Gene therapy for rare immune disease shows promise in Long-Term study
NCT ID NCT06282432
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 9 people with a rare immune disorder called LAD-I who received a one-time gene therapy that adds a working copy of the ITGB2 gene to their own blood stem cells. Researchers want to see if the treatment safely controls the disease over many years and helps patients avoid a bone marrow transplant. The study tracks survival without transplant, infections, hospital stays, and skin rashes.
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 (modified stem cells with a working ITGB2 gene)
- What this could lead to
- If successful, this could show that a one-time gene therapy safely controls LAD-I for years, avoiding the need for a bone marrow transplant.
- What could go wrong
- This is a very small follow-up study (9 people) with no comparison group, so results may not apply to everyone. Long-term risks of gene therapy are still unknown.
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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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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH, United Kingdom
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095-1489, United States
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