NCT03005327

A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

A Phase 2, Open-Label, Multi-Center Trial of Mavorixafor in Patients With WHIM Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: X4 Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 study with an initial 24-week Treatment Period and an Extension Phase. The primary objectives of this Phase 2 study are to determine the safety, tolerability, and dose selection of mavorixafor in participants with WHIM syndrome. Participants may continue treatment in an Extension Phase, if regionally applicable, until mavorixafor becomes available via an alternative mechanism (for example, drug is commercially available, an expanded access program, etc.) or until the study is terminated by the Sponsor for any reason.

Conditions

• WHIM Syndrome

Interventions

Type	Name	Description
DRUG	X4P-001	Mavorixafor will be provided as either 25 mg or 100 mg capsules.

Timeline

• Start date: 2016-12-01
• Primary completion: 2022-06-16
• Completion: 2022-06-16
• First posted: 2016-12-29
• Last updated: 2024-10-30
• Results posted: 2024-10-30

Locations

2 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study