Experimental gene therapy targets rare immune disorder
NCT ID NCT01855685
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.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom
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University College London Hospital (UCLH)
London, NW1 2PG, United Kingdom