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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

ConditionNeutropenia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age12+
SponsorX4 Pharmaceuticals

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorder ≥6 months prior to the screening visit that is not attributable to medications, active or recent infections or malignancy.

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)

Disqualifiers

Hypersplenism

Infection

Malignancy

Autoimmune disease, for example, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, graft-versus-host disease, thyroid disease

Trial design

Design model

Parallel

Treatments tested in this trial

  • Mavorixafor

    Drug

    Mavorixafor will be administered per schedule specified in the arm description.

  • Placebo

    Drug

    Placebo will be administered per schedule specified in the arm description.

Treatment groups

176 Participants
are divided into 2 treatment groups
Group A: MavorixaforExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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
2

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

Secondary outcomes

1

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
2

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
3

Antibiotic Use Due to Infection, Characterized by the Frequency of Antibiotic Use During the Treatment Period

Time frame
Up to 52 Weeks
4

Oral Ulcers, as Assessed by Presence or Absence of Ulcers During the Treatment Period

Time frame
Up to 52 Weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

X4 Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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