A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

ConditionNeutropenia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age12+
SponsorX4 Pharmaceuticals

About this trial

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

Eligibility criteria

Qualifiers

Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy.

Isolated with a permanent (non-cyclic) presentation, for example, elastase, neutrophil expressed (ELANE), colony stimulating factor 3 receptor (CSF3R), C-X-C chemokine receptor 2 (CXCR2), Wiskott-Aldrich syndrome (WAS)

Associated with extra-hematologic manifestations, for example, Barth syndrome, Cohen syndrome, glucose-6-phosphatase catalytic subunit 3 (G6PC3), Kostmann disease

Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)

Disqualifiers

Hypersplenism

Infection

Malignancy

Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease

Trial design

Treatments tested in this trial

  • Mavorixafor
  • Placebo

Treatment groups

176 Participants
are divided into 2 treatment groups

Sponsors and collaborators