 studied people with WHIM Syndrome, a rare condition marked by warts, low antibody levels, infections, and myelokathexis.[1] The study was completed and enrolled 14 participants.[1]

This trial had a randomized placebo-controlled period and an open-label period.[1] Randomized means participants are assigned by chance, and placebo-controlled means some people received a look-alike treatment without active drug for comparison.[1] Open-label means both the researchers and participants know the treatment being given.[1]

The main goal was to show that Mavorixafor could raise circulating neutrophils compared with placebo and reach a clinically meaningful threshold.[1] A key outcome was the time above threshold-absolute neutrophil count, or TAT-ANC, meaning how long the neutrophil count stayed at or above 500 cells/μL over 24 hours.[1]

The open-label part focused on long-term safety and tolerability.[1] Safety was checked through adverse events, laboratory tests, vital signs, ECGs, physical exams, and eye exams.[1]

Chronic neutropenia study

NCT06056297 is studying Mavorixafor in participants with congenital and acquired primary autoimmune and idiopathic chronic neutropenic disorders who have recurrent and/or serious infections.[2] The trial is authorised, is Phase 3, and plans to enroll 214 participants.[2]

This study also includes a placebo capsule comparison.[2] The trial is looking at people with chronic neutropenia who are not receiving chronic G-CSF treatment, as well as the overall population regardless of background therapy.[2] G-CSF is a treatment used to help the body make more neutrophils.[2]

The main outcome is the annualized infection rate based on infections reviewed by a BIAC, which is a committee that checks and confirms infection events in the study.[2] The study also measures ANC responders, meaning participants who meet a target neutrophil response in at least 3 of 6 planned visits during the 52-week treatment period.[2]

For some participants, a positive ANC response means an ANC of at least 1500 cells/μL.[2] For participants whose starting ANC is below 500 cells/μL, a response can also mean at least a 2-fold increase from baseline, which means the count doubles from the starting level.[2]

What the trials measure

Across these studies, the main focus is on neutrophil counts, infections, and safety.[1][2] Neutrophils are a type of white blood cell that help fight infection, so low counts can leave people at higher risk.[1][2]

  • Neutrophil improvement: The WHIM syndrome study measured how long ANC stayed above a set threshold, while the neutropenia study measured how many participants reached a defined ANC response.[1][2]

  • Infection control: The chronic neutropenia study tracked the annualized infection rate, which is the number of infections over a year adjusted for study time.[2]

  • Safety and tolerability: The WHIM syndrome study followed adverse events, lab tests, vital signs, ECGs, and physical and eye exams to see how well people handled treatment.[1]

Who may participate

The WHIM syndrome study included participants with WHIM syndrome.[1] The chronic neutropenia study includes participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are having recurrent and/or serious infections.[2]

These studies are not for the general public; they are for people who meet the trial rules, sometimes called eligibility criteria.[1][2] The available data also show that one study focused on people not receiving chronic G-CSF treatment.[2]

Study design and phases

Both trials are Phase 3 studies, which usually means the treatment has already been studied in earlier research and is now being tested in larger groups.[1][2] Phase 3 trials are important because they help show whether a treatment works in real patient groups and how safe it is over time.[1][2]

The WHIM syndrome study used a randomized placebo-controlled period followed by an open-label period.[1] The chronic neutropenia study is also interventional and uses placebo capsules for comparison.[2] Together, these designs help researchers compare outcomes fairly and collect longer-term data on treatment response.[1][2]

Trial ID Phase Condition studied Status Enrollment
NCT03995108 Phase 3 WHIM Syndrome Completed 14
NCT06056297 Phase 3 Neutropenias Authorised 214

FAQ

  • What conditions are being studied in Mavorixafor trials? The trials are studying WHIM syndrome and congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These are conditions where neutrophils, a type of white blood cell, are too low and infections can happen more often.
  • Who can take part in these studies? People who meet the study rules can join, such as participants with WHIM syndrome or chronic neutropenia and, in one study, those with recurrent and/or serious infections. Some trial groups also focus on people who are not receiving chronic G-CSF treatment.
  • What phase are the Mavorixafor trials? Both trials listed are Phase 3 studies. Phase 3 means the treatment is being tested in larger groups to learn more about how well it works and how safe it is.
  • What are the trials trying to measure? One study measures how long neutrophil counts stay above a set threshold and also checks safety and tolerability. The other study measures annual infection rate and how many participants reach a helpful neutrophil response.
  • Are the studies finished? One trial in WHIM syndrome is completed. The trial in chronic neutropenic disorders is authorised, which means it has been approved to move forward.

Ongoing Clinical Trials on Mavorixafor

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

    Recruiting

    1 1
    Investigated drugs:
    Belgium Czechia France Germany Greece Hungary +6

  • Study on Mavorixafor for Treating WHIM Syndrome in Patients

    Not recruiting

    1 1
    Investigated drugs:
    Denmark France Italy The Netherlands Spain

Glossary

  • WHIM syndrome: A rare condition named for its main features: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. It can cause repeated infections and low white blood cell counts.
  • Neutropenia: A low number of neutrophils, which are white blood cells that help fight infection.
  • Congenital: Present from birth.
  • Acquired: Developed later in life, not present from birth.
  • Primary autoimmune: A condition where the immune system attacks the body’s own cells and causes the problem.
  • Idiopathic: The cause is not known.
  • Recurrent infections: Infections that keep coming back.
  • Serious infections: Infections that can be severe and may need urgent medical care.
  • Phase 3: A late stage of clinical research that studies how well a treatment works and how safe it is in larger groups.
  • Placebo: A look-alike treatment with no active study drug, used for comparison.
  • ANC: Absolute neutrophil count, the number of neutrophils in the blood.
  • Safety and tolerability: How well people handle a treatment and what side effects or problems may happen during the study.

References