Gene therapy offers hope for boys with severe immune disorder
NCT ID NCT01410019
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial tested a gene therapy for X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where boys are born without a working immune system. Five boys who had no matching bone marrow donor received a single infusion of their own blood stem cells that were genetically corrected in a lab. The goal was to restore their immune defenses and help them fight infections normally.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- autologous CD34+ cells modified with a self-inactivating gammaretroviral vector carrying the IL2RG gene
- What this could lead to
- If successful, this could provide a one-time treatment option for boys with SCID-X1 who lack a suitable bone marrow donor, potentially restoring their immune system.
- What could go wrong
- This is an early-phase trial with only 5 participants, so results may not apply to all patients. Gene therapy carries risks such as insertional mutagenesis or incomplete immune recovery.
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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
-
Hopital Necker
Paris, 75015, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.