A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
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.
• 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.
• Congenital Neutropenia, including but not limited to these classifications:
‣ Isolated with a permanent (non-cyclic) presentation, for example, ELANE, CSF3R, CXCR2, WAS
⁃ Associated with extra-hematological manifestations, for example, Barth syndrome, Cohen syndrome, G6PC3, Kostmann disease
⁃ Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
⁃ Shwachman-Diamond syndrome
• Acquired Primary Neutropenia
‣ Chronic idiopathic neutropenia
⁃ Primary autoimmune neutropenia. Other chronic neutropenia (CN) disorders that may be eligible for enrollment can be clarified and approved upon discussion with Study Medical Monitor and Sponsor.
• Have a confirmed trough ANC \<1500 cells/µL during the screening visit (single ANC measurement) and at baseline visit (mean ANC over 6 hours) held at least 2 weeks prior to Day 1 dosing, with no clinical evidence of 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 at least 1 of the following criteria:
• Infection requiring the use of antibiotics (intravenous \[IV\]/oral/topical)
• 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).
• 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.