Bubble boy disease gets a gene fix: new trial offers hope

NCT ID NCT06851767

ENROLLING_BY_INVITATION Disease control Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) Source: ClinicalTrials.gov ↗

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

Summary

This study tests a new gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare immune disorder. Doctors take a person's own stem cells, fix a faulty gene using a technique called base editing, and return the cells to the body. The goal is to rebuild the immune system. The trial includes 18 people aged 3 and older and will follow them for 15 years.

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Conditions

The condition(s) this trial relates to.

T-B+ severe combined immunodeficiency due to gamma chain deficiency X-Linked Combined Immunodeficiency Diseases

As listed by the trial registrant

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

X-LINKED SEVERE COMBINED IMMUNODEFICIENCY X-SCID XSCID

Contacts and locations

Locations

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

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