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

NCT03995108 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 31

Last updated 2025-07-29

No results posted yet for this study

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.

Conditions

  • WHIM Syndrome

Interventions

DRUG

Mavorixafor

Mavorixafor provided as 100 mg capsules.

DRUG

Placebo

Placebo matching to mavorixafor capsules

Sponsors & Collaborators

  • X4 Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Chief Medical Officer · X4 Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-10-24
Primary Completion
2025-12-31
Completion
2025-12-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Denmark
  • France
  • Hungary
  • Israel
  • Italy
  • Netherlands
  • Russia
  • South Korea
  • Spain
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03995108 on ClinicalTrials.gov