Rare disease drug shows promise in reducing infections

NCT ID NCT02231879

Completed Disease control Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested two drugs, plerixafor and G-CSF, in 20 people with WHIM syndrome, a rare genetic condition that causes frequent infections and warts. Participants received each drug for a year and tracked their infections. The goal was to see which drug better reduced infection severity. Results showed some participants did better on plerixafor, but others had drug failures, highlighting the need for more research.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Plerixafor (also known as Mozobil) and G-CSF (also known as Neupogen)
What this could lead to: If plerixafor works better than G-CSF, it could offer a new treatment option to reduce severe infections in people with WHIM syndrome.
What could go wrong: This is a small, early-stage trial with only 20 participants, so results may not apply to everyone. Some participants had drug failures on plerixafor, meaning it didn't work for them.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HYPOGAMMAGLOBULINEMIA are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

agammaglobulinemia common wart inborn error of immunity Infections infectious disease neutropenia syndromic agammaglobulinemia WHIM syndrome WHIM syndrome 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HYPOGAMMAGLOBULINEMIA INFECTIONS MYELOKATHEXIS NEUTROPENIA WARTS

Contacts and locations

Locations

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.