A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopa… (NCT06056297) | Clinical Trial Compass
RecruitingPhase 3
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
United States, Argentina, Australia176 participantsStarted 2024-06-06
Plain-language summary
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.
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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
. Associated with metabolic disorders, for example, glycogen storage disease 1b (GSD1b)
. Shwachman-Diamond syndrome
. 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.
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 3 trial measuring whether mavorixafor actually reduces the rate of serious infections in people with chronic neutropenia, what does my doctor think the chances are that it could help my specific type — whether congenital, autoimmune, or idiopathic — compared to what I'm already doing to manage infections?
2The trial is tracking something called an Absolute Neutrophil Count response as a key measure of success — can my doctor explain what my current ANC levels look like and whether that makes me a realistic candidate worth discussing with the study team?
3Because this trial is still actively recruiting and results aren't in yet, how does my doctor weigh the unknowns of an experimental drug against standard treatments I might try first for managing my recurrent infections?
4This study requires participants to have recurrent or serious infections — can my doctor help me understand whether my infection history fits that pattern, and what the monitoring and visit schedule would look like if we decided to explore enrollment?
5If mavorixafor doesn't end up working well enough or causes side effects during the trial, what would my options be for stepping back to other neutropenia treatments, and would participating in this study affect my access to those alternatives?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Co-primary Endpoint: Annualized Infection Rate Based on Infections Adjudicated by Blinded Infection Adjudication Committee (BIAC) During the Treatment Period
Timeframe: Up to 52 Weeks
2
Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response