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Experimental gene therapy targets rare immune disorder

NCT ID NCT01855685

Terminated Disease control Sponsor: Genethon Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off infections. The treatment involved taking the patient's own blood stem cells, adding a working copy of the faulty gene, and returning them to the body. The goal was to restore immune function and reduce severe infections and inflammation. Only 3 patients were enrolled before the study was terminated.

What this could mean

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

Active substance
gene therapy (lentiviral vector with GP91PHOX gene)
What this could lead to
If successful, this gene therapy could provide a long-term treatment option for X-CGD patients who lack a donor for bone marrow transplant, reducing severe infections and inflammation.
What could go wrong
This was a very early, small trial (only 3 participants) that was terminated, so results are limited. Gene therapy carries risks like immune reactions or the treatment not lasting.

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.

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.

X-LINKED CHRONIC GRANULOMATOUS DISEASE

Contacts and locations

Locations

  • Great Ormond Street Hospital NHS Foundation Trust

    London, United Kingdom

  • University College London Hospital (UCLH)

    London, NW1 2PG, United Kingdom

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