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

WHIM Syndrome Clinical Trial

Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Patients With WHIM Syndrome With Open-Label Extension

Status Active, not recruiting

Clinical Trial Summary

This study has a double-blind, Randomized Placebo-Controlled Period and an Open-Label Period. The primary objective of the Randomized Placebo-Controlled Period is to demonstrate the efficacy of mavorixafor in participants with WHIM syndrome as assessed by increasing levels of circulating neutrophils compared with placebo, and relative to a clinically meaningful threshold. The primary objective of the Open-Label Period is to evaluate the safety and tolerability of mavorixafor in participants with WHIM syndrome. Participants are allowed to continue treatment in the Open-Label Period, if regionally applicable, until mavorixafor becomes commercially available, or until the study is terminated by the Sponsor.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Syndrome
• WHIM Syndrome

• NCT number: NCT03995108
• Study type: Interventional
• Source: X4 Pharmaceuticals
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: October 24, 2019
• Completion date: December 2024