Testing Mavorixafor against a placebo for neutropenia

Inclusion

• +Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
• +Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visitScreeningThe checks done before joining, to see whether a study fits.Read more → that is not attributable to medications, active or recent infections or malignancy.
• +Congenital Neutropenia, including but not limited to these classifications:
• +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
• +Shwachman-Diamond syndrome
• +Acquired Primary Neutropenia
• +Chronic idiopathic neutropenia
• +Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more → can be clarified and approved upon discussion with study Medical Monitor.
• +Have an ANC \<1000 cells/µL during screening (single ANC value from hematology) and confirmed trough mean ANC (mean value of multiple ANC measurements over 6 hours) at baseline visitBaselineYour starting measurements, taken before treatment begins.Read more →, with no clinical evidence of systemic infection.
• +Prior history of recurrent and/or serious infections during the 12 months preceding the screening visit (that is, suffering sequelae of chronic neutropenia), as defined by having at least 2 infections in the last 12 months that meet the following criteria:
• +Infection requiring the use of antibiotics (intravenous \[IV\]/oral); OR
• +Infection requiring a visit to healthcare facility (including but not limited to emergency room visit, urgent care facility, primary care physician's office, or in-patient hospitalization);
• +AND for all potential participants:
• +Infections considered by the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → to be likely related to the potential participant's CN disorder.
• +Participants who are on G-CSF or other active background therapy must have been receiving these therapies during the previous 12 months while continuing to suffer from infections, be on a stable dose and dosing schedule for ≥4 weeks prior to screening visit and remain on this dose and dosing schedule throughout the study (unless ANC \>10,000 cells/µL for ≥4 weeks).
• +Participants must be willing to keep their G-CSF or other background therapy doses/regimens stable (other than for safety reasons) for the duration of the study.