Experimental gene therapy aims to fix immune system in rare childhood disease
NCT ID NCT02757911
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off bacteria and fungi. The treatment involved taking the patient's own blood stem cells, adding a corrected gene, and infusing them back. Only 3 participants were enrolled before the study was terminated, so the results are very limited.
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 (autologous CD34+ cells modified with a lentiviral vector carrying the corrected gene)
What this could lead to
If successful, this gene therapy could restore the ability of white blood cells to fight infections, reducing severe infections and inflammation in boys with X-CGD.
What could go wrong
The trial was terminated early with only 3 participants, so results are very limited. Gene therapy carries risks like immune reactions or the modified cells not lasting long-term.
Disclaimer
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This is a summary of
the original study
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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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Hôpital Necker Enfants Malades
Paris, 75015, France