Rare disease drug shows promise in reducing infections

NCT ID NCT02231879

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, 20892, United States