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A Phase 3, randomized, double-blind, placebo-controlled, multicenter study of mavorixafor in participants with congenital and acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections.

Status
Recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-508482-32-00
Acronym
X4P-001-110
Enrollment
86
Registered
2024-06-14
Start date
2024-07-22
Completion date
Unknown
Last updated
2025-10-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neutropenias

Brief summary

Annualized infection rate based on infections adjudicated by a BIAC during the 52-week treatment period, 2a: Proportion of ANC responders. An ANC responder is a participant meeting the definition of a positive ANC response for at least 3 out of 6 visits [Weeks 4, 8, 13, 26, 39, and 52] during the 52-week treatment period, where a positive ANC response is defined as: (please see next "ID 3"), 2b: (continued from ID 2) • ANC ≥ 1500 cells/µL, with the exception of participants with baseline ANC < 500 cells/µL • ≥ 2-fold increase in ANC from baseline, for participants with baseline ANC < 500 cells/µL

Detailed description

Infection severity based on CTCAE grading, adjudicated by a BIAC during the 52-week treatment period, Infection duration based on duration of infections adjudicated by a BIAC during the 52-week treatment period, Antibiotic use due to infection, characterized by the frequency of antibiotic use during the 52-week treatment period, Oral ulcers, as assessed by presence or absence of ulcers, during the 52-week treatment period, Change from baseline to Week 52/Day 365 in PROMIS SF Fatigue Questionnaire total score

Interventions

DRUGMavorixafor Placebo Capsules

Sponsors

X4 Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to No maximum

Design outcomes

Primary

MeasureTime frame
Annualized infection rate based on infections adjudicated by a BIAC during the 52-week treatment period, 2a: Proportion of ANC responders. An ANC responder is a participant meeting the definition of a positive ANC response for at least 3 out of 6 visits [Weeks 4, 8, 13, 26, 39, and 52] during the 52-week treatment period, where a positive ANC response is defined as: (please see next "ID 3"), 2b: (continued from ID 2) • ANC ≥ 1500 cells/µL, with the exception of participants with baseline ANC < 500 cells/µL • ≥ 2-fold increase in ANC from baseline, for participants with baseline ANC < 500 cells/µL

Secondary

MeasureTime frame
Infection severity based on CTCAE grading, adjudicated by a BIAC during the 52-week treatment period, Infection duration based on duration of infections adjudicated by a BIAC during the 52-week treatment period, Antibiotic use due to infection, characterized by the frequency of antibiotic use during the 52-week treatment period, Oral ulcers, as assessed by presence or absence of ulcers, during the 52-week treatment period, Change from baseline to Week 52/Day 365 in PROMIS SF Fatigue Questionnaire total score

Countries

Belgium, Czechia, France, Germany, Greece, Hungary, Ireland, Italy, Poland, Portugal, Romania, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 5, 2026