This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people living with chronic neutropenia (including congenital, idiopathic, and cyclic). In Part 2 (Phase 2), the safety and tolerability of chronic dosing of mavorixafor will be evaluated in a larger participant population and the impact of 6-month chronic dosing of mavorixafor on participant neutropenia.
Part 1: Participants will receive one oral dose of mavorixafor and be monitored for 8 hours to see if neutrophil cell counts increase. Participants screened after implementation of Protocol Version 8.0, do not need to enter Part 1 and can start with the Part 2 Screening visit (Day -28 to Day -1). Part 2: For participants enrolled in the study before implementation of Protocol Version 8.0, Part 2 will include those who completed Part 1 and exhibited a response to treatment. Eligible participants from Part 1 enrolled in the study before implementation of Protocol Version 8.0, can directly roll-over to the Baseline (Day -3 to Day -1) visit in Part 2. Participants will receive once daily oral dosing of mavorixafor for 6 months and be monitored throughout to see if neutrophil cell counts increase. Study visits can be conducted at-home or at one of many study clinic locations, depending on the participant's preference.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
32
Mavorixafor capsules will be administered per dose and schedule specified in the arm.
USF Health Department of Pediatrics
St. Petersburg, Florida, United States
University of Iowa Hospital and Clinics
Iowa City, Iowa, United States
University of Michigan
Ann Arbor, Michigan, United States
Washington University School of Medicine
St Louis, Missouri, United States
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After a Single Dose of Mavorixafor
Time frame: Baseline through Day 1 and 7 days follow-up
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) After Multiple Doses of Mavorixafor
Time frame: Baseline through Month 6 and 30 days follow-up
Change From Baseline in Absolute Neutrophil Count (ANC) to 8 hours Post-dose On Day 1
Time frame: Baseline, 8 hours Post-dose On Day 1
Change From Baseline in ANC to Month 6
Time frame: Baseline, Month 6
Serum Concentration of Mavorixafor in Relation to ANC and Area Under the Curve (AUC) for ANC (AUCANC)
Time frame: 0 (pre-dose), 60 minutes and 2, 3, 4, 6, and 8 hours post-dose on Day 1
Serum Concentrations of Mavorixafor
Time frame: 0 (pre-dose) up to Month 6
Change from Baseline in Absolute Lymphocyte Count (ALC)
Time frame: Baseline, Month 6
Change from Baseline in Total White Blood Cells (WBC)
Time frame: Baseline, Month 6
Change from Baseline in Absolute Monocyte (AMC)
Time frame: Baseline, Month 6
AUC of ANC (AUCANC)
Time frame: Baseline up to Month 6
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas, Southwestern
Dallas, Texas, United States
University of Washington
Seattle, Washington, United States
AUC of ALC (AUCALC)
Time frame: Baseline up to Month 6
AUC of AMC (AUCAMC)
Time frame: Baseline up to Month 6
AUC of WBC (AUCWBC)
Time frame: Baseline up to Month 6