NCT07902830

Not Yet RecruitingPhase 2

A Study to Investigate the Safety and Tolerability of Granul...

NCT07902830|Not Yet RecruitingPhase 2DMCFDA Drug

A Study to Investigate the Safety and Tolerability of Granulocyte-colony Stimulating Factor (G-CSF) Dosage Reduction in Mavorixafor-treated Participants With Acquired Primary Autoimmune and Idiopathic Chronic Neutropenia Disorders

A Phase 2b, Single-arm, Open-label, Multicenter Study to Evaluate the Safety and Tolerability of G-CSF Dosage Reduction With Mavorixafor in Participants With Acquired Primary Autoimmune and Idiopathic Chronic Neutropenia Disorders

X4 Pharmaceuticals ClinicalTrials.gov

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Other study identifiers

Sponsor study identifier

X4P-001-112

Study Type

interventional

Estimated enrollment

40

Locations

0 countries

Sites

N/A

Timeline

Study Start

December 2026

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Primary Completion

January 30, 2029

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Study Completion

January 30, 2029

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of G-CSF dosage reduction with mavorixafor in participants with autoimmune neutropenia (AIN) and chronic idiopathic neutropenia (CIN) disorders as assessed by the ability to safely reduce G-CSF dosage with mavorixafor treatment while maintaining clinically acceptable absolute neutrophil count (ANC) levels.

Registry Overview

Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.

Recruitment status: not yet recruiting
Estimated enrollment: 40 participants
Study completion date: January 30, 2029
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Listed locations: 0 countries · 0 sites

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.

First Submitted
October 20, 2026
Actual (reported)
First Posted
October 29, 2026
Actual (reported)
Study Start
December 2026
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Primary Completion
January 30, 2029
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
January 30, 2029
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.

Last Updated: October 29, 2026
Status Verified: October 2026
Last Update Submitted: October 20, 2026

Conditions

Acquired Primary Autoimmune and Idiopathic Chronic Neutropenia Disorders Chronic Neutropenia Autoimmune Neutropenia Chronic Idiopathic Neutropenia

Keywords

G-CSFChronic neutropeniaMavorixaforX4P-001Autoimmune neutropeniaChronic idiopathic neutropenia

Outcome Measures

Primary Outcomes (4)

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Up to Week 56
Percent Change in Average Daily G-CSF Dosage
Week 12 through Week 28 or 32
Percentage of Participants Achieving G-CSF Cessation
Week 28 or 32
Percentage of Participants Achieving G-CSF Dosage Reduction ≥75%
Week 28 or 32

Study Arms (1)

Mavorixafor

EXPERIMENTAL

Participants will receive mavorixafor once daily orally for 52 weeks (starting from Week 4 through 56).

Drug: Mavorixafor

Interventions

MavorixaforDrug

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

Also known as: X4P-001

Mavorixafor

Eligibility Criteria

Age12 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Diagnosis of acquired primary chronic neutropenia (CN) disorder ≥6 months prior to the screening visit (Week -2 up to Week 1) that is not attributable to medications, active or recent infections, or malignancy.
On G-CSF for ≥6 months; stable dose prior to start of mavorixafor dosing.

Sponsors & Collaborators

X4 Pharmaceuticals
Industrylead

MeSH Terms

Interventions

mavorixafor

Study Officials

Senior Vice President, Clinical Development
X4 Pharmaceuticals
STUDY DIRECTOR

Central Study Contacts

Patient Affairs and Advocacy

CONTACT

857-529-5779 clinicaltrialinfo@x4pharma.com

Study Design

Study Type: interventional
Phase: phase 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

October 20, 2026

First Posted

October 29, 2026

Study Start

December 2026

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Primary Completion

January 30, 2029

Estimated

Phase 1Completed

NCT03995108

Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

Phase 3Completed

NCT04274738

A Study of Mavorixafor in Combination With Ibrutinib in Participants With Waldenstrom's Macroglobulinemia (WM) Whose Tumors Express Mutations in MYD88 and CXCR4

Phase 1Completed

NCT06914869

Drug-Drug Interaction Potential of Mavorixafor

Phase 1Completed

NCT02667886

Trial of X4P-001 in Participants With Advanced Renal Cell Carcinoma

Phase 1/2Completed

NCT05931718

Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia

Recruiting

Secondary Outcomes (9)

Percentage of Participants Achieving G-CSF Dosage Reduction by ≥ 25%/≥ 50%
Week 12 through Week 28 or 32
Time to G-CSF Cessation
Week 4 through Week 28 or 32
Time to G-CSF Dosage Reduction of ≥25% or ≥50% or ≥75
Week 4 through Week 28 or 32
Change From Week 12 in Average Daily G-CSF Dosage at Week 28 or 32
Week 12, Week 28 or 32
Change From Week 12 in Average Daily G-CSF Frequency at Week 28 or 32
Week 12, Week 28 or 32
+4 more secondary outcomes

Most recent bone marrow examination, within 3 years, shows no evidence of malignancy/high risk of malignant transformation.

You may not qualify if:

Participant is incapacitated and unable to comply with protocol-specific requirements.
A diagnosis of any of the following:
Aplastic anemia
Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome
CN other than CIN or AIN
A medical or personal condition that may potentially compromise the safety of the participant, may preclude the participant's successful completion of the clinical study, or could, in the opinion of the Investigator or the Medical Monitor, interfere with the objectives of the study.
Participant is currently taking or has taken an investigational drug \<30 days prior to the screening visit, or 5 half-lives, whichever is longer.
Participant is pregnant or breastfeeding.

Contact the study team to confirm eligibility.

Planned date reported by the registry; this milestone is not confirmed as achieved.

Study Completion

January 30, 2029

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Last Updated

October 29, 2026

Record last verified: 2026-10

Data Sharing

IPD Sharing: Will not share

Source record

Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.

Source: ClinicalTrials.gov.

JSON downloads include source attribution and retrieval details alongside the unchanged original record or the separately normalized values.

Registry identifier: NCT07902830
Data provider: ClinicalTrials.gov
Source updated: 2026-10-29
Normalization version: ctgov-2

Open registry record

Source retrieved: 2026-10-30 23:02:53 UTC

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (4)

Study Arms (1)

Mavorixafor

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

MeSH Terms

Interventions

Study Officials

Central Study Contacts

Study Design

Study Record Dates

Secondary Outcomes (9)

You may not qualify if:

Data Sharing

Source record