Gene-Editing breakthrough aims to fix immune cells in rare disease
NCT ID NCT06325709
First seen Jun 24, 2026 · Last updated Aug 14, 2026 · Updated 14 times
Summary
This early-stage trial tests a new gene-editing approach for X-linked chronic granulomatous disease (X-CGD), a rare immune disorder. Researchers will collect stem cells from 10 adult male participants, use base editing to correct the genetic mutation, and return the cells after mild chemotherapy. The goal is to restore white blood cell function and reduce life-threatening infections. Participants will be followed for 15 years to monitor safety and effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- base-edited autologous hematopoietic stem and progenitor cells
- What this could lead to
- If successful, this could provide a one-time gene repair that restores white blood cell function, reducing severe infections and hospitalizations for people with X-linked CGD.
- What could go wrong
- This is an early-phase trial with only 10 participants, so results may not apply broadly. The chemotherapy conditioning and long-term immune suppression carry risks, and the gene correction may not be durable or effective enough.
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
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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