Active Cancer NIHR-supported project Digestion, Kidneys & Other Organs

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Participants with Congenital or Acquired Primary Autoimmune or Idiopathic Chronic Neutropenic Disorders

Funder NIHR (part of DHSC)
Recipient organisation NIHR Great Ormond Street Hospital Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Aug 2026

In plain English

AI plain-English summary

Every day, thousands of people with chronic neutropenia inject themselves with a drug to stave off life-threatening infections—but for many, the injections fail, and the infections keep coming. This trial tests whether a daily pill called mavorixafor can do the same job without a needle. Chronic neutropenia leaves patients with dangerously low levels of infection-fighting neutrophils for months or years. The standard treatment, G-CSF, requires regular injections and does not work for everyone. Mavorixafor, taken orally as a capsule, aims to raise neutrophil counts by a different biological mechanism. The study will enrol patients aged 12 and older who have had at least two infections in the past year despite existing therapy. Half will receive mavorixafor plus their usual care; half will receive a placebo plus usual care. Participants take the pill once daily for about a year, attend clinic visits, and keep a symptom diary. If mavorixafor proves effective, it could offer a needle-free alternative for patients who do not respond to G-CSF, reducing infection rates and improving quality of life without daily injections. For the roughly one in three patients whose neutropenia does not respond adequately to current treatment, this would represent a practical shift in daily disease management.

View original technical description
Chronic Neutropenia (CN) is a condition, defined by a low level of neutrophils in the blood for more than three months. The most common clinical consequences of Chronic Neutropenia are infections. The number of infections could be decreased by increasing the neutrophil count in blood by receiving an already approved treatment: Human recombinant granulocyte-colony stimulating factor (G-CSF). G-CSF might not be working for every patient and many still suffer with recurring infections. Also, G-CSF is administered by injection. The study drug - Mavoxifavor is a medication that could also increase neutrophil count and thus reduce the infections in humans with CN and it is taken by mouth in form of capsules.To take part in this study, participants must be older than 12 years, have been diagnosed with Chronic Neutropenia and having recurring infections – at least two in the last 12 months. Participants will be receiving their usual therapy for their Chronic Neutropenia together with the study treatment. Participants will be assigned in 1:1 ratio to receive either study drug or a placebo (a pill that does not contain any medication). Participants will take the tablets once a day for about a year. The participants will be required to attend study visits at the site and also to document in a diary at home information about their treatment and how they feel.

Researchers

Philip Ancliff (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

A prospective randomized, double-blind, placebo-controlled, multi-center phase IIb study to evaluate the efficacy and safety of mocravimod as an adjunctive and maintenance treatment in adult acute myeloid leukemia (AML) patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT)
A prospective randomized, double-blind, placebo-controlled, multi-center phase III study to evaluate the efficacy and safety of mocravimod as an adjunctive and maintenance treatment in adult acute myeloid leukemia (AML) patients undergoing allogeneic hematopoietic cell transplantation (HCT)
Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination with Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease (INCA34176-254).
A Phase 3 Study of Axatilimab (INCA034176) and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children with Hypochondroplasia (Study 111-303)

Plain English summaries and category classifications on this site are generated by AI and may not perfectly reflect the original research.