This Phase 1b study will determine the safety and tolerability of mavorixafor in participants with severe chronic idiopathic neutropenia (CIN) and selected congenital neutropenia disorders. The anticipated enrollment is up to 45 participants.
Participants must meet the following criteria:
- Weigh ≥15 kg
- Sign the informed consent form (ICF) and be willing and able to comply with the protocol
- Participants with severe chronic idiopathic neutropenia (CIN):
- Have a history of ANC ≤500 cells/microliter (μL), lasting for more than 3 months at any time since diagnosis.
- Have been diagnosed with severe CIN more than 12 months ago that is not attributable to medications, infectious, genetic, inflammatory, autoimmune, or malignant causes.
- Be currently treated with a steady-state G-CSF regimen for greater than (>) 15 days before receiving the first dose of study drug.
- Have cytogenetics that do not suggest clonal evolution on the most recent bone marrow biopsy/aspirate, if performed.
- Have no history of associated thrombocytopenia (platelets < 75,000/uL) nor anemia <10.0 g/dL before G-CSF therapy initiation.
- Not have neutropenia secondary to another pathology, such as a systemic autoimmune disorder and in particular rheumatological or hematological.
- Participants with selected congenital neutropenia conditions including GSD1b (GSD1b; SLC37A4), G6PC3 deficiency (G6PC3), or GATA2 deficiency (GATA2):
- May be currently receiving steady-state G-CSF dosing or not have been on G-CSF for >15 days.
- Must have documentation of the mutational status before enrolling in the study.
Four sites have opened:
University of Michigan
Ann Arbor, Michigan, United States, 48109
Contact: Dr. Kelly Walkovich [email protected] 734-615-4368
University of Washington
Seattle, Washington, United States, 98195
Contact: Dr. David Dale [email protected] 206 543 3668
University of Iowa Hospital and Clinics
Iowa City, Iowa, United States, 52242
Contact: Dr. Anjali Sharathkumar [email protected]
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
Contact: Dr. David Wilson [email protected] 908 887-2278
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided. Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04154488
Please visit the clinical trial website for additional criteria, exclusions, and additional information.