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.
All participants will continue their pre-study background therapy, defined as the participant's current treatment regimen. Options include, but are not limited to, granulocyte-colony stimulating factor (G-CSF), immunoglobulin replacement therapy, prophylactic antibiotics, or "watchful waiting".
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
176
Mavorixafor will be administered per schedule specified in the arm description.
Placebo will be administered per schedule specified in the arm description.
Phoenix Children's Hospital
Phoenix, Arizona, United States
RECRUITINGUC Irvine
Irvine, California, United States
RECRUITINGOrso Health
La Jolla, California, United States
RECRUITINGUniversity of California, Los Angeles-UCLA
Los Angeles, California, United States
Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period
Time frame: Up to 52 Weeks
Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response
Positive ANC response: Increase of ANC \>500 cells/microliter (µL) from baseline.
Time frame: Up to 52 weeks
Infection Severity Based on Common Terminology Criteria for Adverse Events (CTCAE) Adjudicated by a BIAC During the Treatment Period
Time frame: Up to 52 Weeks
Infection Duration Based on Duration of Infections Adjudicated by a BIAC During the Treatment Period in Those Participants who Developed Infections
Time frame: Up to 52 Weeks
Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period
Time frame: Up to 52 Weeks
Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period
Time frame: Up to 52 Weeks
Change From Baseline in Patient Reported Outcomes Measurement Information System Short Form (PROMIS SF) Fatigue Questionnaire Total Score
Time frame: Baseline, Week 52
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Orso Health
Torrance, California, United States
RECRUITINGUniversity of Southern Florida
St. Petersburg, Florida, United States
RECRUITINGChildren's Healthcare of Atlanta (CHOA)
Atlanta, Georgia, United States
RECRUITINGUniversity of Iowa
Iowa City, Iowa, United States
RECRUITINGUniversity of Michigan
Ann Arbor, Michigan, United States
RECRUITINGWashington University
St Louis, Missouri, United States
RECRUITING...and 95 more locations