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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for X-LINKED CHRONIC GRANULOMATOUS DISEASE are added.
By submitting, you agree to our Terms of use
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
-
Hôpital Necker Enfants Malades
Paris, 75015, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Experimental gene therapy targets rare immune disorder
- Gene therapy hope for rare immune disease fails to launch
- Gene fix trial aims to tame rare immune disease
- Gene-Editing breakthrough aims to fix immune cells in rare disease
- MRNA therapy gives immune cells a temporary boost against rare infection disease
- Gene therapy aims to fix immune system in rare disease