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.

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.

chronic granulomatous disease granulomatous disease, chronic, X-linked

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ospedale Pediatrico Bambino Gesù

    RECRUITING

    Rome, Lazio, 00165, Italy

    Contact

    Contact

  • Ospedale San Raffaele

    RECRUITING

    Milan, Lombardy, 20132, Italy

    Contact

    Contact

    Contact

    Contact

    Contact