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 However, the study was withdrawn before any participants were enrolled, so no safety or effectiveness data were collected.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- The trial was withdrawn before enrolling anyone, so no results exist.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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Seoul National University Hospital
Seoul, 110-744, South Korea
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Newborn screening study aims to catch rare diseases at birth
- Experimental gene therapy targets rare immune disorder
- Gene fix for rare immune disease: can it stop deadly infections?
- Experimental gene therapy aims to fix immune system in rare childhood disease
- Groundbreaking gene therapy aims to fix immune system in rare disease
- New hope for CGD: stem cell transplant trial aims for a cure