New gene therapy aims to fix immune system in rare disease

NCT ID NCT06253507

First seen Jun 27, 2026 ยท Last updated Jun 27, 2026

Summary

This study tests a new gene therapy for people with chronic granulomatous disease (CGD), a genetic disorder that weakens the immune system and makes them prone to serious infections. The treatment takes the patient's own blood stem cells, adds a missing gene in the lab, and puts them back into the body. Up to 10 people aged 3 and older will be treated and followed for 3 years to see if the therapy helps control the disease.

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Conditions

The condition(s) this trial relates to.

autosomal recessive disease chronic granulomatous disease granulomatous disease, chronic, autosomal recessive, cytochrome b-positive, type 1 retinitis pigmentosa 47

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States