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 States176 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 range12 Years
SexALL
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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
✕. Hypersplenism
✕. Infection
✕. Malignancy
✕. Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease
✕. Nutritional deficiency, for example, vitamin B12, folic acid, copper, caloric malnutrition
✕. Drug-induced cause, for example, chemotherapy, clozapine, antiretrovirals, antibiotics, monoclonal antibodies.
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