Last reviewed · How we verify
NCT06056297
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
Phase 3 trial testing Mavorixafor in Neutropenia in 176 participants. Currently enrolling.
1 September 2027
Quick facts
| Lead sponsor | X4 Pharmaceuticals |
|---|---|
| Phase | Phase 3 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 176 |
| Start date | 6 June 2024 |
| Primary completion | 1 September 2027 |
| Estimated completion | 1 November 2027 |
| Sites | 114 locations across United States, Argentina, Australia, Belgium, Canada, Colombia, Czechia, France |
Drugs / interventions tested
- Mavorixafor (MAVORIXAFOR) — full drug profile →
- Placebo
Conditions studied
- Neutropenia — all drugs for Neutropenia →
Sponsor
X4 Pharmaceuticals — full company profile →
Who can join
12 and older, any sex, with Neutropenia. Patients with the condition only — healthy volunteers not accepted.
What's being measured
Primary outcomes are the specific endpoints the trial is designed to prove or disprove.
-
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 -
Co-primary Endpoint: Number of Participants Meeting the Definition of a Positive Absolute Neutrophil Count (ANC) Response
Time frame: Up to 52 weeks
Positive ANC response: Increase of ANC \>500 cells/microliter (µL) from baseline.
Sponsor's own description
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.
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
-
Neutrophil diversity and function in health and disease.
Zhang F, Xia Y, Su J, Quan F, et al · · 2024 · cited 109× · PMID 39638788 · DOI 10.1038/s41392-024-02049-y -
WHIMS2 new homozygous variant
FERNANDEZ SN, H M, R M. · · 2026 · DOI 10.21203/rs.3.rs-9779576/v1 -
CXCR4 antagonism corrects neutrophil abnormalities and reduces pneumonia severity in a pharmacological mouse model of CXCR2 loss-of-function-mediated neutropenia.
Nguyen CH, Zmajkovicova K, Sekirnik A, Taplin S, et al · · 2025 · PMID 41451234 · DOI 10.3389/fimmu.2025.1658987
Verify or expand the search:
- PubMed search for NCT06056297
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Mavorixafor
Trials testing the same drug.
- NCT06914869 — Drug-Drug Interaction Potential of Mavorixafor · Phase 1 · completed
- NCT04154488 — A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders · Phase 1, PHASE2 · completed
- NCT04274738 — A Study of Mavorixafor in Combination With Ibrutinib in Participants With Waldenstrom's Macroglobulinemia (WM) Whose Tum · Phase 1 · completed
Other recruiting trials for Neutropenia
Currently open trials in the same condition.
- NCT07434063 — Real-world Study on the Prevention of Neutropenia After Tumor Treatment With Mecapegfilgrastim Injection · recruiting
- NCT07066085 — Serial Blood Count Study · EARLY_PHASE1 · recruiting
- NCT06537726 — Breath Analysis for the Detection of Invasive Fungal Infections · recruiting
- NCT06145321 — Continuous Versus Bolus Administration of G-CSF in Children With Cancer · Phase 4 · active not recruiting
- NCT06422533 — Ceftolozane/Tazobactam vs. Piperacillin/Tazobactam for the Treatment of Bacteremia in Hemato-oncological Patients · NA · recruiting
Other X4 Pharmaceuticals trials
Trials by the same sponsor.
- NCT06914869 — Drug-Drug Interaction Potential of Mavorixafor · Phase 1 · completed
- NCT04154488 — A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders · Phase 1, PHASE2 · completed
- NCT04274738 — A Study of Mavorixafor in Combination With Ibrutinib in Participants With Waldenstrom's Macroglobulinemia (WM) Whose Tum · Phase 1 · completed
- NCT02923531 — Addition of X4P-001 to Nivolumab Treatment in Participants With Renal Cell Carcinoma · Phase 1, PHASE2 · terminated
- NCT02823405 — X4P-001 and Pembrolizumab in Patients With Advanced Melanoma · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06056297 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by X4 Pharmaceuticals
- Last refreshed: 4 June 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06056297.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing