Gene therapy hope for rare immune disease fails to launch
NCT ID NCT00778882
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial aimed to test a gene therapy for X-linked chronic granulomatous disease, a condition where the immune system cannot fight certain infections. The treatment involved taking the patient's own stem cells, adding a working copy of the defective gene, and returning them to the body. However, the study was withdrawn before any participants were enrolled, so no safety or effectiveness data were collected.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Gene-modified stem cells (VM106)
What this could lead to
If successful, this approach could provide a way to restore immune function in patients with chronic granulomatous disease, reducing severe infections.
What could go wrong
The trial was withdrawn before enrolling anyone, so no results exist. Gene therapy carries risks like insertional mutagenesis and immune reactions.
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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Seoul National University Hospital
Seoul, 110-744, South Korea