New drug cocktail aims to boost stem cell harvest for rare immune disease

NCT ID NCT03055247

First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This phase II trial tests whether a combination of ibuprofen, G-CSF, and plerixafor can safely and effectively mobilize stem cells from the bone marrow into the blood in people with X-linked chronic granulomatous disease (X-CGD). Only 3 adults aged 18-45 are being enrolled. The goal is to collect enough stem cells for future gene therapy, which could help control the disease.

What this could mean

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

Active substance
Ibuprofen, G-CSF, and Plerixafor
What this could lead to
If successful, this could improve stem cell collection for gene therapy in X-CGD, potentially enabling a one-time treatment that reduces infections.
What could go wrong
This is a very small early-phase trial with only 3 participants, so results may not apply broadly. The drug combination may cause side effects like bone pain or allergic reactions.

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

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Ospedale Pediatrico Bambino Gesù

    RECRUITING

    Rome, Lazio, 00165, Italy

  • Ospedale San Raffaele

    RECRUITING

    Milan, Lombardy, 20132, Italy